GLOBAL ggff forumFEBRUARY 2013 VOL 5 • ISSUE 1
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IN THIS ISSUE Special Section Drug Development in Emerging Regions Part 2
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11% Indian
Non- Indian 89%
INFORM ADVISE REACH EDUCATE ASSOC. NEWS 14 36 44 86 100
Open Forum 4 Mapping Regulatory, Health Technology Andrzej Czarnecki Assessment, and Coverage Decision- Making Processes: A Key to Announcement: Multistakeholder Understanding 28 Statement from DIA Lawrence Liberti Worldwide Executive Director 6 Efficient Regulatory Medical Writing Statement from for Global Submissions in “ICH President, DIA Board of Directors 7 Outlier” Authorities 31 President’s Message 8 Frank Hubbard Ling Su ADVISE 36 We have the answers and solutions to Executive Director’s Message 9 Into the Cloud: New Technology 1 Paul Pomerantz Blowing In Big Benefits to your life science translation questions. Regulated Content Management inform 14 Whether it’s translating an informed consent or linguistically validating a in Life Sciences 37 Clinical Drug Development Jennifer Goldsmith PRO instrument into 52 languages, Corporate Translations can manage in Emerging Markets 15 Divide and Conquer 41 any translation request. Our unique processes, elite translation teams, and Jean Soul-Lawton Martin Magazzolo LQQRYDWLYHWHFKQRORJ\VROXWLRQVKDYHSURYHQWREHPRUHDFFXUDWHHIÀFLHQW Early Clinical Drug Development in and cost-effective than those of our competitors. We understand the India-Opportunities and Challenges 16 reach 44 critical need for quality translations, and we deliver. That’s why Corporate Savita Dhillon Translations is the preferred supplier of ISO 9001:2008 translation and Recent Development of Activities in Latin America linguistic validation solutions to the world’s leading life science companies. Phase 1 Clinical Studies in Korea 18 DIA’s 5th Latin American Min Soo Park Regulatory Conference 45 www.corptransinc.com Clinical Trials in the Commonwealth North America of Independent States (CIS): 1-855-727-6003 Factors for Success (from Feasibility DIA Presents RIM 2013 47 to Close Out) 21 Sarah Powell Andrei Kravchenko Medical & Scientific Maxime Stevens Communications 2013 48 Translation | Linguistic Validation | Desktop Publishing | Translation Memory Management | Document Libraries Sino-American Pharmaceutical Marketing Pharmaceuticals 2013 49 Professionals Association (SAPA): DIA 2013 Keynote Speaker Discusses Bridging the Gap between Industry in China and the United States 26 Innovation 51 Laura Hong “Exhibitor Invites” Return for Sean Zhang DIA 2013 49th Annual Meeting 52 Weiguo Dai “Hello, But I Must Be Going Now” 53 Bin Shi Keith Wenzel DIA President’s Personal Invitation to Students 55
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The Global Forum (ISSN: 1944-1991) is a publication of the Drug Information Association. Editorial Office: Drug Information Association (DIA), 800 Enterprise Road, Suite 200, Horsham, PA 19044-3595, USA; phone - 215.442.6100; PUBLISHING fax: 215.442.6199. INFORMATION Copyright © 2012, Drug Information Association.
The Global Forum (ISSN: 1944-1991) is published six times a year, in February, April, June, August, October, and December. Paul Pomerantz, Executive Director Periodical postage paid at Horsham, Andrzej Czarnecki, MD, PhD, DSc, Pennsylvania, and additional mailing offices. Editor-in-Chief Thirteen dollars of each member’s annual Global Forum Staff membership fee is for a year’s subscription. Judy Connors, Associate Director, Prices include postage and are subject to Editorial Services change without notice. Notify DIA eight weeks Chris Slawecki, Senior Copywriter in advance of address change with a copy Jenny Kimbel, Senior Editorial Assistant of the mailing label. Back issues of most Joe Krasowski, Sr. Digital Content Strategist previously published issues are available from DIA.
Mission PUBLICATIONS MAIL The Global Forum provides a multidisciplinary, AGREEMENT NO. 41103506 neutral forum for communicating information 2 RETURN UNDELIVERABLE CANADIAN related to drug development and lifecycle ADDRESSES TO CIRCULATION DEPARTMENT, management on a global basis. The Global PO BOX 1051, FORT ERIE, ONTARIO L2A 6C7 Forum disseminates content that is relevant to Postmaster: Send changes of address to members’ professional experiences, including Global Forum, 800 Enterprise Road, Suite 200, international industry and regulatory updates VOL 5 ISSUE 1 Horsham, PA 19044-3595, USA. and news of the association and its programs. The magazine is circulated six times a year as a Design: Imagevolution - 610.867.4460 benefit of DIA membership.
DIA is a neutral organization that does not Publishing and Subscription: advocate for or against any issue. The views Drug Information Association (DIA), expressed by the individual authors or 800 Enterprise Road interviewees in the Global Forum are theirs and Suite 200, Horsham, PA 19044-3595, USA. do not necessarily represent the views of the Drug Information Association. Contact Information for Advertising: Mike Boucher Influence Media Director, Client Publishing Services The DIA Global Forum 267-419-8735 CONTENTS
EUROPE Report on the DIA Europe Training Course 57 Amsterdam On-Location 59 Jenny Kimbel EuroMeeting Themes Drive the Debate in Medicines Development 65 Robin Gallon
CHINA DIA China Successfully Held ICH Pharmacovigilance Training 71 Cocktail Therapy: Victory in Just One Battle 73 Donglei Mao HSA & MEB Sign MOU at Asia Regulatory Conference 2013 76
japan 9th Japan Annual Meeting Reflections 77 Junichi Nishino
india Association News 100 Gene Based Therapies: DIA is pleased to announce Ethical Challenges in India 80 Stephen P. Spielberg, MD, PhD, 3 Arun D. Bhatt former FDA Deputy Commissioner for Medical Products & Tobacco, EDUCATE 86 as TIRS Editor-in-Chief effective March 1 101 PROFILE Beatriz Vicén Banzo 87 FDLI Honors DIA Board Member Patient Perspective Minnie Baylor-Henry 103 Cynthia Ryan, Vestibular DIA Welcomes New Disorders Association (VEDA) 89 Director of Finance/CFO 104 Karen Ball, Sturge-Weber Bayard Gardineer Foundation (SWF) 91 DIA Welcomes New Director, SIAC Corner 94 DIA Europe 106 New SIAC for Medical Science Jytte Lyngvig Liaisons 94 GOVERNANCE Corner Don’t Be Caught off Guard, DIA 2012-2013 Philanthropy Grants 110 Meeting Tips for DIA’s Annual Meeting 96 Members on the Move 112 Lisa Palladino Kim SIAC Name Change 98 gf GLOBAL FORUM
OPEN FORUM
Innovations in DIA Publications and Regulatory Science
The New Year has brought with The other previously announced it a change in DIA publications, change, the members would have which affect the Global Forum and already seen when they received other communication sources. the paper copy of Therapeutic Those members that read the Innovation & Regulatory Science electronic version of Global Forum (TIRS), a replacement of the 4 will not notice and will not be Drug Information Journal that surprised by the disappearance has been with us for over 40 of our paper edition. The change years. It is a continuation of DIA to a paperless version has been scientific publishing, but it is also announced on the title page of a reflection of a change in focus
VOL 5 ISSUE 1 the last several issues of 2012. and adjustment to the current and This move will be accompanied future needs of membership. It is by a better access to the forum a welcome modification of the DIA ANDRZEJ content via other electronic media, publication focus, which creates a e.g. a tablet, android,etc. With this solid foundation for the provision CZARNECKI change we have reached the end of information and communication of an era during which many of us to the outside world in years to kept copies of issues of GF on the come. shelf for future reference on many topics, which this publication This issue’s Special Topic in the has covered in the Special Topic INFORM section has been put section over the last 4 years. together, just as in the previous From now on the ‘shelf’ will be issues, by Jean Soul-Lawton (Big on the DIA website or in a form ‘Thank you’ to Jean from the of personal electronic storage. Editorial Board, myself and the readers). It provides a second part of the update on ‘Emerging Markets.’ The majority of articles cover the conduct of clinical trials in several ‘emerging’ countries. Those readers that remember the implementation of GCP, and where and how CTs were conducted some 25 years ago, and since views on outcomes of regulatory then, can only admire how big decisions. With a much clearer a change ‘emerging’ countries understanding of fast evolving Global Forum have gone through during these science and regulatory science, past few decades. This section we may see a much more positive Editorial Board also includes an article on Health and constant change in the field Technology Assessment (HTA), of drug innovations supported Andrzej Czarnecki, MD, PhD, DSc which presents an interesting by the regulatory science. This Editor-in-Chief tool that allows for comparison development, and applicability of between the markets. this science is very high on the Richard Chamberlain, PhD agenda of regulatory authorities, ECS, Inc. The pharmaceutical sector has which has been very clearly achieved substantial progress in Ronald D. Fitzmartin, PhD, MBA indicated in the two profiles of Dr. FDA all its activities. The change also Gerald Dal Pan and Dr. Tatsuya applies widely to the regulatory Kondo published in the Global Alberto Grignolo, PhD environment where it is driven Forum issues last year. PAREXEL partly by legal aspects, but predominantly by the development Welcoming the New Year, we Justina A. Molzon, MS Pharm, JD of science. No surprise therefore look with optimism on a positive CDER, FDA that the co-chairperson of our future of drug development led by Euromeeting, to be held next a close collaboration of scientists Sarah Powell 5 month in Amsterdam, Beatriz from academia, regulatory and Thomson Reuters Vincen Banzo, in her profile industry, and supported by interview, clearly states that patients, members from all these Nancy D. Smith, PhD “those involved in regulatory representations brainstorming Potomac, MD affairs require continuous and discussing ideas on the training in regulations but also independent platform provided Jean H. Soul-Lawton, DPhil in associated science” and by the DIA in meetings and GlaxoSmithKline therefore a ‘must’ for this kind developing publications. of job is a “continuous update in Veronica Todaro the understanding of scientific/ Happy New Year to all. Parkinson’s Disease Foundation technological innovations.” From J. Rick Turner, PhD this perspective the DIA scientific Quintiles journal TIRS, new by design and broadened scope, is very well positioned to publish scientific information, which will allow readers to follow the progress in the important areas of innovation looked at and seen from the perspective of regulatory science.
Many of us remember the Oxford debate on the European regulatory environment and achievements (“DIA Oxford Debate and Future of Medicines,” GF June 2011), which clearly expressed the audience’s gf GLOBAL FORUM
Announcement Statement from DIA Worldwide Executive Director
It is with mixed emotions that I DIA was founded nearly 50 years announce my departure as DIA ago with a singular purpose: Worldwide Executive Director, to create a neutral forum to effective March 3, 2013. It has discuss scientific issues in the been a privilege and honor to development, regulation, and serve DIA, and I am committed use of medical products. In this to working with the DIA purpose, there is a clear passion Board of Directors and Global for the health of patients and the Management Team to facilitate the improvement of their lives. It is a organization’s seamless transition passion that is evident every day to new executive leadership. in the work of our volunteers and staff, and I am proud to count you The last three years as DIA’s as colleagues. 6 executive director have been among the most rewarding of I did not take my decision to my professional career. DIA is an resign lightly. In March, I will outstanding organization, led by assume a position as the first dedicated volunteers and supported CEO for the American Society VOL 5 ISSUE 1 by a committed and competent of Anesthesiologists, a rapidly professional staff. During our time growing, influential, and member- together, we have ushered in a driven organization. This position Pula new era of organizational stability, at ASA represents an exciting Pomerantz expanded our offerings around opportunity to advance the Worldwide Executive the globe, developed new content educational, scientific, and policy Director, DIA areas, and enhanced stakeholder goals of the organization. It is not relations, particularly among a position that I sought, but it is patients, students, and global one I found I could not decline. health organizations. During this transition period, As a result of our strategic plan please feel free to contact me. and the hard work of many Thank you for the opportunity to individuals, DIA is continuing its be part of DIA. I look forward to successful transformation into a staying in touch and offer my best world-class, knowledge-driven wishes for your future success. organization. Much remains to be done, but DIA is in an excellent Sincerely, position and in very good hands. By following the course of action embodied in our strategic plan, I am confident that DIA will continue to prosper and grow. Paul Pomerantz Worldwide Executive Director, DIA ANNOUNCEMENT Statement from Ling Su, PhD President, DIA Board of Directors
Dear Colleagues, manage a smooth transition and continue building on our 50-year As you know, DIA has announced history. We will soon start a search that Worldwide Executive for the new executive director. I Director Paul Pomerantz will can assure you that the board and leave his position with DIA staff are committed to continuing effective March 3, 2013. Paul our direction and programs in has accepted a position as the service of our members and CEO for the American Society stakeholders. In the meantime, of Anesthesiologists based in please let me know if you have Chicago and Washington, D.C. any concerns. This is an exciting opportunity to lead an association of health care I thank you for your support and professionals that he felt he could look forward to continuing to work 7 not decline. with you.
Paul’s leadership during his Sincerely yours, three years with DIA has laid the groundwork for our current and future success. Under his direction, we have achieved rapid Ling SU global expansion and significantly President, DIA increased our stakeholder relationships. DIA is becoming a Ling Su, PhD Board of Directors world-class, knowledge-driven President, DIA Board of Directors organization. We thank Paul for his service and wish him the best of success in his new position.
While Paul will be missed, we are confident in the ability of our Board of Directors, Global Management Team, and staff to gf GLOBAL FORUM
PRESIDENT’S MESSAGE
Emerging Regions and New Challenges This issue of your Global Forum rest of the Board of Directors, and continues the discussions of drug our worldwide staff, I have greatly development in emerging regions enjoyed working with Paul during that began in our previous issue my time as President-elect and (Volume 4, Issue 6, December now Board President. 2012), which focused on the While I turn this page into a new Asia-Pacific region. I invite you chapter for DIA with personal to read the second part of this sadness, I also turn it with two-part series, which explores confidence because I know that 8 national and regional aspects of we have worked with Paul to drug development in Russia, India, ensure that DIA as an organization Korea and elsewhere. maintains a strong foundation. We This focus on every region of the have a refined strategic direction world continues to sharpen DIA’s for the near-and long-term future,
VOL 5 ISSUE 1 focus on stakeholders – members, which we finalized in the strategic volunteers, staff and supporters – planning exercise at our recent in every region, and on developing Board of Directors meeting in Ling SU pertinent education and training Beijing. President, DIA opportunities to them no matter We have a strong management where they are located. As a result team at worldwide headquarters Board of Directors of their efforts, I can invite you as well as within each DIA region. to such in-person opportunities I am personally fortunate to have as our 4th Pharmacovigilance a unique place in DIA’s leadership Conference or 6th Regulatory continuum and continue to rely Conference in India, our 25th upon, and be extremely grateful EuroMeeting: Amsterdam 2013, for, the good and practical advice and programs in the US that will that comes from consulting with advance medical and scientific Immediate Past President Dr. Yves communications, and marketing Juillet and President-elect Minnie pharmaceuticals. Or join us through Baylor-Henry. We expect this your internet connection for training continuity to drive our activities and in benefit/risk assessment and value to our membership. communication, how to prepare for a safety inspection and other online Let’s expect to face changes and programs. challenges, but let’s also expect to find opportunities in them. I also invite you to join me and extend best wishes to Paul Pomerantz as he moves into a new chapter of his professional life as Chief Executive Officer of the American Society of Anesthesiologists. Along with the W orldWIDE Executive Director’s Message
By now you are aware that I will brings together so many diverse soon transition from my position interests to solve the scientific as DIA Worldwide Executive and regulatory challenges in the Director to become Chief development and use of medical Executive Officer for the American products. DIA has been and will Society of Anesthesiologists remain a vibrant and essential effective March 3, 2013. This part of the health care industry change returns me to the arenas and those this industry serves. in which I have spent most of Over the years, DIA has had many my career: Health care delivery, Presidents and many Executive policy, and medical continuing Directors. Despite changes in education and research. While I people, DIA has remained faithful am very enthusiastic about my to the values of its founders: new position, I will miss DIA, my Neutrality, multi-stakeholder 9 volunteer and staff colleagues, engagement, global views and and most of all, our important scientific integrity. work. It has been a tremendous honor to serve DIA for the last However, DIA is at an inflection three years. In this final column, I point. We are a reflection of the would like to reflect on our years community we serve and are together. likewise challenged by the same PAUL economic, organizational and I feel privileged to have helped societal pressures including health POMERANTZ shape DIA at a critical point in its care reform, industry consolidation Executive Director 50 year history. This is a unique and restructuring, globalization, and outstanding organization and the impact of new technology. that has strived to make, and has For DIA to achieve our purpose made, a difference in the quality in this new landscape, a very of health care and the lives of different structure and strategy patients. No other organization will be required. DIA’s Board of Directors and staff leadership have been working towards this end and you will see ongoing reports on DIA’s strategy in your Global Forum. gf GLOBAL FORUM
A professional transition always Globalization: When I started at fellowships to our EuroMeeting provides an opportunity for DIA, DIA was a North American years ago – but I am glad to have reflection. While much remains organization with international helped build the value of the to be accomplished, I do believe outreach. Today, I believe DIA has patient and patient engagement that our work over the last three become a truly global organization with DIA in North America and years has achieved much to reflected by our representative globally. build the foundation for DIA’s board and global rotation of transformation. Four critical presidents, global management Transformation from an Events- areas involve people, expanded team, and regional offices. More based to Knowledge-driven stakeholder engagement, than this, our global outlook and Organization: Today’s challenges globalization and transformation strategy has engaged DIA with are clear. People have less time to a knowledge-driven scientific other like-minded organizations and funds available than in years organization. such as the WHO, ICH, World past, and their need for timely, Bank and Gates Foundation, all relevant information to support Management Team: According of whom view DIA as a partner decision-making is continuous and to Jim Collins, author of Good in identifying and solving global urgent. To truly meet the needs to Great, the most important health care issues, and as a of our modern stakeholders, DIA imperative of any organization is partner in training and capacity must implement new means to “getting the right people on the building. engage our stakeholders and bus, the wrong people off the to make relevant information bus, and the right people in the Stakeholder Engagement: DIA available in “real time.” We must right seats.” I feel that we have has traditionally been viewed determine how DIA can help 10 built an “all-star” management as an organization of industry address key issues through a team. Each one has excelled in and regulatory professionals. In knowledge agenda and identify their career prior to DIA. The mix recent years, DIA has engaged the ways that we can leverage and of skills, cultures and attributes is voice of the patient as an equal deliver this content across our creating a talent cocktail of sorts, stakeholder in innovation, product multiple platforms. This transition
VOL 5 ISSUE 1 and unbeatable synergy. Our new development, and regulatory requires a reshaping of how DIA additions, Director for DIA Europe policy. The patient is now engaged works. It impacts our website, our (Dr. Jytte Lyngvig) and new CFO in DIA in many ways, through programs and our publications; (Bayard Gardineer III) complement our DIA Europe and North but more profoundly, this will our regional executives in China, America fellowship programs; as change DIA’s role in the world India, Japan, Latin America volunteers in our communities and of science. DIA has started to and North America and global as speakers, panelists, editorial engage this transformation in executives in headquarters for board members and authors; several ways: Human Resources, Information and, increasingly, on our advisory Technology and Operations (see councils and soon on our board. First, the Drug Information sidebar). This team has inspired The patient voice is essential to Journal has been re-launched and challenged me, and I believe decision-making and policy on as Therapeutic Innovation & they have inspired our members, medical products and regulation. Regulatory Science, and has just volunteers, other stakeholders and Patient engagement has announced its new Editor-in- staff, too. significantly strengthened DIA’s Chief, Dr. Stephen Spielberg, to relevance in today’s environment. help strengthen our journal as a primary source for applied science For this I claim no credit – DIA’s surrounding medical products. patient initiatives began with Dr. Yves Juillet’s vision for patient Second, our ongoing digital strategy has resulted in improvements to DIA’s website and community networking (such as DIA ConneX). Starting with this issue, your Global Forum will be “digital only.” Emphasis will be Now, a few closing personal placed on the development of reflections: DIA Worldwide DIA’s regional web capability and mobile sites. Long term, DIA is First, I greatly enjoyed this Management Team exploring the means to become experience and the thing I enjoyed a first-in-class web-based the most was meeting members Carlos E. Fulcher, MBA knowledge center. at our events. The conversations Worldwide Deputy Executive we had in receptions and Director Third, DIA has begun to engage hallways, about your experience Bayard Gardineer III, CPA stakeholders in defining our needs from serving on a committee, or Worldwide Director and knowledge in emerging an article you read or session you Finance/CFO areas. I’d like to briefly mention just attended, was really the best Elizabeth Lincoln, MA two specific new areas: We part. That’s really what I enjoyed Worldwide Director have elevated awareness that the most. And although we have Human Resources regulatory approval is no longer a long way to go, I’m very proud Jane Y. Cai, PhD the finish line, but that pricing of the chapter that we’ve written Director, DIA China and reimbursement and post- together during my tenure at DIA. marketing safety must be critical Jytte Lyngvig, PhD considerations in modern medical Anesthesiologists work extensively Director, DIA Europe product development. We have with pharmaceuticals and devices, Kaushik Desai, MPharm also helped to elevate and and they’re very concerned about Director, DIA India product safety and quality, and illuminate the interface between Ko Sekiguchi, MBA the integrity of the supply chain. drugs and devices, and have Director, DIA Japan 11 made clear that DIA plans to be a I will stay involved with DIA in my Susan Cantrell, RPh key voice as we learn how drugs, next position with ASA because Director, DIA North America devices, and even IT and other that does something I’ve always types of technology, can work wanted to do – build stronger together for the patients’ good. connections between healthcare These are still in their early stages providers and DIA. This next step but will certainly form part of our is one more chapter in working future. with DIA: I’ll become a DIA member. Many challenges remain. We all want DIA to become well-known and well-understood. How do we establish DIA as a “brand” as well-known as the WHO and other global organizations? When we review membership data, it is clear that people engage with DIA through certain seasons of their career. But how do we get them to view DIA as their professional career partner? For DIA, these will be keys to a successful future. gf GLOBAL FORUM
paul Pomerantz Worldwide Executive Director
12 VOL 5 ISSUE 1 13 INFORMINFORM ggff
Clinical Drug 11% Indian
Non- Development in Indian Emerging Markets 89%
INFORM now houses the Special
Sections: Devised to educate
and update you on advances in a
specific area of research, drugs,
diagnostics or devices. Clinical Drug Development in Emerging Markets
Jean Soul- Lawton 15 DPhil, is a In the previous edition of the are reviews of running phase Director in Global Forum we introduced this 1 trials in India and Korea, and the Respira- topic (Part 1), reviewing some we gain an insight into running tory Medicines aspects of the practicalities of studies in Eastern European Discovery clinical drug development in countries including the Ukraine. and Develop- Emerging Markets. The Asia We also hear about the differing Pacific region was a focus, and requirements for clinical regulatory ment Group at included in the section was a submissions in ‘ICH Outlier’ GlaxoSmith- review of venues for collaboration countries, and the efforts to Kline. She has of regulatory agencies and other identify common features of more than 20 bodies such as the World Health health technology assessment years’ experi- Organisation (WHO), co-location agencies and their processes. ence work- of regulatory agencies and science Also included are the activities of ing in Clinical parks, practical experiences in the Sino-American Pharmaceutical departments the regulatory review process for Professionals Association (SAPA) within the clinical trial applications, and the and its efforts to bridge the gap pharmaceutical establishment of investigator sites. between the industries in China industry. and the USA. Related to this In Part 2 of this special topic, special section on Emerging we look at the practicalities of Markets, you can also read in this running studies in different phases issue of the Global Forum, in the and countries, the preparation Latin America section of REACH, of marketing applications for about the plans for the 5th Latin regulatory review, the complexities American Regulatory Conference of health technology assessments, (LARC) to be held in May in and efforts to bring together Bogota, Colombia, with a theme pharmaceutical development of ‘Pharma-Co-Vergence.’ professionals within and across regions. Specifically included gf GLOBAL FORUM | INFORM
Early Clinical Drug Development in India-Opportunities and Challenges
11% Indian
Savita Dhillon, MD is Medical Director, Medanta Non- 16 Duke Research Institute (MDRI), a joint venture clinical research Indian collaboration between Duke 89% University and Medanta –The Medicity in India. VOL 5 ISSUE 1 Before joining MDRI, Dr. Savita
was with Johnson & Johnson Fig: Proportion of origin of molecules in Phase I trials registered with CTRI on 14th June 2012 Medical India with oversight for all clinical trials including inves- In the last 15 years, India has conduct of repeat Phase I studies tigator-initiated projects. Prior to emerged as a major destination for compounds which have that, Dr. Savita worked as group for clinical trials. According to already undergone Phase I studies leader for Ranbaxy Laboratories the Clinical Trial Registry India outside India. Phase IIa studies (CTRI), the total number of phase I could be done concurrently in India and was involved with the studies registered as of 14th June India as part of a global trial. evaluation of clinical lead candi- 2012 was 127; the total number dates for feasibility of develop- of ongoing studies was 42. Out of India brings potential opportunities ment. Savita has deep expertise the total number of phase I trials, for carrying out clinical trials, in drug and device development 89% were compounds developed in terms of good recruitment and in particular clinical devel- in India. speed, reduced costs and opment and regulatory affairs. increases in health and allied As described in the Indian infrastructure1. Along with these Dr. Savita received her MD from regulations, first-in-human clinical advantages, there are some P.G.I.M.S, India and a degree in studies for compounds developed unique opportunities which India executive program in Business outside India are not allowed; brings of relevance to early phase Management from IIIM, Calcutta. however, it would be possible to trials like access to a largely do a first-in-human study in India treatment- naïve population with if the compound is of medical a high rate of advanced disease, relevance to the Indian population patient populations of orphan or if it addresses an unmet need. diseases and a diverse genomic The regulations allow for the pool. As well as opportunities for development of niche busters (orphan drugs), there are understanding and interpretation well as the independence of the opportunities for re-purposing of of how to handle Indian versus ethics committee involved and the existing drug molecules, for foreign compounds2. its review and decision-making exploratory biomarker studies and procedures3. for pharmacogenomic studies1. The regulatory infrastructure is still evolving and therefore There have been challenges in The regulatory scenarios for early sometimes clinical trial approvals the participant recruitment and drug development in force today can be delayed. There have consenting processes due to are depicted in the table below been announcements by the social, economic and cultural and need to be more facilitative: Indian government that there diversity in India. The commitment will be a strengthening in level and time devoted by the clinicians in the conduct of clinical POSSIBLE SCENARIOS PERMISSION FOR CONDUCT OF trials have been a concern, CLINICAL TRIAL IN INDIA though this appears to have been improving with reinforcement Phase I for the compounds developed Yes in India of good clinical practice (GCP) training and growing regulations. Phase I for the compounds which Yes have already undergone Phase I Trials In conclusion, in view of all outside India the challenges, some of the Phase I for the compounds which Possible but Agency comfort level recommendations to the sponsors would simultaneously undergo Phase I is less planning to conduct early phase trial in COO* & India trials for their molecules in India 17 are: First-in-human studies for compounds No developed outside India • Be rigorous in the site First-in-human studies for compounds Possible identification, qualification and developed outside India but also ad- selection process dresses an unmet medical need in India • Preferably engage with Phase IIa for any compound Yes accredited sites Microdosing for any compound No • Ensure that the institutional • Country Of Origin ethics committee for the project clinical trial regulatory capacity has the expertise in reviewing There are certain regulatory, soon. In general, the regulatory early phase clinical studies operational and ethical challenges requirements for early phase related to conduct of early research are more restrictive than • Ensure that there is audio- phase clinical trials in India. facilitative when compared with visual recording of the Indian regulatory authorities other global regulatory bodies. informed consent process are in the process of building and a questionnaire to check up the infrastructure required There are few specific confined participants’ comprehension to monitor the clinical trials research units in India which have and understanding regarding as strictly as is desirable. The access to patients and are located study information regulatory authorities have come in hospitals, and few experienced up with various regulations in physicians and clinical research • Plan a few co-monitoring visits the last five years. Still there is a professionals in early phase trials. • Discuss all the clinical studies need for laws on compensation, with the regulatory authorities punishment for defaulters and In the inspections conducted prior to study start declaration of “conflicts of by the Central Drugs Standard Control Organization (CDSCO) in interest” by investigators and • Raise public awareness of the India in 2011, Indian regulatory members of ethics committees. research With the increasing number of authorities reported irregularities partnerships and co-development related to subject recruitment and References/citations from this article there is a need to have a uniform informed consent processes as are available upon request. gf GLOBAL FORUM | INFORM
Recent Development of Activities in Phase 1 Clinical Studies in Korea
Min Soo Park 18
VOL 5 ISSUE 1 Over the past 10 years, the a decade has pushed its way trend towards globalization of up to its current status as a clinical drug development has leading country in Asia. Part of received a great amount of the reason for this is that Korea attention as well as scrutiny; has been involved in a public- nevertheless it has become a private partnership (PPP) model. widely accepted phenomenon in Launched in December 2007, the global community. Starting the Korea National Enterprise for with successes in late phase Clinical Trials (KoNECT), a not- development, Asian countries for-profit organization supported have emerged as an attractive by the Ministry of Health and region for global development, Welfare, led the initiative. As a evidenced by a rapid growth in part of the infrastructure-building the number of trials placed there. project, KoNECT selected, Among the Asian countries that through competition, and have shown a capability in drug supported the establishment of development, Korea has been 15 regional clinical trials Centers one of the ‘late comers.’ Korea of Excellence that continuously first appeared in the global arena underwent improvement through in the early 2000s and within annual evaluations to meet the global industry standards. The Clinical Trials Training Academy at KoNECT has operated nationwide for suitable sites only within With a booming growth in training programs to meet specific advanced countries. Proof-of- research and development professional needs since 2008. concept and early phase clinical activities of domestic There are programs targeted pharmacology studies, previously pharmaceutical companies, many for clinical investigators, clinical regarded as milestones that can of the phase 1 units in Korea have research coordinators (CRCs), only be achieved by sites in core acquired a substantial amount clinical research associates countries, have recently started of experience and expertise in (CRAs), trial pharmacists, to find their place in certain sites a rather short period. Analysis clinical pharmacologists, outside core countries. There of phase 1 studies approved by pharmaceutical medicine specialists, biostatisticians, and pharmacoepidemiology and data management specialists. Currently KoNECT runs certification systems for physician investigators, CRCs and CRAs. Research grants were made available for development of new trial technologies. To protect and ensure the safety of research participants and quality of data, regulatory agencies as well as ethics committees at individual sites strengthened the 19 regulations as well as tightening the surveillance on the ethical conduct of trials. Investigative sites, mostly university hospitals, reshaped their internal oversight Figure 1. and governance systems. With respect to the quality of studies, is a slow but definite change in Korea FDA in 2011 shows over the official reports of the USFDA the pattern of different phases of 60% involved pharmacokinetic inspections have shown that there studies placed in the Asian region, or drug interaction components. was almost no difference between and notably in Korea (Figure 1). There were 20 first-in-human sites in North America and sites in The PPP model in Korea has studies, 23 phase 1 studies Asia. This all-out effort resulted in helped domestic pharmaceutical in cancer patients, and 8 high-quality data, ethical conduct, companies develop new drugs by pharmacokinetic studies in non- and reliable performance in global providing Centers of Excellence cancer patient populations. clinical trials. fit for early phase trials in Korea Unlike most studies from global without the need to go the core pharmaceutical companies, many In terms of early phase clinical countries. Most of the clinical trials studies from domestic companies development, most global centers out of the 15 regional require services from the phase 1 pharmaceutical companies have CTCs have set up outstanding units ranging from study design heavily, if not almost exclusively, phase 1 units that deserve global and protocol development to depended on the core countries, recognition in terms of operating pharmacokinetic analysis and mainly the US and EU, until systems, facilities, personnel, clinical study report writing. recently. The critical value of and clinical pharmacology and In addition, in most of these the data obtained from early technical support. Many global units there are full time clinical phase trials, especially phase 1 pharmaceutical companies that pharmacologists and clinical studies, along with the difficulty, have successfully run late phase investigators as well as teams complexity, and intensity of these clinical trials in Korea have started of research nurses dedicated to studies have traditionally made to bring early phase studies to early phase studies. To this end, pharmaceutical companies look certain sites in Korea. it is important to recognize the gf GLOBAL FORUM | INFORM
active roles that the members of phase 1 facilities and are within In summary, despite a relatively the Korean Society for Clinical the university hospital setting short history of clinical Pharmacology and Therapeutics with easy access to most of the development, Korea has proved have played in drug development hospital medical care facilities itself capable in late phase in Korea. Their strong academic and services. Many university development and has established and research activities along hospitals have huge medical care an environment suitable for with active participation as key facilities with mostly greater than early phase trials. With its well- opinion leaders in the policy- 2,000 beds and daily outpatient built infrastructure, training making and project planning populations of up to 10,000. They systems, experienced personnel, by the government have had also have a strong IT environment growing expertise, awareness a great influence in the PPP including electronic medical record for patient safety and efficient model. Most of these units have systems that facilitate database- operating systems supported already acquired internationally driven feasibility assessment and by IT technologies, Korea looks acceptable quality standards promote development of next- forward to contributing further to for human research protection generation electronic data capture the crucial, early phases of global programs or institutional review systems. drug development. board (IRB) activities as shown by the Association for the Accreditation of Human Research Protection Programs (AAHRPP) Min Soo Park, MD, PhD accreditation or the Forum for graduated from Yonsei University College of Medicine in Ethical Review Committees in the 20 Seoul, Korea, and was trained in Pediatrics and Neonatol- Asian and Western Pacific Region ogy at Severance Hospital, Yonsei University. He received his (FERCAP) recognition. These Master of Science degree in Clinical Pharmacology at Univer- units have had the opportunity to sity of Aberdeen, UK, and PhD in Medicine at Ajou University, conduct a wide variety of early Korea. He is currently Associate Professor in Pediatrics and phase studies including first- VOL 5 ISSUE 1 in-human studies that involve Clinical Pharmacology at Yonsei University College of Medi- healthy volunteers as well as cine and holds a dual position as Professor at Yonsei Uni- patient populations with various versity School of Pharmacy. As the Director of Clinical Trials diseases. They are confident and Center of Severance Hospital, Yonsei University College of ready to prove their competency, Medicine, he is in charge of the governance and support of competitiveness and excellence clinical research activities. He is currently Vice President of in the global community. Another the Korean Society of Pharmaceutical Medicine and a co- important aspect to be highlighted director of Joint Graduate Program of Pharmaceutical Medi- is the high quality medical care cine and Regulatory Sciences at Yonsei University School of systems in many major university Pharmacy and College of Medicine. hospitals in Korea. There are a few university hospitals that have been accredited by the Joint Commission for International Health Care Quality Standards for Patient Care and Organization Management. Most clinical trials centers in Korea have dedicated Clinical Trials in the Commonwealth of Independent States (CIS): Factors for Success (from Feasibility to Close Out)
Andrei Kravchenko 21
Summary The health care services in Ukraine and Russia were built with Clinical research processes are the aim of providing a service, becoming more complex, resulting free of charge, for the population. Maxime in an increase in the regulatory Taking into account the low gross Stevens burden, recruitment challenges domestic product (GDP) per and an increase in costs. Today, capita (Table 1) in these countries, the USA is still the region where expensive medications and costly the majority of clinical trials are health care services cannot always designed and executed; however, be covered by the state budget. many other geographical areas Thus, participation in clinical trials are becoming involved in drug has been desired by patients with development, bringing more and severe and chronic diseases, and more data to the “general clinical in oncology diseases, for example, trials dataset.” The peculiarities approximately 165,000 people of clinical trials in one of these are diagnosed every year in emerging regions, namely the Ukraine alone, making the country Commonwealth of Independent attractive for clinical trials that States (CIS), with a focus on require centres for recruitment in Ukraine (that is an unofficial CIS this disease area. member state), are discussed in this article. gf GLOBAL FORUM | INFORM
Table 1. GDP, Per Capita in US Dollars (2012) trials. The majority of CRPs hold a Medical Doctor’s degree and Russian Ukraine Belarus United this facilitates communication Federation States between site staff and monitoring GDP per 10,351 3,035 5,702 46,546 personnel, which leads to better Capita, $ data quality.
Another similar statistic for CIS Russia, Ukraine and Belarus can When considering Ukraine as a countries is health expenditure. broadly be described as countries model of a CIS country for running This parameter is broadly defined that have a high recruitment clinical trials, the following factors as activities, the primary purpose potential. This is because they will facilitate the achievement of of which is to promote, restore, have large populations that the best results at each step of or maintain health1. Total health are concentrated in big cities the process. expenditure as a percentage of that would enable fast patient 1. Feasibility stage. Local GDP for 2009 in Russia, Ukraine recruitment. They also have Clinical Research professionals are and Belarus was 5.4%, 7% and centralized medical services very efficient in contacting sites, 5.8% respectively, in comparison and broad networks of large and sites are very responsive due with 16.2% in USA. specialized clinics with patient to the ease of communication registers and referral schemes. Thus, health care systems in all between medical doctors from the CIS countries have similarities; However, there are differences clinical research organizations this is especially the case in in the size of the population, (CROs) and sites who speak 22 Russia, Ukraine and Belarus infrastructure and resources “the same language.” Even more due to the fact that the systems among Russia, Ukraine and benefits are achieved when derived from the Union of Soviet Belarus, which contribute to the personnel from ongoing or recent Socialist Republics (USSR). The difference in clinical trial records in trials are involved, to give an most prominent characteristics each of the countries2 (Figure 1). indication of feasibility. VOL 5 ISSUE 1 of the system are centralization, 2. Study start. state coverage of medical Preparation of insurance, established networks of the documents health care settings for particular for submission pathologies and their respective to Competent databases (e.g. in oncology, skin Authorities and venereal diseases, psycho- (for the clinical neurology diseases). trial approval) Other common characteristics should include a in terms of the environment for preliminary careful clinical trials in these countries cross-check of the include: content of all the documents and • All of these countries are their translations outside the original three to achieve an International Conference of almost 90% Harmonization (ICH) regions success for timely Figure 1. Number of clinical trials per year study approval • All have confirmed high and therefore, recruitment potential An important peculiarity of the study start. The regulatory clinical trial markets in CIS process is well described for the • The Russian language is respective documents and can widely used in many public countries is the profile of clinical research professionals (CRPs) be tracked. In contrast to many organizations and institutes in other countries, contracting with Ukraine and Belarus. involved in monitoring of clinical study sites may be done after (in other words, only Ukrainian by sites, as this type of activity submission. insurance companies can be used requires licensing and is usually for insurance of the trial subjects’ done by specialized vendors. It is important to mention that health and life in Ukraine). Typically, destruction of study clinical trial agreements (CTAs) materials including IMP in the for clinical trials in Ukraine are An example of improvements in country carrying out the clinical completed for institutes and regulatory process in Ukraine trial is cheaper than returning investigators separately. Often is that recently implemented the materials to the Sponsor for CTAs for study team members changes in clinical trials legislation destruction. (e.g. sub-investigator or study (order of the Ministry of Health of coordinator) are required. In each Ukraine #523 dd. 12 Jul 2012) Historically, the main reasons particular case, the distribution of aim to shorten the Clinical Trial for considering new regions for roles and responsibilities and their application time. A further factor clinical trials were the availability inclusion in the CTAs for each that helps in the regulatory of trial subjects, time and costs. site are made by the Principal process is that dates of the Competition for subjects, along Investigators in agreement with forthcoming meetings at CAs are with escalating research and the management of the institute/ often predictable, and therefore development (R&D) costs, has clinic. submissions 35 - 40 days prior forced many major pharmaceutical to the targeted meeting at the CA companies to look towards Preparation of documents has leads to the most time-efficient emerging markets to conduct their some peculiarities, such as the results if no additional requests clinical studies. These markets requirement for an Apostille from the CA are made. offered an opportunity to reduce for Delegation of Authority. R&D time and costs if managed 23 An Apostille is required for Following receipt of the clinical appropriately, and permitted Competent Authorities (CAs) to trial approval by the CA, an access to large, treatment naïve confirm that the applicant of the application for an import license populations.3 The major emerging clinical trial has rights to act on must be made immediately. It is regions are still Eastern Europe, behalf of the Sponsor, and the important to mention that Russia Asia and Latin America.4 The Apostille is a “version” of a legal and Belarus are members of a Commonwealth of Independent confirmation that is performed customs union that ease logistics States (CIS), formed in 1991, is for official documents circulated in clinical trials. Ukraine requires located in two of these emerging between different countries. separate logistics including regions (Asia and Eastern Europe) Legal confirmation is required for customs clearance. Customs and includes Russia, Belarus, documents circulated between procedures are different in the Armenia, Azerbaijan, Uzbekistan, countries if at least one of them amount of time required and the Kazakhstan, Kyrgyzstan, is not in the list of “Convention of taxation charged for different Moldova, Tajikistan. Ukraine 5 October 1961 Abolishing the types of imported goods, but and Turkmenistan are unofficial Requirement of Legalization for roughly 4 – 6 weeks must be member states of CIS. In addition, Foreign Public Documents.” An allowed for getting permissions Russia, Belarus and Kazakhstan additional time saving at this stage and importation of study materials entered into a Customs Union is achieved by learning which including Investigational Medicinal (http://www.tsouz.ru/Docs/ documents can be in the Russian Products (IMP). kodeks/Pages/default.aspx) which language. This minimizes the plays an important role in the While amount of translation needed, and 3. Study conclusion. importation of goods, especially coming to the end of the study, maintains consistency with clinical for clinical trials. trial applications in other CIS it is advisable to prepare an countries. Special attention must archiving plan and allocating some Utilizing its medical and be paid to insurance of study time to its implementation with demographic potential in the subjects. Insurance for subjects site staff. It is important that IMP mid-nineties, Russia was the participating in clinical trials must (used and unused) is returned to first of the ex-USSR countries be done by an insurance company the Sponsor/CRO or destroyed. to become a viable location for that is resident in the country Destruction cannot be carried out international multicenter clinical gf GLOBAL FORUM | INFORM
trials. Around that time the first hospitals (431,000 hospital beds, subjects are equally accessible. clinical trial investigators started 9.4 per 1000), and provides an Table 2 shows some factors that to work under Investigational New excellent background for the influence a subject’s accessibility. Drug (IND) regulations. By the country to enter the clinical trial end of the century, the number arena and take one of the leading Conclusions of investigators working under positions in the region. The CIS is the biggest community an IND in Russia increased more of countries in the CEE region than 30-fold reaching a total As of the 1991 dissolution of the and is emerging in terms of the number of 170 investigators.5 Soviet Union, Belarus was one number and complexity of the Nowadays, Russia has 63 higher of the world’s most industrially clinical trials performed within its medical schools (universities, developed states by percentage borders. Common historical roots, institutes) and postgraduate of gross domestic product (GDP) similar healthcare processes and education institutions (institutes as well as being the richest CIS 6 procedures, cultural peculiarities and academies) (http://www. state. With 46,965 physicians and language simplify the spruce.ru) and is ranked 8th place (4.87 per 1000 inhabitants, coordination of clinical trials in the in the world by the number of one of the highest in the world) region. Providing detailed study foreign students in the country (http://belstat.gov.by/homep/ start-up plans has assured up to (http://www.russia.edu.ru/edu/ ru/indicators/pressrel/demogr. 90% success in gaining approval inostr/why/). Medicine is the most php), Belarus is attractive for to carry out international clinical in-demand subject for foreign Sponsors to place clinical trials trials in CIS. In addition, a reliable students in Russia (19.2% of all there; however, the number of partnership with companies foreign students in Russia study international trials are lower than 24 having deep local knowledge is medicine) (http://www.russia.edu. in Ukraine and Russia. the best approach for CROs and ru/news/1358). With 715,800 The total population in all three sponsors interested in achieving physicians, Russia also has great CEE countries is close to 200 the best performance in running coverage of the population in million inhabitants; this provides clinical trials in the CIS countries. terms of medical care, and has
VOL 5 ISSUE 1 a large pool of potential clinical one of the world’s highest rates References/citations from this article trial subjects, but not all of these of this parameter (5.1 per 1000 are available upon request. inhabitants) (Russian Federal Statistics State Service, http:/ Table 2. Geography in CIS countries www.gks.ru, end of 2010). Russian Ukraine Belarus Ukraine has approximately 48 Federation million inhabitants and is the Area, sq km 17,098,240 603,700 207,600 second most populated Central Population 8.2 76.2 46.6 Eastern European (CEE) country density per sq after Russia. It is interesting that km even though it is one of the three CIS-forming countries, Ukraine Roadways, km 982,000 169,495 94,797 did not ratify the CIS Charter, and Data from http://data.un.org and https://www.cia.gov officially is not a member of CIS, but nevertheless is participating in CIS activities. Medical resources in Ukraine include 19 higher medical schools and postgraduate education institutions, 225,000 physicians (4.9 per 1000 inhabitants which is one of the highest in the world) and 2800 Andrei Kravchenko, MD, PhD Maxime Stevens
Head of Representative office in Ukraine of Head of International Business Development HCR (Harrison Clinical Research) Deutschland HCR (Harrison Clinical Research) Group GmbH GmbH Max has extensive experience in the surgical, Dr. Andrei Kravchenko began in the pharmaceu- medical device and more recently in the CRO tical industry as a CRA in 2002, and became a environs. She spent many years in the surgical clinical trial manager in 2003. He has served as training field, focusing on both innovative lapa- head of the Ukrainian office of HCR since 2007. roscopic procedures, and later neurological and He holds his PhD in dermatology. vagal therapies for paediatric epilepsy. For two decades, she has built and trained global busi- ness development teams across Europe, MENA and South Africa. She also worked closely with the WHO for many years and sat on the FDA Medical Devices Board.
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Sino-American Pharmaceutical Professionals Association (SAPA): Bridging the Gap between Industry in China and the United States
Laura Hong, Sean Zhang, 26 Weiguo Dai, Bin Shi
VOL 5 ISSUE 1 Over the last decade, there has was founded in 1993. SAPA’s been a shift in the pharmaceutical mission is to promote the industry that is bringing drug advancement of pharmaceutical discovery and development in science and biotechnology, to China to the forefront. As the make contributions benefiting industry evolves to be more global public health education, to in scope, emerging markets play promote scientific exchange and an increasingly important role in business cooperation between pharmaceutical development. US and China, and to foster the Companies in China and other career growth of pharmaceutical emerging markets, with fewer professionals. overheads, deep talent pools, and strong government support, have The need for such a bridge is an opportunity to gain ground by evident given SAPA’s rapid growth growing the industry outside of into a membership of nearly 4000, the United States. including individuals from drug discovery, preclinical sciences, In order to bridge the gap drug product development, clinical between industry professionals research and development (R&D), in the US and China, the Sino- Contract Research Organizations American Pharmaceutical (CRO)/ Contract Manufacture Professionals Association (SAPA) Organizations (CMO), academia, and other professionals across the US, China, Hong Kong, Taiwan, Japan and other Asian Pacific areas. Originally founded in New Jersey of the United States, SAPA with local communities, as well new opportunities and job has spawned several regional as contributing to the China Red openings in emerging markets, organizations including one in Cross for the fight against SARS. and foresee the globalization of New England (SAPA-NE), one in Additionally, SAPA-GP has held the biomedical industry, including Greater Philadelphia (SAPA-GP), a number of investment forums what unprecedented challenges and one in Shanghai, China. for biopharmaceutical companies we may encounter and how we and New Technology/Scientific can prepare for new paradigms SAPA is committed to promoting Districts, such as those for that may emerge in the future. pharmaceutical science and ZhangJiang, Shanghai Pu Dong, technology and its essential Suzhou, Wuxi, Benxi, Chongqing, role in fostering member’s and ShanDong, to name a few. Laura Hong, MD, PhD is career development. In service There are many opportunities for the immediate past president to science, SAPA facilitates Chinese-American professionals of SAPA-GP. Dr. Hong is an communication among scientists, to impact innovation, restructuring Associate Principal Scientist at policy makers, government and globalization within the Merck Research Laboratories officers, educators, and journalists pharmaceutical industry. SAPA’s specialized in vaccine and bio- from both the United States and membership works to create logic drug development. China through its interdisciplinary a stronger impact by hosting conferences, symposia, scientific meetings, symposiums, Sean Zhang, MD, FCP is the seminars, webinars, workshops member conferences, and current President of SAPA-GP. and diverse publications. SAPA workshops to promote scientific Dr. Zhang is a Medical Director Programs draw upon the foremost exchange and business and Liaison to Bristol-Myers experts and the most current cooperation between the US Squibb (BMS) China R&D at the 27 Department of Discovery Medi- information about advances in and China. In particular, the drug discovery and development cine and Clinical Pharmacology organizations host an Annual (DMCP) of BMS. to inform analysis and discussion Conferences for pharmaceutical of public health issues, and to professionals and executives, promote scientific exchanges and as well as representatives Weiguo Dai, PhD is the Sci- networking opportunities. from the regulatory authorities entific Director and an Inaugural Janssen Fellow in drug product - the State Food and Drug Jingsong Wang, MD, Head development at Johnson and Administration (SFDA) of China of China R&D and Head of Johnson. Dr. Dai is currently the and the US Food and Drug Translational Medicine, Asia President-Elect of SAPA-GP, Administration (FDA). This allows Pacific at Sanofi, speaks highly and also serves as 2012-2013 for the exchange of ideas and of SAPA and the importance of Vice Chair of Physical Pharma- for the education of attendees cy and Biopharmaceutics (PPB) its mission. “What I like the most on new approaches to increase Section in AAPS. [about SAPA] is the quality of its innovation and productivity in scientific programs, the desire pharmaceutical development. for continued improvement, the Bin Shi, PhD is the General It also provides a forum for caring it has for its members, and Secretary and Chair of Media discussing current trends in the broad professional networks Communication of SAPA-GP. emerging markets. As bridging Dr. Shi is a Research Fellow at across both the US and China,” platforms, SAPA organizations Merck Research Laboratories said Wang, who previously served provide venues for the Chinese and leads multiple projects as President of SAPA-GP. national biopharmaceutical in RNAi therapeutics, In vivo industry entrepreneurs, Pharmacology and Oncology SAPA’s various programs aim franchises. not only to benefit its members, government officials and but also the community at large. academic professionals to meet An example is SAPA’s work to their overseas counterparts in organize educational seminars the US. It also helps to explore on severe acute respiratory syndrome (SARS) in conjunction gf GLOBAL FORUM | INFORM
Mapping Regulatory, Health Technology Assessment, and Coverage Decision-Making Processes: A Key to Multistakeholder Understanding
A need for clarity abilities to pay and to allocate resources. This diversity adds It has been established that the to the complexity of medicines’ most important aspects of the development and clouds the regulatory review of medicines transparency and predictability are predictability, transparency, of the HTA and coverage body timeliness, and quality. It has also review process, ultimately been recognised that the health resulting in unequal access to Lawrence technology assessment (HTA) of medicines for the 501 million Liberti these medicines can benefit from patients in Europe. these same characteristics. 28 In fact, in a recent review and HTA agencies all have a reimbursement survey conducted common goal: the efficient by the Centre for Innovation allocation of scarce resources in Regulatory Science (CIRS), in the face of increasing health industry respondents indicated VOL 5 ISSUE 1 care demands, new medical that a high level of transparency technologies and ageing (94%) existed for regulatory populations. However, this goal requirements while an extremely is accomplished through many low level of transparency (13%) different approaches, and unlike surrounded the requirements for regulatory submission and review HTA. processes for which regional and international standards have Furthermore, a lack of timeliness been developed, procedures in HTA can contribute to for HTA and coverage bodies significant delays in patient remain fragmented, with often access to new medicines. This is heterogeneous requirements. For particularly true in countries with example, although there is only emerging pharmaceutical markets one regulatory agency whose where adding the time required for decisions apply across Europe, HTA assessment to that country’s there are over 30 HTA agencies Lag Time (the delay in submission at national and regional levels to an emerging market country with different processes and from the time of approval in the requirements and more than 30 first market) and the subsequent coverage bodies with differing regulatory review time in that market, can result in years of unequal access to important new therapies. If regulatory reviews can to inform efficient and timely HTA conduct those functions in order be aligned on a regional or activity3. Ultimately, this would to show where in the system jurisdictional basis by focusing enable the identification of best such activities occur and how on the elements of common practices across HTAs from which they relate to one another. science, can HTA decision- emerging agencies could chose, making frameworks be similarly allowing them to build their own • In step three, a “task bar” of aligned, based on scientific high-quality, efficient, science- key activities is developed principles and the use of based, bespoke systems. for the HTA function in order common data? Several national to characterise a selection of and international initiatives are Advances in Process defining elements of the HTA now underway to examine this Mapping process. These activities are issue, but these have not yet Scientific Advice, Therapeutic Since 2010, CIRS has undertaken examined the subject in detail Value, Economic Value, a project to systematically map in the Emerging Markets. This Reimbursement Rate, Public the processes in jurisdictions gap is largely due to the nascent Consultation, and Coverage that have formal HTA activities state of the HTA development in with Evidence Development. to elucidate the organization of these economies, where currently Each activity is given an different health care systems only a handful of countries, – identifying icon that is shown in and to allow the systems to be Indonesia, South Korea, Taiwan the HTA task bar if that activity compared with one another. It was and Malaysia – have formal is a normal part of that agency’s envisioned that these maps could HTA agencies that undertake actions. be used as a straightforward single technology assessment. instrument to visually convey Using this process, CIRS While the other countries with 29 the structure and differences has begun to identify key emerging pharmaceutical markets of various HTA and coverage similarities and differences also necessarily conduct value systems to all stakeholders with among HTA agencies. Variability assessments of new medicines, an interest in healthcare. The CIRS in the connection between the formal procedures have often HTA Process Maps are developed assessment of a medicine’s not yet been codified. As these in a stepwise fashion. therapeutic and economic value, countries begin to assess other the government’s role in the established HTA processes upon • In the first step, agencies evaluation process, the structural which to model their emerging involved in the HTA process setting of decision makers and systems, a clear understanding are identified and their status how evidence flows to support of the specific HTA processes as government or independent decision making were among undertaken by other countries and bodies is specified. External the differences identified by the their relevance to the Emerging connections between the construction of the Process Markets would be of great sponsor of a new medicine Maps. General similarities across benefit1. and the regulatory, HTA and jurisdictions could also be coverage bodies and internal Due to the diversity of HTA observed in HTA processes and connections among the requirements and decision- procedures. agencies are numbered to making processes, it is recognised indicate the typical order in The maps permit simple visual that it is critical to develop a which these contacts occur. comparisons and are extremely clear map of the processes in useful for agency and industry each jurisdiction. This has been • In step two, seven functions that training and educational purposes, undertaken by the International represent significant measurable providing a consistent terminology Society of Pharmacoeconomic key components of the system for cross-stakeholder discussion and Outcomes Research (ISPOR) are defined: Regulator, Market (Figure 1). For industry, the to provide a general overview Authorisation, HTA, Price potential impact of HTA mapping of key aspects of HTA activities Authority, Recommender, on product life cycle management in selected countries2. In turn, Decision Maker and Provider. is significant: it identifies science- this process mapping can help These functions are then establish appropriate benchmarks mapped onto the agencies that gf GLOBAL FORUM | INFORM
based HTA requirements transparent science-based HTA HTA requirements during the applicable to all jurisdictions while recommendations. development and review of new characterizing regional variations medicines. in requirements and processes. Summary and the way Process changes over time can forward Enabling stakeholder be assessed using a consistent understanding of the processes Despite the fact that HTA and mapping approach. For agencies, surrounding the movement coverage decision making the granular mapping of HTA of scientific evidence from processes and practices activities permits benchmarking development through the are heterogeneous across and identification of best practices registration and reimbursement jurisdictions, these systems and has the potential to streamline represents a significant step in share many common elements and enhance both HTA and achieving expedited and equal that can form the basis for regulatory review processes. international patient access to activity benchmarking and Additionally, the mapping system innovative medicines. best-practice identification, offers the rapidly evolving enhancing the quality, timeliness, References/citations from this article emerging market counties a way transparency and predictability are available upon request. to assess how best to organise of decision-making processes.4 their agencies and how to apply Multistakeholder initiatives are their limited resources to the LAWRENCE E. LIBERTI, underway that are looking to align goal of providing efficient and MSc, RPh, RAC,has worked for the past 34 years in and with the pharmaceutical in- 30 dustry, in the fields of regula- Figure 1. Process Maps as a comparative tool tory affairs and clinical R&D.
Since 2009 he has served as the Executive Director of VOL 5 ISSUE 1 CIRS (the Centre for Innova- tion in Regulatory Science, Ltd; formerly the CMR Inter- national Institute for Regula- tory Science), an independent division of the IP and Science business of Thomson Re- uters.
Mr. Liberti is a pharmacist with a master’s degree in pharmacognosy (both from the Philadelphia College of Pharmacy and Science). He is a Fellow of the American Medical Writers Association and is a recipient of their Golden Apple award for ex- cellence in teaching. Efficient Regulatory Medical Writing for Global Submissions in “ICH Outlier” Authorities
Frank Hubbard 31
Figure 1. Overview of non-ICH Clinical Deliverables Worldwide
Over the past several years, Taiwan, Korea and the Association Health Authorities, particularly in of South East Asian Nations emerging markets, have required (ASEAN) markets. clinical documents beyond International Conference on Regional/National Harmonization (ICH) Common requirements including subset Technical Document (CTD) in analyses: order to support successful New The Health Authorities from Drug Applications. This includes these local markets are not Health Authorities in markets with signatories to ICH. Thus, it is potentially high patient access not surprising that local/regional needs such as Brazil, China and clinical data requirements to Mexico (Figure 1). Regulatory support marketing approval vary success in these increasingly significantly across these Health important markets is dependent Authorities. Previously, conduct on the Sponsor’s ability to meet of local registration studies may these additional clinical document have been required; more recently and information requirements. greater flexibility has been seen. As an introduction to the topic, For example, a national subgroup this article explores those clinical from within a single multi-national documents required as part of pivotal/non-pivotal phase III trial marketing applications to China, in the relevant patient population may be sufficient to achieve registration. gf GLOBAL FORUM | INFORM
Regardless, what is becoming Controls (CMC)/Nonclinical and essentially be duplicates of the apparent is that expert clinical Clinical) that should incorporate parent global CSR. and regulatory input is required certain specific elements from the to build the optimal package to ICH CTD Clinical Overview and Taiwan demonstrate the relevance of Summary. China Item 28 (Clinical In order to obtain regulatory mainly foreign clinical data to Study Summary provided in Part approval for a new chemical entity these markets as is described IV of the IDL application), is a (NCE) in Taiwan, a Bridging Study below. clinical document meeting the Exemption (BSE) is usually sought China specific format that may be that involves submission of a China substantially based on the Clinical ‘Bridging Study Checklist’ perhaps Overview and Summary. The China Import Drug License prior to the NDA application. The (IDL) is the marketing application China Supplementary ‘Checklist’ requires provision of and incorporates a variety of Clinical Study Report the necessary supporting data clinical programme and study- and includes an assessment level documents (Figure 2); note One caveat to the China IDL is the against ICH E5 Appendix D (A that the format of the China CTP requirement for the provision of a Medicine’s Sensitivity to Ethnic (Clinical Trial Permission) and the standalone supplementary report Factors) criteria. In addition, a IDL is the same. One can view for patients at China recruitment provision of subgroup data is the CTP as analogous to the EU centres. This report is submitted required on Asian patients (e.g., Clinical Trial Application (CTA). in addition to the ICH Clinical Taiwanese/Japanese/Chinese/ The clinical/regulatory Study Report (CSR) from the Korean/Hong Kong) as part of the 32 documentation differences same study. A key requirement is BSE application. between the IDL and ICH CTD are to include an analysis of Chinese magnified within Module 1 and patient data on key safety and The Taiwan Guidelines on Drug especially Module 2. efficacy endpoints. The statistical Review and Registration, Article methods and presentation should 22-1 clarify that “When applying Provided in Part I of the IDL VOL 5 ISSUE 1 broadly follow that of the ICH for bridging study assessment, application, China Items 3 CSR. This supplementary report manufacturers should fill out (Rationale for Drug Development) needs to be a “standalone” the checklist for bridging study and 4 (Summary and Evaluation document as in essence it is assessment and provide a of Major Research Results) are standing in the place of a local complete clinical data package, cross-functional documents registration study CSR. Some preferably with data on ethnic (Chemistry, Manufacturing and sections of the China CSR will groups in Asia.” There is no formal requirement for pooling of Asian patient data but this may be requested by Taiwan Food and Drug Administration (TFDA). Most multinational companies will proactively provide a pooled analysis of the Asian data, especially when few Taiwanese subjects are enrolled in the programme.
South Korea
In order to obtain regulatory approval in South Korea for NCEs, it is necessary to provide Figure 2. Basic Format of China application Package vs CTD in a Bridging Position Paper (BPP) either: • Data from a Korean clinical clinical evidence of lack of ethnic 3, the ACTD differs in format from study to bridge to ‘foreign’ data; sensitivity. the ICH CTD.
• Clinical data from Korea (and ASEAN Countries While the format differs between other Asian) patients to gain a the ICH CTD (5 modules) and bridging study waiver; or Comprised of 10 countries, the ACTD (4 modules), the clinical Association of Southeast Asia content remains the same for • Justification for a bridging data Nations (ASEAN) was established most marketing applications waiver (strict criteria apply) in August 1967 to foster their - no new ASEAN documents overall health and economic are required. One exception The BPP will typically development. In order to facilitate is Vietnam where in some incorporate: trade and investment in the circumstances local Vietnamese region, the ASEAN committee • An assessment in relation to clinical data may need to be on standards and quality has ICH E5; provided in a supplementary CSR. endeavored to harmonize national • Western/Japanese and/or standards with international Conclusion Chinese pharmacokinetic data; standards. The ASEAN CTD (ACTD) is part of the marketing Emerging markets are striving • Primary/secondary objectives authorization application dossier for improved health standards; and safety in Korean patients; that is common for all ASEAN therefore it is vital that the members. Sponsor demonstrates that their • Subgroup analyses data guided drug is safe and effective in the by input from the Korea Food The content of the ASEAN CTD relevant population. While the 33 and Drug Administration (KFDA): Part IV (Clinical) is the same as pharmaceutical industry places an Total vs. Korean, Non-Korean the ICH CTD – no ASEAN specific emphasis on ’emerging markets,’ vs. Korean, Asian vs. Korean, documents are required to be a discrete number of non-ICH Total vs. Asian; and authored. The Clinical Summary clinical deliverables are required to and Overview are included along support regulatory submissions. • Product overview and summary with Clinical Study Reports in Bear in mind that: of the clinical development ASEAN Part IV. As shown in Figure program.
Data on a sufficient number of Korean patients (no clear KFDA definition) are required to support the claim that no additional local studies in Korean patients are required. Waivers are available for certain categories of applications (e.g. orphan drugs), but strict criteria are applied. In general it may be perceived that there is a low probability of success in obtaining a “Bridging Data Waiver” category for NDAs covering solid oral dosage forms, even if an ICH E5 conforming evaluation shows a low likelihood of ethnic sensitivity. Finally, equivalent data from Asian Countries are considered supportive only and are unlikely to Figure 3. ASEAN CTD vs. ICH CTD be directly usable for registration in lieu of Korean data even with gf GLOBAL FORUM | INFORM 2 5 t h A n n u A l
• Requirements vary between countries Frank Hubbard, PhD, is currently a Group Manager within EuroMEEting Scientific Communications at AstraZeneca (AZ) where he manages • Support from Clinical and a staff of Medical Communications Scientists, who lead cross-func- Regulatory functions is essential tional clinical and regulatory authoring teams to develop market • Most documents can be applications for ICH and non-ICH accepting countries. A trained MstErdAM cancer geneticist, Frank possesses an earned doctorate in the life A 2013 provided post NDA/MAA (although subgroup analyses sciences from New York University with over 15+ years in clini- may be identified prospectively cal research experience in oncology, cardiovascular disease, CNS, prior to unblinding of data) respiratory, GI, and functional genomics. He is a recognized expert 4-6 March 2013 | Amsterdam RAI | Netherlands in both ICH Common Technical Documents (CTD) and non-ICH mar- • Science is not the only driver keting applications. behind non-ICH clinical requirements Celebrating our 25th Anniversary – Regulations and the local environment also need to The Annual EuroMeeting is DIA’s largest conference in Europe, attracting more than 3,000 be considered in order to YOU ASKED, WE DELIVERED! professionals from industry, government, academia, as well as patient groups and students. demonstrate the relevance More for your membership! It is unique in the quality of its content and speakers, and in the opportunity to network with of “foreign” clinical data likeminded professionals, regulators and thought-leaders. Expect future change! Plan ahead; 34 consult with your regulatory affairs Make sure you are part of this unique annual opportunity to join fellow representatives to understand professionals at the largest European conference of its kind, featuring: what additional documents and new subgroup analyses will be needed beyond an ICH CTD. VOL 5 ISSUE 1 Take advantage of a new member benefit More than 100 scientific sessions in 17 parallel themes Finally, an encouraging look to designed to expand your knowledge. As part of 200+ exhibiting companies what could be ahead: A “Tripartite your DIA membership, you can now view one of Special hot-topic sessions Agreement” has been forged our best-in-class archived webinars—FREE! REGISTER between Japan’s Ministry of NOW! 18 pre-conference tutorials Health, Labour and Welfare, the Our webinars provide access to diverse topics A whole range of networking events included China SFDA and the Korean FDA to examine the effect of ethnic from anywhere in the world and provide you differences. In the long term, with up-to-the-minute information about the The Exhibition provides a once-a-year opportunity to make contact with the crème-de-la- this collaboration may lead to latest developments in the health care industry. crème of the pharmaceutical world. Multiple networking events provide ample opportunity mutual acceptance of foreign data to meet old and new contacts, discuss sessions, and exchange professional views. generated in these three markets, Visit www.diahome.org/benefits though no definitive outputs are for complete details. Visit www.diahome.org/EM2013 anticipated just yet. to download the complete programme and register online. References/citations from this article are available upon request.
For more information call DIA Europe at +41 61 225 51 51 or email: [email protected] 2 5 t h A n n u A l EuroMEEting AMstErdAM 2013 4-6 March 2013 | Amsterdam RAI | Netherlands
Celebrating our 25th Anniversary
The Annual EuroMeeting is DIA’s largest conference in Europe, attracting more than 3,000 professionals from industry, government, academia, as well as patient groups and students. It is unique in the quality of its content and speakers, and in the opportunity to network with likeminded professionals, regulators and thought-leaders.
Make sure you are part of this unique annual opportunity to join fellow professionals at the largest European conference of its kind, featuring:
More than 100 scientific sessions in 17 parallel themes 200+ exhibiting companies REGISTER Special hot-topic sessions NOW! 18 pre-conference tutorials A whole range of networking events included
The Exhibition provides a once-a-year opportunity to make contact with the crème-de-la- crème of the pharmaceutical world. Multiple networking events provide ample opportunity to meet old and new contacts, discuss sessions, and exchange professional views.
Visit www.diahome.org/EM2013 to download the complete programme and register online.
For more information call DIA Europe at +41 61 225 51 51 or email: [email protected] ADVISEADVISE ggff
ADVISE includes the “how
to” articles you have become
accustomed to reading in the
former Best Practices section:
Time management, skill
development, technology, software
topics and more are examined for
day-to-day implementation in your
own jobs and offices. Into the Cloud: New Technology Blowing In Big Benefits to Regulated Content Management in Life Sciences
Jennifer Goldsmith
Vice President 37 Vault, Veeva Systems
For more than two decades, paced globalization, increasing life sciences organizations compliance and stronger focus on have purchased, configured cost reduction. and deployed a series of ever- expanding and complex content Despite these radical shifts in management systems. At first, the business environment, life these systems had to be heavily sciences content management customized for the unique needs systems have remained largely of life sciences companies. Over unchanged. They are built on time, industry-specific applications the same basic platforms and that better support life sciences- technologies, and they are specific processes have evolved. often only affordable to larger organizations with significant These content management time and resources to invest in tools continue to tack on new systems development projects capabilities, but they have failed and customization. to fundamentally change to meet the new business challenges Consequently, many life sciences facing the life sciences market: organizations have grown greater collaboration, fast- frustrated with the cost and complexity of implementing, maintaining and updating inadequate content management systems — a process that can gf GLOBAL FORUM | ADVISE
often cost hundreds of thousands Collaborate Closely real time via the web, life sciences of dollars every year. Even worse, organizations can work closely smaller companies are left with Over the last five years, the with their partners and affiliates few, if any, good options for the number of external partners (co- around the globe, as well as with implementation of IT systems development partners, clinical various departments across the supporting their regulated content research organizations, co- organization. management processes. marketing partners, ad agencies and more) that life sciences The life sciences industry requires companies employ has increased The Cloud: Open an entirely new solution, a content by as much as 50% or more. The management technology that use of these partners has reduced Collaboration, truly fits the new needs of all life costs and increased speed to Secure Content sciences companies regardless market. However, this new model of their size. One technology has has also created significant When cloud computing was first introduced to the mainstream, emerged that may support this content management challenges, skeptics tried to find vulnerabilities kind of change: cloud computing. specifically surrounding in the security of cloud (See sidebar.) collaboration. applications. However, these concerns were quickly dismissed Cloud Computing for While traditional on-premise as respected organizations Regulated Content content management — from Amazon to the U.S. Management systems often support Department of Defense —began working collaboratively within adopting cloud computing for 38 Cloud computing may be the key an organization, they are everything from the management to regulated content management not designed for external of customer information to in the life sciences industry. One collaboration. It takes too long, the management of sensitive of the biggest reasons is the fact is too complicated and too government materials. that multitenant Software-as-a- expensive to give secure access Service (SaaS) applications built From a content management VOL 5 ISSUE 1 to all external collaborators. in the cloud enable the sharing Rather than deal with these perspective, cloud content of resources, resulting in massive obstacles, companies tend to management solutions often include advanced access-control economies of scale. It’s this simply pull content out of their frameworks that enable secure model that also provides many controlled repositories and of the benefits hailed by pundits authentication, provide advanced distribute it via e-mail or secure logging of user activity and allow such as scalability, flexibility, fast FTP. These methods, however, administrators to control which implementation, low maintenance, are inherently risky because they users have access to highly simple integration, cost efficiency skirt the compliance checks and sensitive documents. Multiple and easy configuration. When it audit trails maintained by the core layers of firewalls also ensure comes to content management content management system. high levels of protection against specifically, the cloud enables life intrusion and identify and close sciences companies to meet the In contrast, cloud-based content security vulnerabilities. needs of the current business management systems allow life climate to collaborate closely, sciences companies to truly Today, cloud applications are often connect globally, comply swiftly collaborate rather than simply considered even more secure and manage costs effectively. administrate. New users, both than other systems because they internal and external, can be enable access to world-class added in minutes, not weeks security infrastructures. or months. By enabling secure access to appropriate content in Administrators simply provide affiliates. This enables content on-premise or hosted systems a secure log-in. Content is access to participants around are difficult and costly to change. visible by all authorized team the globe, thereby eliminating the Even simple changes, such members immediately and common, less secure approach as adding new fields, tracking without significant IT intervention. where central offices email or FTP document distribution and Additionally, the documents documents out to local affiliates to changing security structures stay in a controlled environment make their changes and then back require elaborate system throughout the collabora- again. validation, development, tion process, providing a more installation and deployment. compliant system and This approach resulted in a This can require thousands of reducing risk. lack of visibility (for example, dollars and as long as four to local content changes were not six months to implement. That’s Connect Globally accessible to the central offices too long for teams that need to and vice versa), a proliferation focus on creating, assembling Annual global spending on of uncontrolled content copies and delivering content, rather pharmaceuticals is expected to and a sense of version confusion than implementing and validating grow to nearly $1.2 trillion by as people were unsure which system changes. 2016, according to a report from document version was the most the IMS Institute for Healthcare current. In contrast, by providing In contrast, cloud systems can 1 Informatics. In addition, the equal and immediate access for reduce the time to deploy and IMS reported that annual global all, cloud content management validate such changes by more spending growth will increase from systems avoid these issues, than 50%. This is because cloud $30 billion in 2012 to $70 billion resulting in greater transparency content management systems 39 in 2016. This is driven, overall, and better communication. automatically receive updates as by volume growth in emerging part of the underlying application pharmaceutical markets and Comply Swiftly structure, are easily tailored higher spending by developed to meet changing regulatory From product development nations. requirements through simple to manufacturing, from drug configuration, and significantly Given this explosive growth, safety to marketing, the rate and reduce the time and effort of both small and large life sciences scope of regulatory change and validation by sharing that burden companies will be challenged with reinterpretation are increasing across organizations. The result is managing their content globally. exponentially. Across the less time spent on IT systems and Affiliates around the world, globe, regulatory authorities are more time spent on core business including geographies where fundamentally changing the way in functions. affiliate offices have only a few which life sciences organizations people, will need the same system conduct business. Manage Costs access, speed and performance Effectively that central offices have typically Because much of a life sciences enjoyed. organization’s information Technology is notoriously is contained in the form of expensive, and life sciences Because the cloud is ubiquitous, content such as safety reports, content management systems cloud content management promotional materials and are no exception. But with an systems provide — for the first health authority submissions, increasingly challenging global time — a truly global view of the increasing rate of regulatory how content is created and change presents a special used. Systems in the cloud are challenge for today’s content accessible from wherever users management systems. These have an internet connection. Additionally, cloud solutions are cost-effective enough to deploy to even the smallest local gf GLOBAL FORUM | ADVISE
economic environment, greater and, instead, adopting a highly manage their content today and in competition, expiring patents and predictable pay-as-you-go-model. the future. It is no longer enough a shift from the blockbuster drug Additionally, costly administrative to patch, update and upgrade models of yesterday, companies tasks common with on-premise existing, on-premise systems. must find ways of maximizing systems (upgrades and back-ups, technology effectiveness while for example) are eliminated with Fundamental business change reducing overall costs. cloud technology since there is no requires fundamental technology hardware or software to manage change. New cloud applications Cloud computing makes content on site. All upgrades and back- provide that leap forward – and, management affordable to even ups are handled by the solution according to analysts, are the the smallest organizations or provider. fastest growing technology. For business units by reducing both the first time, companies of all up-front costs, as well as costs 2013: The Year for sizes can meet the challenges of associated with ongoing system Fundamental Change in today and tomorrow, without the updates and maintenance. As a Content Management complexity, cost and uncertainty result, companies typically save of traditional, enterprise on- anywhere from 30% to 50% over Collaborate closely. Connect premise or hosted content on-premise or hosted systems. globally. Comply swiftly. Manage management implementations. costs effectively. These simple How? By completely avoiding requirements are having a References/citations from this the capital costs of servers, profound effect on how life article are available upon request. software and maintenance sciences organizations need to 40 VOL 5 ISSUE 1 Divide and Conquer
Martin Magazzolo
Global Practice 41 Director, Software Solutions Group, CSC Life In an aggressively competitive and fast-changing market, pharma Sciences organizations need to make some tough choices - particularly as growing compliance requirements drain internal resources. Staying ahead means remaining lean and agile - which increasingly means letting go of non-core activities.
Whilst life sciences organizations rethink their business models in are known for being risk-averse, order to stay lean and agile. clinging protectively to their data, this strategy will hold Yet at the same time intensifying them back going forward. With regulatory demands are creating markets showing no immediate new complexity around content sign of recovery and competitive management. In the face of pressures fiercer than ever, these two conflicting scenarios, pharma organizations must now companies are realizing they have no choice but to entrust aspects of their operations to specialist service providers - those offering superior economies of scale in handling non-core activities. gf GLOBAL FORUM | ADVISE
AstraZeneca shows rapid access to new technologies, evolve. Whilst intellectual property the way facilitating easy scalability and needs to be safeguarded, it has leading to substantial cost become clear that strategically AstraZeneca’s chief technology savings. The ability to scale IT organizations now need greater officer Angela Yochem has been infrastructures both up and down visibility than traditional bespoke pretty open about the company’s in terms of capacity is a huge electronic content management need to relax its internal controls, pull for companies — especially solutions have allowed. and to reach out to a wide range when companies will most likely of external sources. Her view is only need to pay for the resources Pharma organizations can take that shifting the responsibility for they actually use — because the reassurance from the fact that certain aspects of IT delivery to services are being provisioned as content as a service, or cloud- third parties frees the company a service. based information delivery, has to focus on opportunities that evolved considerably — to offer improve the business’s bottom As well as introducing additional secure transmission and the ability line. Yochem is not alone either: cost efficiencies — because to partition one company’s data to improve their abilities to staff are freed from repetitive from that of others. This means collaborate and expand mobility administrative burdens — the that data that needs to be secured other large pharma organizations prospect of holding content can be, whilst information vital to too are making significant changes somewhere external and central a collaborative effort can be made to the ways they function, having offers other benefits. In the life available to authorized parties via recognized that such capabilities sciences industry, information a common but secure Internet- as content management are is continually being created, based framework — removing a 42 commodity components of updated, used and read by layer of complexity and permitting their business, not competitive multiple people in different the participants in a project to differentiators. departments and locations. access the same common core of The more global markets have data directly. Harnessing external services adds become, the more widely content
VOL 5 ISSUE 1 up too. Buying or building vast, needs to be shared. Whatever Planning is key costly internal solutions that cater companies feel about entrusting Any shift from internal control to to peak workloads is wasteful. valued assets to third parties, content as a service requires a Owning systems and running them there is now a competitive willingness on the parts of IT and carry cost burdens too, in terms imperative that scientists and business leaders to surrender a of license fees, maintenance and thought leaders have seamless certain amount of control over upgrades. Also, systems’ fitness access to information that has the the location of their data, of for purpose could be short-lived potential to advance discovery. as the needs of the business course. But rather than needing evolve and change. Facilitating advanced control over where data is held, collaboration what companies really need is Whilst outsourcing services have assurance that their data will not always had good reputations, Collaboration is a driver too. This be managed securely and a virtualization technology and is increasing between academia, dedicated managed service cloud-based delivery models have biotech companies and even provider with a purpose-built proven transformational - enabling competitors. With such a diverse data centre will be much better group of people involved in the equipped to ensure this than drug development and marketing an internal IT department ever process today, the ways content could be. is gathered and managed must Where there is resistance to the proposed changes — in defense of people’s jobs, legacy investments and the interwoven business processes linked to those bespoke solutions — cloud- A clear IT strategy is crucial: to accept that content-as-a- based solutions can provide a business processes must be service will lift a costly and way forward here too, offering well thought out and technology onerous burden from the industry. organizations a migration path. solutions considered in terms of both short-term and long-term Those quick to embrace the So long as they plan well. implications and benefits. opportunity will gain most because of the extra agility cloud- Success in adopting content Fueling competitive based solutions offer them. As as a service demands a best- advantage organizations begin to map out practice approach that starts new growth strategies, innovative with the appointment of a high- Any concerns about system thinking and a dynamic, service- level internal sponsor — such performance have been largely based approach to content as the head of research and addressed, as cloud-based management will go a long way in development, chief scientific services have matured and the setting them up to reach out and officer or even CEO — to drive success of software-as-a-service- exploit new markets. and support the initiative through based applications has been all of its phases. Next, the project proven. References/citations from this will need an IT leader — perhaps article are available upon request. an external partner, or the chief Sure, electronic content information officer or chief management is a more complex technology officer. proposition. But increasingly IT leaders — and, close behind, business leaders — are beginning 43
NEW: DIA Online Career Center Whether you are job hunting, want to expand your professional skills and knowledge, or are looking to connect with others in your field, you can find what you need at DIA’s online Career Center. DIA’s newly launched Career Center is designed to meet the professional development needs of our stakeholders around the world. The new Career Center, found at http://www.diahome.org/en-US/Career-Center.aspx, provides comprehensive resources for every stage of your career. The new Career Center features: • Job listings and Information: Job seekers can search health care positions, or post their resume for employers to view. Employers can post positions and find employer strategies.
• Career Resources: Career Corner, a daily online newspaper featuring articles, blog posts, videos, and photos from hundreds of media outlets, is your partner in the job search process and provides many helpful resources to assist you along the way.
• Meetings and Training: This section is designed to help you find solutions to critical issues and challenges. Links to DIA conferences, live and online training courses, webinars, and eLearning modules provide timely and authoritative information to advance the overall medical product industry and individual professional development.
• Membership: Take advantage of all the benefits of membership to network, learn, and lead. This section will point you to free webinars, members-only publications, and other benefits of membership that can help your career.
• Networking and Communities: DIA has many opportunities to interact with a global network of professionals who share your interest in a particular topic. This section will help you navigate our Communities (formerly SIACs), ConneX, and Student and Patient Initiatives. REREAACHCH ggff
latin AMERICA
REACH: Reflects the global nature of DIA and exposes readers to
what we are doing around the
globe and how advances in each
region can have a worldwide
impact. This is where Upcoming
Events now resides, in an easy-
to-read sidebar box for each
region outlining their educational
offerings. latin AMERICA
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D IA’s 5th Latin American Regulatory Conference Leading on Pharma-Co-Vergence: Enhancing regional industry’s collaboration to achieve regulatory convergence After a great success in October The recent expansion and 2012, the fifth edition of the Latin increasing operation of the American Regulatory Conference industry in Latin America has (LARC) is to be held on May 15th- brought a real need for regional 16th, 2013 in Bogota, Colombia. cooperation, hence the focus This year meeting’s focus is of the 5th LARC on pharma-co- pharma-co-vergence and brings vergence. The term — coined in in exciting new features resulting 2012 by DIA’s LARC Scientific from specific regional needs, Committee Co-Chair, Justina feedback from the DIA attendees Molzon, MS Pharm, JD, CAPT. at the past 4th LARC, and DIA’s USPHS, Associate Director global knowledge on current for International Programs, global trends. CDER, FDA — refers to drug regulatory authorities working in a collaborative way to best utilize gf GLOBAL FORUM | REACH
resources for promoting improved agencies and inclusion of current expands operations in Latin public health globally; and stems global topics were taken into America to serve as the only from the English words a) Pharma: account to bring about the most global and impartial forum for all pertaining to pharmaceuticals; b) complete LARC program ever. stakeholders in the region. co: together, joint, jointly, mutually, and c) verging: being on the edge Colombia was selected by DIA’s References/citations from this article or margin of something—the limit 5th LARC Scientific Committee are available upon request. or point beyond which something as the best location this year begins or occurs. since its market is precisely an excellent example of the NORTH AMERICA Fostering “pharma-co-vergence” expansion of the pharmaceutical UPCOMING among the Latin American industry in the region in recent Regulatory agencies and, of years. It became the 4th largest LATINEVE AMERICANTS course, the industry, is a real market in Latin America, valued CONFERENCE need regionally, especially with at US $4.1 billion at 2011 retail the positive sales increase and prices. Additionally, the Pan- Latin American Regulatory forecasts for the industry in Latin American Health Organization Conference (LARC) 2013: “Pharma-Co-Vergence” America. Collaboration is certainly (PAHO) certified in 2011 the May 15-16, 2013 most needed for it to maximize Colombian Regulator, INVIMA, as Bogota, Colombia gains and to evolve in an optimal the National Regulatory Authority way. for Drugs in Latin America. “This 10th Latin American is the highest PAHO qualification, Conference on Clinical Research: 46 The LARC Program in 2013 thereby positioning INVIMA as includes exciting new plenary The Role of Clinical a National Regulatory Authority Research in the Future of and concurrent sessions, tailored within the international sanitary Medicine to the needs of the industry environment.” October 20-22, 2013 regionally and stemming from Maksoud Plaza Hotel São Paulo, Brazil VOL 5 ISSUE 1 discussions with the Latin This year’s conference constitutes American community itself, the 3rd activity in the Andean the past LARC Attendees and country since last year and is a For more information contact Alejandro Bermudez-Del- DIA’s Advisory Council for Latin testament to DIA’s commitment Villar at Alejandro.Bermudez@ America. Likewise, the presence to foster collaboration and dialatinamerica.org. of Latin American Regulatory cooperation regionally, as DIA NORTH AMERICA
Sarah Powell
Liquent, Inc. 47
DIA Presents RIM 2013 Modern computer and other The DIA Regulatory Affairs Special scientific technologies have made Interest Area Community and it possible for drug researchers an expert program committee and developers to generate and have teamed to build upon analyze more data than ever DIA’s inaugural conference, before. In response to louder presented in April 2012, and will calls for patient safety, and the present Regulatory Information multiple review jurisdictions called Management 2013, April 3-4, at into multinational or global clinical the Baltimore (MD) Marriott Inner trials, such data are also subject Harbor at Camden Yards. All to more (and more intense) four committee members have scrutiny during regulatory review returned from last year’s program: and approval than ever before. As Sarah Powell, RAC (Liquent, Inc.); a result, for the past several years, Linda F. Bowen, MS, RAC (Sanofi); regulatory departments have Dominique E. Lagrave, PharmD, been asked to do more than ever MSc (Dendreon Corporation); before, too. and Andrew P. Marr, PhD (Marr Consultancy, Ltd., UK).
This committee has worked with the RA SIAC to develop a program that will address topics of gf GLOBAL FORUM | REACH
essential importance to regulatory compliance within organizations,” peers regarding what is (or is information management (RIM) Sarah concludes. “Just a few not) working with regard to RIM professionals in an environment years ago, this topic was rarely and, perhaps more importantly, of mergers and acquisitions, discussed in this focused of network with each other and multinational corporations, and a forum. Attendees have an exchange contact information to the Food & Drug Administration opportunity to learn from their keep the discussions going after Safety & Innovation Act (FDASIA), the conference.” presented along two tracks, Business Considerations and Tools & Technology. In addition, For more information about Regulatory Information vendors will present the latest Management 2013, please visit http://www.diahome.org/ information management products and services on tabletop exhibits. RIM2013.
“We received some terrific feedback on the evaluation forms from the conference last year. We took this feedback into consideration in planning this year’s conference,” Sarah explains. “For example, speakers will be asked to ensure that their 48 presentations are globally focused and there will be opportunities for attendees to spend more time looking at tools that assist with RIM.” VOL 5 ISSUE 1 “Separating the conference into two tracks allows for the opportunity to provide more information to the participants than a single track would allow,” she continues. “The format allows attendees to attend the sessions Medical & Scientific that are most relevant to their jobs thus ensuring maximum value from attendance at the Communications 2013 conference.” The Medical & Scientific formed just last year (see related Communications 2013 Annual article in this issue under “SIAC Exhibits, business sessions, tools Forum, scheduled for March Corner”). and technology sessions, and 19-21 at the Sheraton Wild plenary sessions are planned Horse Pass Resort & Spa in This meeting has been integrated to combine into a valuable Chandler, AZ, truly reflects to provide content that meets the educational experience for the multidisciplinary and needs of professionals who work every professional who works collaborative nature of modern in pharmaceutical-based medical with regulatory information and drug development, review and and scientific communications its management. “Regulatory approval, as it has been jointly through a single unified offering information management developed by three DIA Special presented along three tracks: continues to receive Interest Area Communities medical communications, acknowledgement for the (SIACs): Medical Communications, medical writing – regulatory and importance it plays in strategic Medical Writing and Medical publication, and medical science decision making and regulatory Science Liaisons, a new SIAC liaisons. “A separate MSL track is where they believe that they have accordance with ICH guidelines extremely important from the nothing more to learn – those on Module 2.5 of a Common sense that we’re now recognized are the ones who I hope say, Technical Document (CTD). as a significant entity within ‘Wow, there’s something different The annual Core Curriculum, the DIA organization,” explains going on here. I might just which delivers comprehensive Dr. David L. Cram (Corcept abandon all my other meetings. and foundational instruction on Therapeutics), a member of the This is the meeting that I want building medical communication MSL SIAC Core Committee. “For to go to!’ We’re hoping we skill sets and how they provide this year’s Medical & Scientific provide something for the very value to internal and external Communications Forum, program experienced as well as for the customers, will also be presented co-chairpersons Rebecca inexperienced MSL professional.” this year. Vermuelen and Ramineh Zoka are both big powerhouses who Prior to this forum, four engender a lot of respect within preconference tutorials For more information the MSL world. It’s important to will provide more detailed about our Medical have that representation be part instruction on quality in & Scientific of and drive this program. In the promotional materials; medical Communications 2013 communication compliance past, most of the attendees at Annual Forum, please these meetings have probably obligations; the art and science visit http://www.diahome. been drug information people of managing a contact center; with a relatively minor group and preparing a Clinical Overview org /Flagship-Meetings/ of MSLs. MSLs have gone to for pharmaceutical products in MedComm2013.aspx. other meetings presented by 49 other companies because those meetings catered to them. Now, topics that you might have seen in those forums are coming into the MSL track for DIA. This has now become our forum.”
What message does David, as part of the MSL SIAC leadership, hope attendees bring back to their workplaces after attending our Medical & Scientific Communications Forum 2013?
“I am hoping that they return and say that this was something that I got a lot out of. Very inexperienced MSLs, who are either just entering industry or have only been there for a short time, don’t know what they don’t Marketing Pharmaceuticals 2013 know. Hopefully, they will come In an era of shrinking health for marketing and promotion – is and be simply dazzled,” David care dollars and a highly more important than ever. The explains. “But I am also hoping competitive market, marketing ongoing implementation of the that the experienced MSLs – the and promoting pharmaceuticals, Affordable Care Act (ACA), the ones who have been around biologics, over-the-counter FDA Safety & Innovation Act for a long time, who have ‘seen drugs and medical devices – (FDASIA) and PDUFA V make this everything’ and are at that point and complying with various marketing environment even more regulatory and legal requirements complicated and challenging. gf GLOBAL FORUM | REACH
Since 1989, DIA has annually evaluation of health developed and presented a economic information marketing conference to advance is clarity concerning the state of the marketing the standard of art (and science) and provide evidence required professionals with practical advice for the proactive on these and myriad ancillary dissemination of issues. This year, DIA will present health economic Marketing Pharmaceuticals 2013: information for Workshop for Regulatory/Legal/ payers/plans, etc., Communications Professionals called ‘competent & Promotional Review Teams, and reliable scientific February 20-21 at the Grand Hyatt evidence,’ and the Washington in Washington, DC. fact that this standard is quite different The program committee includes than the ‘substantial Thomas W. Abrams, MBA, RPh, evidence standard’ Director, Office of Prescription required to support Drug Promotion, CDER, FDA. Mr. other forms of Abrams is scheduled to present company product the CDER update during the “FDA promotion and label Update: Recent Enforcement claims,” explains 50 Actions” session that will open Philomena McArthur this workshop, and participate (Johnson & Johnson in the FDA Q&A session that will Pharmaceutical conclude it. Research & Development L.L.),
VOL 5 ISSUE 1 The committee has developed who will chair one a workshop that will provide of these sessions. compliance, the pre-conference first-hand updates from senior “Contributing to this issue are tutorial OPDP/APLB & Compliance FDA regulators on the level of the lack of clarity regarding what 101: A Primer, taught by Lucy evidence FDA requires for making constitutes appropriate data, a Rose, MBA (Lucy Rose & promotional claims, the status of lack of transparent regulatory Associates, LLC), a longstanding social media guidelines, recent policy in this area, and other member of DIA’s instructional guidance documents and new initiatives underway that intersect faculty, will provide a strong policies being considered, and with these principles, such as introductory foundation to FDA other aspects of the current the proliferation of comparative advertising and promotional regulatory and legal environment effectiveness research and the requirements from the Office of for marketing drugs and devices. need for such ‘real world’ data Prescription Drug Promotion and Health economics are becoming by many different stakeholders. the Advertising & Promotional increasingly important in the In order to push forward, it Labeling Branch, plus the drug development landscape, is important to identify and current regulatory compliance a point this conference will understand these underlying environment. address through sessions titled scientific and analytical issues “Fundamental Issues in the and what companies can do For more information in meeting the many different Regulatory Evaluation of Health about Marketing stakeholder needs despite the Economic Information.” What Pharmaceuticals 2013, are some of these “fundamental complex challenges and lack of issues”? clarity in this area.” please visit http://www. diahome.org/ Flagship- “One of the main issues If you are new or relatively new Meetings/MP2013.aspx. surrounding the regulatory to advertising or promotional DIA 2013 Keynote Speaker Discusses Innovation
As preparations continue for our with effective, novel approaches DIA 2013 49th Annual Meeting: to address them,” Dr. Kraft said. Advancing Therapeutic Innovation “Increasingly, in health care & Regulatory Science, Keynote and biomedicine, this involves 51 Speaker Dr. Daniel Kraft took leveraging and pulling together time from preparing his address different capabilities, technologies to share his thoughts about the and approaches in new ways, meeting theme with the Global particularly at points where Forum. A Stanford- and Harvard- technologies and other inventions trained physician-scientist, converge – for example in mobile, entrepreneur and innovator, big data, artificial intelligence, Dr. Kraft has more than two 3D printing, all the way social decades of experience in clinical networks and user interface practice, biomedical research design.” Daniel and healthcare innovation. He Kraft chairs the Medicine track for “From my mix of clinical Singularity University and also experience, biomedical research serves as Executive Director for and involvement with both FutureMed, a program which traditional biotechnology and the explores convergent, exponentially startup world, I’ve seen the most developing technologies and their exciting and effective innovations potential for biomedicine and happen when the ‘problem’ is health care. well understood from the clinical, regulatory, reimbursement, What does the term “innovation” patient and other perspectives, mean to Dr. Kraft, and how has and the innovations utilize multi- he observed and applied this and cross-disciplinary lessons meaning in the course of his and insights from other fields career? and experiences which often come from outside of traditional “To me, innovation means biomedicine.” understanding and approaching a problem, unmet need, inefficiency “Effective Regulatory Science or other challenge, and coming up is critical to advancement of gf GLOBAL FORUM | REACH
Therapeutic Innovation” because low-cost ‘omics,’ big data and DIA2013 49th Annual Meeting: regulatory frameworks and the analytics, synthetic biology, 3D Advancing Therapeutic Innovation understanding and perceptions printing, the utilization of stem & Regulatory Science will be of them shape how clinicians, cells and regenerative medicine, presented June 23-27 at the scientists, engineers, biologists to catalyze innovation?” Boston (MA) Convention & and others see the ‘box’ and Exhibition Center. how they may need to constrain their innovations and ideas to fit To keep informed with Annual Meeting within them,” Dr. Kraft continued. “The challenge, that regulation updates, please visit http://www.diahome. can sometimes lag technology org/Flagship-Meetings/DIA2013.aspx. and the rapid pace of innovation, remains.”
“Regulatory science can, hopefully, help large systems adapt to foster new therapeutics and approaches while still maintaining safety, efficacy and quality.”
What message is Dr. Kraft hoping 52 to share through his Keynote Address?
“I hope that in my keynote I can inspire attendees to see and approach the world of biomedical VOL 5 ISSUE 1 and healthcare innovation bit differently, by appreciating how various technologies – which in many cases are moving exponentially faster, smaller, cheaper and smarter – can be “Exhibitor Invites” Return for applied to ‘re-invent’ and ‘disrupt’ healthcare and biomedical innovation in new, powerful and DIA 2013 49th Annual Meeting effective ways. How can we Last year’s DIA 2012 48th Annual Through this “Exhibitor Invites” work together and leverage the Meeting in Philadelphia debuted program, DIA was able to explosion of faster computing, DIA’s “Exhibitor Invites” program. promote our DIA 2012 48th artificial intelligence, and mobile “We knew that we were reaching Annual Meeting to prospects in and wearable technologies, with our own customer database with the external contact databases the growth of new fields such as our many meeting promotions,” of participating vendors, outside explains Susan Cantrell, Director, of DIA’s own customer database. DIA North America. “But we Contact lists from the nearly 100 wanted to try to contact a broader vendors who participated in this audience, both because we want program were carefully secured to deepen our existing market and and de-duped not only from the because we believe our customer shared exhibitors’ contact lists base may be shifting.” but from the DIA Annual Meeting registration list. Each participating vendor was encouraged to send three separate emails to this To learn more about this “Exhibitor Invites” program for our DIA merged and purged list, in a 2013 49th Annual Meeting, please visit http://expo.jspargo.com/ template previously developed exhibitor/dia2013marketingsupportopps.pdf or contact Craig. and approved by DIA, to present [email protected] (Companies A-L) or Michele.LaFrance@ two special promotions: A $100 jspargo.com (Companies M-Z). discount off a full or one-day meeting registration, and a $100 one-day only exhibit hall pass. 53 The results of this program: 93 vendors had helped attract 212 “Hello, But I Must Be Going Now” new DIA 2012 attendees! DIA’s Annual Meeting is a true DIA 2013 49th Annual Meeting: collective effort that comes Advancing Therapeutic Innovation together through the dedication & Regulatory Science will continue of a cadre of program committee to expand services available to members; other DIA members and DIA Annual Meeting exhibitors volunteers; and DIA staff. Work to increase their company and on each year’s program actually begins before the previous brand visibility in Boston through Keith year’s meeting has finished and numerous marketing industry Wenzel support opportunities (such continues throughout the year. as digital signage and carpet Perceptive Keith Wenzel (Senior Director overlays) and provide more Informatics at Perceptive Informatics) has return on their booth purchase. served on the DIA Annual Meeting Helping exhibitors more broadly program committee since July communicate the value of their 2010, when work began for the products and services at our 2011 Annual Meeting in Chicago. DIA 2013 49th Annual Meeting He is serving as co-chair of the will facilitate their contribution to Processes & Technologies for the clinical research enterprise, Clinical Research Track for our to therapeutic innovation, upcoming DIA 2013: 49th Annual and ultimately to the patient Meeting: Advancing Therapeutic populations that their research Innovation & Regulatory Science, intends to help. June 23-27 in Boston, MA, and will wrap up his three years of committee service upon its conclusion. gf GLOBAL FORUM | REACH
A frequent speaker for DIA educational offerings in North America and in Europe, Keith also served on the program committee for Measuring Study Endpoints in Multinational Clinical Trials presented by the Study Endpoints Special Interest Area Community (SIAC) in 2009. He continues to Chair the Study Endpoints SIAC Core Committee and is also a member of the eClinical SIAC.
What was the most surprising thing that Keith has learned while serving on the Annual Meeting program committee?
“How difficult it is to utilize and maximize inclusion of the diverse content of the abstract submissions while designing 54 a cohesive track,” he begins. “From the outside looking in, it is difficult to understand that, each year, there are many abstracts that we are unable to use, not
VOL 5 ISSUE 1 because of abstract quality but because of the meeting’s focus converging and collaborating sessions that have real-world case and developing industry trends. in clinical processes – and studies; and, yet, still embracing I have also been very impressed because of increasing topic visionaries and their cutting-edge, with the intelligence and breadth overlap between IT, eClinical, even if not yet proven, solutions.” and depth of industry knowledge validation, study endpoints, of, not only of the program clinical data management and Looking back upon his three years committee, but also the DIA staff. document management – all of of service, Keith says, “It has The DIA staff does an excellent these tracks needed to come been a juggling act of competing job of supporting and coordinating under one umbrella,” Keith priorities on the professional side, the Program Committee through suggests. “In the same way that but that is true of this industry the lengthy, complex process of eClinical Technology is blurring in general. The positives far designing the final program.” the lines between individual outweigh the negatives. I have applications and processes, the been fortunate to make new What changes have had the Clinical Research Processes acquaintances and to learn from most impact on clinical research and Technologies Track needed the expertise of my fellow track processes and technologies to converge to complement, and program chairs. DIA staff during Keith’s service on this enhance and reinforce these sub- does an excellent job of making Committee and how will this tracks. Over the last few years, efficient use of the program Track at DIA 2013 reflect these we’ve placed major emphases committee’s time and setting changes? on: updating the industry on expectations. The effect on my personal life has been minimal, “Out of the need to consolidate tried and true processes and but it seems that I always end up several individual tracks to technologies that have been reviewing abstracts on a plane on reflect the numerous ways in improved or completely been re- my way to or back from vacation!” which multiple disciplines are invented; increasing the number of Student Poster Award Winners with the Student Poster Committee and other DIA leadership at last year’s DIA 2012 Annual Meeting.
DIA
Dear Colleague, President’s In 1995, I presented a student poster to our 31st DIA Annual Meeting, while I was studying for my PhD at the University of North Carolina. My advisor at that time, Dr. Harry Guess, was heavily involved with DIA and served as DIA Board Presi- Personal dent in 1992. I think the topic of my student poster was product registration in China, but I clearly remember my first reaction to the DIA Annual Meeting. It truly opened my eyes to a new world, of new information and new people, and was a Invitation to very good starting experience for a student taking my professional first steps. Information and technology in today’s environment may be totally different com- pared to when I was a student years ago. But DIA still offers students a unique 55 Students and very valuable platform. When I presented my own poster, for example, knowledge about product registration in China was quite limited in the US. The DIA student poster program presents an opportunity to showcase your own expertise to the scientific, academic, regulatory and industry leadership which attends our Annual Meeting from literally all around the world. The Call for Student I later learned that DIA was interested in presenting an educational program in Poster Abstracts will China. Although I was based in the US, I worked remotely with the regulatory agency in China, and so I stepped up to help organize the first DIA meeting in remain open until Monday China (in Beijing, in 1997). I clearly remember the topic was clinical trials and March 11, 2013. To learn statistics because this was the very first time that biostatistics indrug develop- more about this process ment was introduced to the pharmaceutical world in China. Working alongside other DIA members on these and other DIA activities enriched my own skills and or submit your abstract, experiences, helped me evolve and grow, and prepared me to assume more please contact Joanne. leadership roles throughout my career. [email protected] or I graduated in 1996 and have attended every DIA Annual Meeting since 1997. visit http://www.diahome. Our Annual Meeting student poster sessions can help you even more than they org/Flagship-Meetings/ helped me when I was a student many years ago. Please take advantage of this tremendous opportunity by accepting this invitation, which I extend on behalf DIA2013/Meeting- of our entire global multidisciplinary network of DIA members and volunteers, to Program/Posters.aspx. submit a student poster abstract for our upcoming DIA 2013 49th Annual Meet- ing: Advancing Therapeutic Innovation & Regulatory Science, June 23-27 at the Boston Convention & Exhibition Center. Thank you for your time and consideration. Sincerely Yours,
Dr. Ling Su President, DIA Board of Directors Strategic Advisor, Life Sciences, Sidley Austin LLP gf GLOBAL FORUM | REACH
UPCOMING
NORTH EVE AMERICANTS Online Training Course: Regulatory Affairs: Regulatory Information Development of a Clinical The IND, NDA, and Management 2013 Study Report Postmarketing April 3-4 February 5, 7 &12 March 17-20 Baltimore, MD 12:00-2:00PM ET Irvine, CA Register by February 24 to Save Online Training Course: EUROPE Online Training Course: Fundamentals of Project Clinical Statistics for the Risk Management and Safety Management Nonstatistician Communications Strategies April 9, 11, 16 & 18 February 13-15 & 20-21 March 18-19 11:30AM-1:30PM ET Time Varies Horsham, PA Register by February 25 to Save Introduction to Signal Solution Provider Webinar: Detection and Data Mining Oncology Clinical Trial Medical and Scientific April 15 Endpoints - Keys to Communications 2013 Annual Horsham, PA Maximizing Forum Register by March 25 to Save Success in Your Development March 18-21 Programs Chandler, AZ Overview of Drug Development February 14 Register by February 25 to Save April 15 11:00AM-12:30PM ET Horsham, PA Register Online for FREE! Eight-Part Benefit/Risk Register by March 25 to Save 56 Webinar Series: Assessing Marketing Pharmaceuticals the Benefits and Risks of CMC Workshop 2013 2013 Medicines: April 15-17 February 19-21 Part Four: Clinical Judgment Washington, DC Washington, DC and Pragmatic Considerations Register by March 25 to Save for Endpoint
VOL 5 ISSUE 1 Premarketing Clinical Safety Selection and Display in Global Considerations and Pharmacovigilance Benefit/Risk for Regulatory Strategy February 25-26 March 20 Development Horsham, PA 11:00AM-12:30PM ET April 16-17 Register by February 4 to Save Register for Two or More in This Horsham, PA Series and Save 15% Register by March 26 to Save Eight-Part Benefit/Risk Webinar Series: Advanced Clinical Vendor Eight-Part Benefit/Risk Assessing the Benefits and Oversight: Vendor Lifecycle Webinar Series: Assessing Risks of Medicines: Management the Benefits and Risks of Part Three: Framing for March 26-27 Medicines: Benefit/Risk Assessment and Horsham, PA Part Five: Stated Choice Communication Register by March 5 to Save Methods for Valuing Benefits February 27 | 11:00AM-12:30PM ET and Risks Register for Two or More in This Online Training Course: April 24 Series and Save 15% How to Prepare for a Safety 11:00AM-12:30PM ET Inspection Register for Two or More in This The Leadership Experience March 26-28 Series and Save 15% March 5-7 11:00AM-1:00PM ET Horsham, PA DIA/FDA Statistics Forum 2013 April 28-May 1 Regulatory Affairs for North Bethesda, MD Biologics March 11-12 DIA 2013 49th Annual Meeting: Horsham, PA Advancing Therapeutic Register by February 18 to Save Innovation and Regulatory Science June 23-27 | Boston, MA EUROPE
Some of the course participants with their trainer Dr. Erich Hochueli
57
Report on the DIA Europe Training Course Quality by Design for Chemical and Biotech Products – A hands-on course for the pharmaceutical industry and regulators
This new course, held in Vienna, new released ICH Q11 guideline Austria at the end of September ‘Development and Manufacture 2012 provided a comprehensive of Drug Substances (chemical description of the strategies for entities and biotechnological/ developing a product according biological entities).’ to the ICH guidelines ICH Q8, Q9, Q10, and also the brand- In this interactive course, the key elements of Quality by Design for small molecules and biotech products were discussed. Participants learned with practical work on case studies (solid dosage form of a small molecule gf GLOBAL FORUM | REACH
development, QA/QC and manufacturing; Dr. Erich Hochuli, ICB Consulting, Switzerland, an expert in biotech manufacturing; Prof. Dr. Johannes Khinast, Head of the Institute for Process and Particle Engineering, University Christa Wirthumer-Hoche of Technology and Scientific Prof. Johannes Khinast and manufacturing process Director of the Research Centre use of Quality Risk management for a biotech product), how to for Pharmaceutical Engineering in can make the big difference for use Quality Risk Management Austria; together with Dr. Christa competitive manufacturing of drug (QRM), Process Characterisation, Wirthumer-Hoche, head of the substance and drug products, Design of Experiments (DoE), unit for Marketing Authorisation for new drugs and for generics, and Development of a Design and Life cycle Management for small molecules and for Space and Control Strategy. of human medicinal products biotech products. There was a The case study demonstrated at AGES PharmMed (Austrian lot of very positive feedback from that a systematic approach to Medicines and Medical Device the participants. They are very pharmaceutical development is Agency), led the case studies for a enthusiastic about using the newly faster, needs fewer resources and small molecule tablet formulation learned QbD approach in their leads to robust processes. and a monoclonal antibody drug daily lives.” substance. The trainers — Dr. Siegfried Buoyed by the success of this 58 Adam, QA Manager, Hermes Dr. Fritz Erni stressed that, QbD training, DIA Europe will be Pharma, Austria; Dr. Fritz Erni, a “A systematic approach to holding the next training course Switzerland-based industry expert pharmaceutical development with on QbD, with the same trainers, in with long experience in research, science based tools and adequate November 2013 in Dubai. VOL 5 ISSUE 1 Amsterdam On-Location 59 Jenny For those attending DIA’s 25th Annual Kimbel EuroMeeting, March is the perfect time to visit Sr. Editorial Assistant Amsterdam. Popular with visitors from around the world, the city is well-known for its laid-back atmosphere, famous canals, rich history, theatres and museums Visitors can also enjoy cosy Amsterdam is also a thriving urban outdoor terraces, fabulous shops, centre — a pulsing metropolis in and beautiful parks, where there’s a compact cityscape. You’ll find always so much to see and do. that sights in the city centre are all within walking distance, and No other city has so many most are less than 30 minutes by sights per square kilometre. But tram or metro from the conference location at RAI.
Amsterdam is most famous throughout the world for its canals. The reflection of the historic facades, bridges and boats in the water creates a unique atmosphere. But the city’s web of waterways also offers plenty of surprises even for city residents, such as a number gf GLOBAL FORUM | REACH
of canal houses that are open network to visitors, featuring beautiful connecting city interiors dating from the 17th, districts, and 18th or 19th centuries. Perhaps commuters can the gardens hidden behind these choose a variety houses offer the biggest surprise of methods of all - with only the sound of to reach their chirping birds, these gardens offer destination as an oasis of tranquillity, shutting out efficiently as the hustle and bustle of the city possible. This around them. includes the train, tram, metro, bus highlights include the cosy 17th Amsterdam is one of the most and ferry. The Iamsterdam City century Jordaan district, the lively bicycle-friendly cities in the world. Card gives you unlimited use of Pijp with its terrace cafés and Amsterdammers are born on bikes the GVB public tranport system Albert Cuyp Market, the Plantage and you’ll see them whizzing by at for 24, 48 or 72 hours. district, where nature, culture, breakneck speeds. Thanks to the history and science go hand in many bike paths in the city, pedal MUST-SEE SIGHTS hand, the Eastern Docklands power is all you need to uncover boasting modern architecture Which sights shouldn’t you the city’s gems on your own, or as and design, and the up-scale miss? Although Amsterdammers part of a guided bike tour. museum Mecca that is the Oud- believe that the only thing anyone Zuid area. Then why not head to 60 While the Netherlands enjoys “should” be doing is “as they Westerpark area and discover moderate temperatures year- please,” there are a few gems Cultuurpark Westergasfabriek or round, you can (and should!) count on the weather to change quickly, so plan to dress in layers. Traveling Between RAI Conference Centre (Euro- VOL 5 ISSUE 1 We recommend you pack an Meeting Venue) and the City Centre umbrella, a sweater and perhaps even a scarf to be prepared for the From Amsterdam Central Station (CS), take the Am- March weather. It’s also a good stelveen express tram 51 (travel time: 12 minutes, exit idea to bring some comfortable at the Amsterdam RAI station) or tram 4 (travelling time: walking shoes that can handle 30 minutes, exit at the RAI Europaplein). several hours of sightseeing. From the Amstel station, take the Amstelveen express tram 51 (travelling time: 5 minutes) or the bus (route In early March, visitors can see 15, 69 or 169), which will bring you to the RAI within 10 the flower fields beginning to minutes. In this case you should get off at RAI Europap- bloom and trees are turning green. lein. You can expect a wide range of temperatures – perhaps all in one From Amsterdam Sloterdijk station, the best way to reach the RAI is with . day. Rain is quite common, but it’s express tram 50 mixed with gorgeous days when everyone heads outside to soak up the sun. that number among the city’s the imaginative architecture of top sights: famous museums GETTING AROUND THE CITY the Amsterdam School (Museum include the Rijksmuseum, Van Het Schip). Amsterdam Noord Gogh Museum, Anne Frank House Travelling within Amsterdam is is both fascinating for its daring and the Hermitage Amsterdam. straight-forward and convenient. architecture along the banks There’s the equally celebrated The city centre is fairly compact, of the IJ and the historic dike 17th century canal district, with most tourist sites in walking houses in the Nieuwendammerdijk declared a UNESCO World distance from each other. There neighbourhood. The district Heritage Site in 2010. Other is also an extensive transport of Oud-West also has some surprises in store including millions of visitors every year. The historic riding academy Hollandse following sites and monuments Manege, the seven buildings should also be of interest and of United Europe and a giant are an essential part of the bookcase that’s part of a housing Amsterdam experience. estate. Anne Frank is one of Amsterdam’s And speaking about must-see best-known historical figures. sights - don’t miss taking a boat Anne and her family lived in tour through the canals, exploring hiding from the Nazis for more the city by bike, and enjoying than two years in a house on the sunny terrace breaks in between. Prinsengracht. Eventually she was No matter what your interests, deported to the Bergen-Belsen there is plenty to discover. concentration camp in 1945, where she later died at the young MUSEUMS age of 15. Today, Anne’s spirit lives on through her diary and Museums are the main tourist the million visitors who come to attraction in Amsterdam. Most Amsterdam every year to learn famous are the Rijksmuseum, more about her short life. Anne’s Van Gogh Museum and Stedelijk original diary and other notebooks Museum, but there are many are on display in the Anne Frank The Anne Frank more. In fact, Amsterdam has If you go: 61 over fifty museums which attract Huis. Huis is situated in the centre of Amsterdam at Prinsengracht 263-267. It is open daily from 9 a.m. to 7 p.m. and admission is 9 Euros. Trams 13, 14 and Museums under Renovation in 2013 17 and buses 170, 172 and Two of Amsterdam’s most famous museums will be under reno- 174 stop nearby, at the vation in early March during the EuroMeeting. ‘Westermarkt’ stop. Amsterdam’s Rijksmuseum is the largest museum in the Nether- lands. Its collection features some of the country’s most famous The Hermitage Amsterdam is works, including ‘The Night Watch’ by Rembrandt and pieces by the Dutch branch of the world- Vermeer and Frans Hals. The collection also includes Delftware, famous Hermitage in Saint sculptures, Asian art, prints and many other culturally significant Petersburg, Russia. Located on objects. the banks of the Amstel River, The Rijksmuseum will be under renovation until April 2013, the Hermitage Amsterdam is an however a selection of the best-loved Golden Age masterpieces exhibition space and cultural will be on display in the Philips Wing or on the 1st floor of the education centre with a focus museum. A number of pieces will also be displayed at the Rijks- on Russian history and culture. museum branch in Schiphol Airport. Hermitage Amsterdam displays rotating selections of pieces from If you go: Rijksmuseum is located at: P Cornelisz Hooftstr 1, the Hermitage collection in Russia. 1071 CH AMSTERDAM, 020-6747000. It is open daily from 9 These include paintings, graphic a.m. to 6 p.m. Admission is € 14. works, sculptures, applied art and The Van Gogh Museum, which features not only the largest col- archaeological discoveries. During lection of Vincent Van Gogh’s works, but also chronicles his life, the time of the EuroMeeting in will unfortunately be closed to the public in early March. How- March, the Hermitage will also ever, approximately 75 paintings by Van Gogh and other works have 75 paintings by Vincent Van on paper will be on display in the Hermitage Amsterdam. Gogh on display. gf GLOBAL FORUM | REACH
Heineken Experience include a If you go: The Hermitage is mini brewery, a tasting bar, as located at Amstel 51, 1018 EJ. well as the ‘Stable Walk,’ where It is open daily from 10 a.m. visitors can access the stables to 5 p.m. and admission is € to view Heineken’s iconic Shire 17.50. By tram, take line 9 to horses which still deliver beer Waterlooplein. Or take Metro throughout the city. A visit to the 51, 53, or 54 to Waterlooplein, Heineken Experience attraction Nieuwe Herengracht exit. takes about 90 minutes, and two drinks are included in the For a true sense of the city, stroll admission price. through Amsterdam’s charming Canal District and discover the If you go: Located at city’s hidden gems in the canal Stadhouderskade 78, the house museums. The city’s Heineken Experience can be elegant canal-side mansions reached by tram 4, 7, 10, 16, 24, house some of the most the or 25. Admission is € 17. fascinating museums with collections ranging from stately PLACES TO SOAK UP THE to quirky and including everything ATMOSPHERE: from ancient artefacts to cutting- The Canal Ring treads a tight edge art. A great place to start rope between being a tourist exploring Amsterdam’s hidden attraction that requires drinking museums is Het Grachtenhuis and eating facilities and a major (Museum of the Canals). This residential area that needs quiet. recent addition is a tribute to the The Canal Ring includes the city’s Canal District, with multimedia two main nightlife and culture exhibitions showing how the squares. Leidseplein features now grand district is really an such legendary concert halls as engineering marvel built on swamp Paradiso and Melkweg along with land. From here, visitors can easily the more highbrow city theatre explore other museums housed in Stadsschouwburg and multimedia canal mansions and the museum centre De Balie and Melkweg. is even equipped to help them Rembrandtplein has several plan their itineraries. bars which cater to tourists (both international and from the rest Het Grachtenhuis If you go: of Holland), two large nightclubs is located at Herengracht 386. (Escape and Air), and ‘old world’ Admission is €12, or free with an evocative treasures such as De Iamsterdam card. By tram, take Kroon and Café Schiller. But line 1, 2 or 5 to Koningsplein. meanwhile the whole area is For something a little different, dotted with neighbourhood-bound visit the former Heineken Brewery, ‘brown cafes’. a national monument and listed in The Jordaan is probably the the European Routeof Industrial most famous neighbourhood Heritage. Millions of hectolitres of in the Netherlands. Akin to the Heineken beer were brewed here reputation enjoyed by London’s until 1988, when the Heineken Cockneys, this once working- brewery in Zoeterwoude took over class bastion was renowned for production from the Amsterdam tight community bonds, radical brewer. Attractions at the politics and a love for drink and over-the-top sing-a-longs. The Albert Cuypmarkt is of culinary delights. Be sure Gentrification of decades past arguably the best-known and to try at least one of the many worked to attract more galleries, busiest outdoor market in Europe. Dutch specialties on offer in restaurants, specialty shops and It attracts thousands of visitors Amsterdam! upwardly-mobile residents to its every day, and is especially higgledy-piggledy scenic streets. popular on Saturdays. More than Food to try: So what is Dutch Its maze-like streets offer hidden 250 stalls sell everything from food exactly? Generally speaking, courtyards, cafés (both traditional cheese, bike chains and fresh it’s simple, hearty, meat and and trendy), art galleries and produce to fried snacks, shaving potatoes fare. Favourites include pleasantly nosy neighbours. One cream and shoes. The Albert erwtensoep (pea soup with ham also has access to some of the Cuyp Market is a true feast for and smoked sausage), stamppot more scenic outdoor markets in the senses and it’s Amsterdam’s (mashed potatoes mixed with the country. busiest daily market. The market vegetables, served with meat is located in the Pijp district, and gravy) and suddervlees SHOPPING: surrounded by many pleasant (slowly braised meat). Thanks to cafes and small shops. its proximity to the sea, fish and Amsterdam offers an array seafood also feature heavily on of shopping opportunities for If you go: Albert Cuypmarkt is Dutch menus, especially plaice, everyone from the exclusive open Mon-Saturday, 9 a.m. to 5 mackerel, eel, mussels and boutique shopper to the bargain p.m. Take tram lines 4,16, 24 or shrimp. The Dutch are also crazy hunter. 25 to get there. for their friet (chips/fries)! Even upscale restaurants often serve Located in the heart of the city’s AMSTERDAM FOR YOUR their meals with a side of friet and 63 historic canal district, the area TASTE BUDS mayonnaise. of nine narrow streets called the Negen Straatjes (Nine Streets) One of the best things The Dutch have a passion for intersect the main canals between about travelling is getting to cheese, so be sure to sample the Leidsestraat and the Jordaan taste the local cuisine and some, whether served in plain district. The area is dotted with discover new specialties. The cubes with mustard (kaasblokjes), great restaurants, cafés, art Netherlands also has its share or deep-fried with a crispy coating galleries, jewellers, boutiques and antique shopsWith an exceptional array of styles, trends and prices, AMSTERDAM FAST FACTS: this area is truly a shopper’s paradise. • Dating back to the 13th century, Amsterdam began The two main shopping streets as a fishing village and trading centre on the Amstel in Amsterdam’s city centre river are the and the Kalverstraat • There are more bikes in Amsterdam than permanent . Of the two, Leidsestraat residents the Leidsestraat has the more exclusive shops like Filippa K, • Amsterdam’s maze of canals is its most Karen Millen, Paul Warmer and distinguishing feature- Amsterdam actually has more Shoebaloo. The Kalverstraat is canals than Venice, Italy! home to all the shops you would expect to find in a large European • There are 51 museums to visit in the city city like H&M, Mexx, Zara and Esprit. • Amsterdam’s coat of arms, which can be seen all over the city, features the XXX symbol, which To truly experience Amsterdam, symbolises St Andrew’s crosses. check out the open-air neighbourhood markets, which • About six million souvenir clogs are produced in the feature a variety of sights, snacks, Netherlands each year and shopping. gf GLOBAL FORUM | REACH
Artis ZOO: If the weather is nice, visit the oldest zoo in Holland, right in the centre of Amsterdam. It consists of four main areas: Zoo, Planetarium, Botanical Gardens and Geological and Zoological museum. In the zoo itself you will find animals from all over the world. In addition, a unique canal aquarium shows you which animals roam the canals (Kaassoufflés and kaasstengels). favourites. For a more elegant of Amsterdam. You can also tour For something a little fancier, try meal, try Restaurant d’Vijff the peaceful gardens. Located geitenkaaskroketten (a croquette , which occupies five Vlieghen at Plantage Kerklaan 38-40, filled with goat cheese). adjoining 17th century houses at admission is € 18,95. Spuistraat 294-302. Coffee houses: For a taste of Red Light District: The majority authentic Amsterdam flavour and Other things to see: of people have heard about The Dutch culture, don’t miss a visit Wallen — also known as the to an authentic Dutch pub known Beatrixpark: If you find yourself rosse buurt to Amsterdammers as a ‘brown café’ (bruin café). with just a short break from the and the Red Light District to These local watering-holes are a meeting, check out Beatrixpark. 64 visitors — well before their visit. quintessential part of Amsterdam’s One of the nicest parks in The oldest part of Amsterdam, culture – and are the favoured Amsterdam, Beatrixpark was the neighbourhood is chock- haunts of some colourful local originally designed as a romantic full of interesting shops, pubs, personalities. In general, brown park, but after World War II it has been redesigned into the more fantastic restaurants, leaning
VOL 5 ISSUE 1 cafés are laid-back and the fare is gabled houses and the city’s most pretty simple. In addition to local modern style of functionalism. charming canals, in addition to and regional beers, soft drinks The oldest part of the park is the stereotypical red-lit windows and coffee or tea, you can also the nicest, as it kept its original and peep shows. Don’t miss sip jenever – a Dutch spirit similar romantic character influenced by the vibrant Nieuwmarkt square, to gin. Light meals and snacks English parks — with a small lake the gothic Oude Kerk or a walk (hapjes) are also usually on offer. and open landscape. How to get along the centre of Amsterdam’s A typical Dutch pub snack is there: Walking — from the RAI Chinatown, the Zeedijk (also bitterballen (breaded and deep- congress centre, facing the main home to an impressive Buddhist fried balls with a ragout filling), entry to the right and turn left temple). Although it is generally often served with mustard. direction the Amstelhal of the RAI. Pass in front of this building and considered to be a very safe area, care should still be taken when Restaurants: Moeders behind the canal you will see the walking through the quieter streets (Mothers), located at park — it is located at the rear all Rozengracht 251, offers traditional exhibit halls. of the area. There is a strict “no Dutch foods (you can even order photography” policy. a sampling plate of typical dishes) with a fun, informal atmosphere. Helpful Websites: Restaurant Greetje, at Peperstraat 23, is another great For full tourist info: www.iamsterdam.com choice for “home-cooked” Dutch For detailed EuroMeeting information: www.diahome.org/ flavours in a friendly atmosphere. EM2013 For maps and information on public transportation: Tucked in a side street between www.gvb.nl two canals, ’t Zwaantje (Berenstraat 12) is a cosy brown To plan a specific route on public transportation: www.9292.nl café serving no-nonsense local Robin EuroMeeting Themes Drive Gallon, the Debate in Medicines Head of Public 65 Relations, DIA Europe Development The themes at DIA Europe’s 25th With a focus on better public Annual EuroMeeting in Amsterdam health protection, greater in March will examine the issues transparency of the processes, of the day, provide a forum for improving the public perception creating solutions and focus on of the pharmaceutical industry the hot topics that most exercise and the way it is portrayed in the medicines development the media as well as the rational community. For many, the most use of medicinal products, the critical challenge facing the drug proposed areas for discussion development community in the for the EuroMeeting 2013 are current restrictive environment classified into general disciplines throughout Europe is the return including pharmacovigilance and on investment. For small and regulatory affairs for medicinal medium-sized enterprises this products and medical devices, challenge is even more difficult to R&D and clinical trials. overcome. In a rapidly evolving legislative environment, the The scope of presentations will EuroMeeting covers most areas cover the experience gathered of interest for any pharmaceutical after the implementation of the industry professional, providing a new Pharmacovigilance legislative prime opportunity to get a fresh framework, as well as from the update on most advances in drug patients’ and Health Technology R&D, regulatory, and quality of Assessment (HTA) perspective human medicinal products as well – What did happen to the as medical devices. ‘Information to Patients’ initiative from the EU Commission? Experts gf GLOBAL FORUM | REACH
and authorities in the fields will be conditions, specific settings and pharmaceutical legislation since presenting their considerations for other difficulties in performing 1995 — some say even longer. debate. clinical trials in the elderly will be The Pharmacovigilance (PV) discussed in terms of design, theme will look at the impact of The theme Health Technology modelling and recruitment. And these changes and explore how Assessment (HTA)/ practical experience will be both regulators and the industry Sustainability of Health Systems given. The European Commission are rising to the challenges of in Europe is led by Luca De launched the “healthy ageing” implementation. The sessions will Nigro, Coordinator, Drugs initiative and has funded several look at some of the more practical Monitoring Registers Unit, Italian new research projects whose aspects of implementation such Medicines Agency, Italy; and Lidia initial results will be presented as the Pharmacovigilance System Retkowska-Mika, Director Legal and analysed. These cohort Master File (PSMF) but also Department, Offices of Medicinal studies and developments in explore the impact of some of the Products, Medical Devices and the biobanking domain form less tangible elements such as Biocides, Poland. Recent EU innovative new tools for drug transparency and communication pharmacovigilance legislation has development and testing. issues. Additionally, the theme added new instruments which will look at other advances in may be helpful to bring closer Legal/Transparency-Risk PV such as the IMI PROTECT regulatory activities and HTA and Assessment is led by Burkhard initiative relating to benefit/risk this theme will be devoted to Sträter, a Partner at the Kanzlei methodology and the use of new discussion on new developments Sträter law firm in Germany. technologies. in this area. Approval procedures for 66 pharmaceuticals, biosimilars Clinical Research and Gesine Bejeuhr, Regulatory Affairs/ and clinical trials as well as for Development is led by Nermeen Quality, vfa Research-Based paediatric investigation plans Varawalla, Founder and CEO, Pharm Companies, Germany; are based on European public ECCRO, UK; together with Andrei and Markus Pasterk, COO and administrative law. This sets the Kravchenko, Head of Office,
VOL 5 ISSUE 1 VP of Science, International legal framework for scientific Harrison Clinical Research, Prevention Research Institute, assessment and for protection Ukraine. The potential to adopt France will steer Development of intellectual property rights game changing technologies of Medicinal Products for of innovators. The European and innovative methodologies, Paediatric, Elderly and Other Commission’s sector investigation the demand for “real world” Special Populations. Special establishes a new cartel-law- outcome data and the continued populations such as children, based framework for generic drive for efficiency make clinical the elderly or ethnic minorities applications and Regulatory research today more challenging require particular attention Data Protection. The aim of this and exciting than perhaps during drug development. The theme is to provide scientists ever before. These drivers of Paediatric Regulation started and regulators with guidance on change notwithstanding, the slowly to deliver more, better data European approval procedures for relevance of more traditional for medicines used in children. clinical research and marketing approaches to deliver data quality The European Commission’s authorisations. and operational effectiveness conclusions after five years of remains. The sessions in the experience are eagerly awaited Vicki Edwards, Senior Director, Clinical Research theme present and will be discussed. Practical European Pharmacovigilance, the latest thinking and best solutions for questions that often Abbott, UK; and Jan practices in the context of the occur during paediatric clinical Petracek, CEO, Director of current environment, addressing trials will also be presented. Pharmacovigilance Services, both newer trends and reviewing Pharminvent, Czech Republic established approaches. Our changing demography, with lead the Pharmacovigilance a dramatic increase in the elderly theme. Some say that the new Susanne Keitel, Director, European population, means reconsideration pharmacovigilance legislation Directorate for the Quality of of other aspects. Multiple represents the biggest change in Medicines and Healthcare (EDQM), Council of Europe, EU; and the new requirements for Compliance, Senior Director and John Kerridge, Quality Leader, combinations regulated as drugs. Janssen Biologics, Netherlands, EU QA/External GMP Relations, The theme will consider the participants will hear that data Lilly, France are the co-leaders likely reactions of the European about products is exchanged for the theme Quality (including Parliament and Council as well between industry and agencies in Falsified Medicines). Supply as the latest work towards global the context of a dossier. However, chain security and medicine convergence by the International product data or study data is availability, vitally important areas, Medical Device Regulators Forum. also captured in databases not will be discussed in the context Participants from the industry necessarily in context of a specific of the EU Falsified Medicines will learn how to adapt their dossier. Data is often copied, Directive (2011/62/EU) provisions regulatory strategies to avoid any but it would be easier to reuse which are due to implemented in risk of regulatory bottlenecks. As or refer to the authoritative data 2013. Active substance supply, well as providing the regulatory sources for various purposes. The the future of pack serialisation context, practical guidance on theme will also examine how the and the impact of the changes on developing combination products use of standards, standardised the EU Qualified Person operating will be given. In addition, the processes and tools improves in the global environment will development and approval of consistency and reliability of be considered. Additionally, e-Health/tele-health solutions information. This applies to data anticipated guidance on the designed to improve patient managed in conventional ways prevention and management of adherence to their medication and also using methods such as drug shortages will be explored. will be addressed. The future social media. The challenge is The Quality theme will also involve regulation of companion that improved transparency raises experts discussing key technical diagnostics, including the new doubts about the security of 67 topics and examining the future role for notified bodies and the confidential data. of international harmonisation involvement of drug authorities activities concerning in the premarket phase are also Gonzalo Calvo Rojas, President pharmacopoeias and other on the agenda. Case studies of the European Association standards. will provide advice on how to for Clinical Pharmacology and develop companion diagnostics Therapeutics (EACPT), Spain; Devices, In Vitro Diagnostics in compliance with the regulatory and Judith Creba, Head EU and Drug/ Device Combination requirements in the EU and US. Liaison & Policy, Novartis Pharma, Products is jointly led by Switzerland, together lead, Shayesteh Fürst-Ladani, Managing In eTools and Data Involvement of Experts in the Director, SFL Regulatory Affairs Management, experts will Drug Approval Process. There and Scientific Communication, elaborate on how data (content) is a growing perception that Switzerland; and Sinisa Tomiæ, can be seen, processed and involvement of all stakeholders Counselor for European Affairs, interpreted in the context of during the regulatory review Agency for Medicinal Products various purposes. For example, process of drug marketing and Medical Devices, Croatia. the Common Submission authorisation applications is This theme will especially focus European Platform, already a valuable tool for widening on the impact of the European available at the HMA website, the perspective under which Commission’s proposals for the allows a simple and secure the assessment is conducted, regulation of medical devices means of exchange of submission increasing the quality of the and IVDs on the drug/device information between applicants outcome. Experts and patients are combinations, borderline products and regulatory agencies, in a privileged position to provide and companion diagnostics. The reducing the burden for both a reliable and practice-based impact of the most significant industry and regulators. Led by view on how the demonstrated proposed changes to the Hans van Bruggen, Director, and expected benefits and risks EU’s medical device and IVD eCTDConsultancy, Netherlands; of any new therapeutic options Regulations will be assessed, and Rob Middel, Head of are perceived. As with any other including the potential impact R&D Business Development, contribution, transparency in of the new “scrutiny procedure” Pharmaceutical R&D Quality & the process of involvement of gf GLOBAL FORUM | REACH
patients and experts, and in the Marcelino, Member CMDh, Vice President, Head of Clinical management of potential conflict Medicines Evaluation Department, Development Service, Covance, of interest, is paramount. The National Authority of Medicines China, will lead presentations European Regulatory System has and Health Products (INFARMED), and discussions on the multiple made significant steps forward Portugal. challenges of globalisation and in this respect during the last ways to understand and manage years. Discussions will focus on Isabelle Stöckert, Head Global those challenges, turning them identifying the added value of Regulatory Affairs Europe/ into opportunities for more patient and expert contributions. Canada, Bayer Healthcare successful drug development. They will also address the daily Pharmaceuticals, Germany; The aim being to spare limited problems that academic and and Melek Bostanci Önol, Head resources, promote common patient bodies and regulators of Regulatory Affairs & Quality global harmonisation and enhance need to face in order to secure Assurance, Boehringer Ingelheim, healthcare for the benefit of quality and transparency during Turkey, co-lead a fascinating patients. the whole process. theme titled, Effectiveness and Efficiency of the EU Regulatory Regulatory Science contributes In the regulatory affairs business, System — Does new legislation to the scientific basis of regulation most activities and the majority enable innovation and facilitate and decision making. The urge of resources are focused co-operation? This theme of society for transparency and on or around known active provides high-level discussion understanding has caused FDA, substances. During Known Active on the efficiency of the current EMA and its member states to Substances, seven sessions shed legislation and various new put regulatory science high on 68 light on the different aspects of legislative proposals in Europe. their agenda. The spin-off of such known active substances covering It will deal with issues such as: interest is highlighted in this theme regulatory product life-cycle and How will they shape the regulatory led by Christine Gispen-De Wied, the rules for assessment as well system? How can quality be Clinical Coordinator; Member as specific aspects for fixed built into a new legislation? Is it of SAWP, Medicines Evaluation
VOL 5 ISSUE 1 combination medicinal products. effectively implemented? Has it Board (MEB), Netherlands; and Specifically, an approach on reached its primary objective? Hubert Leufkens, Co-Opted known active substances in the Does it improve public health? member, CHMP, Chairman, self-care sector is discussed, Does it increase efficiency and Medicines Evaluation Board as well as a more thorough decrease bureaucracy? Does it (MEB), Netherlands. The first understanding of a known active increase competitiveness? session starts out by setting the substance in terms of biosimilars. scene: What can we learn from Also, in pharmacovigilance Globalisation is one of the regulatory science? What is the legislation, known active drivers that can bring a new scope? What can be gained? substances play a key role and are drug to the global market faster, In the subsequent session, this among the base data elements especially the emerging and is illustrated through regulatory for the new European database developing market. To further procedures, modelling of the on authorised products (EVMPD). speed up the drug development, decision making process, and One session will focus on the the industry, regulators, and communication with consumers. efforts spent to build up this academics are working together The new concept of adaptive database. The view of industry trying to harmonise regulations; licensing is one potential and authority will be presented to standardise quality; to solution considered as a means for all aspects with respect to overcome cultural differences; of providing faster access efficiently fulfilling agencies’ and to better understand various to medicines, for example in legal demands, safe resources, markets; to drive cross-national meeting unmet needs, without and keep products on the market. communication and collaboration. compromising safety and efficacy. The theme is jointly led by Maren Christer Backman, Member von Fritschen, Managing Partner, CMDh, EU Coordinator and Growing Demand for Director Regulatory Affairs, Senior Expert, Medical Products Quantitative Approaches in PharmaLex, Germany; and Marta Agency, Sweden; and Ning Xu, Drug Development is co-chaired by Jürgen Kübler, Global Head, Clinical Sciences, CSL Behring, Pre-Conference Tutorial Opportunities at the 2013 EuroMeeting Germany; and Steven Teerenstra, Biostatistics Working Party Subject Instructor(s) (BSWP), Statistical Evaluator, Analysis of safety data from clini- Jürgen Kübler, Global Head, Medicines Evaluation Board, cal trials Clinical Sciences, CSL Behring Netherlands. The last decade AG, Germany Joachim Vollmar, has witnessed a rapid growth International Clinical Development in both the amount and the Consultants, LLC, USA accessibility of data – matched Update on practical work with Susanne Winterscheid, Head of by the expectation to optimally variations under the revised regu- Project Management of Licensing exploit it. This theme will touch lation Division 3, BfArM, Germany on areas where sound statistical Maren von Fritschen, Managing approaches play an integral role Partner, Director Regulatory Af- in ensuring informed decision fairs, PharmaLex, Germany making. There will be a thorough discussion of new approaches Introduction to EU medical device Sabina Hoekstra-van den Bosch, as well as a critical appraisal regulation Senior Manager, Standards & of current recommendations Regulations, Philips Healthcare - Global Quality & Regulatory, and guidelines. The theme Netherlands aims to provide a forum for discussion between industry, Erik Vollebregt, Attorney, Axon Lawyers, Netherlands academic and regulatory thought 69 leaders. Relevant experience Recent and current developments John Lisman, Lawyer, Lisman Le- will be shared and discussed, in pharmaceutical law gal Life Sciences, Netherlands reflecting on lessons learned. Paul Van Dongen, Lawyer, Nau- Recommendations for best taDutilh, Netherlands practice will also be developed. Scope of the Paediatric Investi- Paolo Tomasi, Head of Paediatric Jan Willem Van der Laan, Senior gational Plan (PIP), condition vs Medicines, European Medicines indication and other simplification Agency, EU Pharmacological Toxicological initiatives in paediatric medicines Assessor, Medicines Evaluation Julian Britton, Associate Director, Board (MEB), Netherlands will Immunology Western Europe, Ab- bott Laboratories, UK steer Developments in Non- Clinical. New therapeutic Health Technology Assessment Katarzyna Kolasa, Head of Market areas as well as new technical (HTA) of drugs and medical de- Access Central Europe, Biogen methodologies are challenging vices Idec, Switzerland the classical way of thinking in the Cynthia Iglesias Urrutia, Senior non-clinical area. Furthermore, Research Fellow, University of the public and political pressure York, UK to reduce the use of animals is The European Qualified Person Brian Edwards, Principal Consul- a factor that cannot be ignored. (QP) for pharmacovigilance; every- tant, NDA Regulatory Science Ltd, These trends come together in thing you ever wanted to know but UK new areas such as the in-vitro were afraid to ask Vicki Edwards, QPPV/Senior assessment of toxicology, but also Director, Affiliate Vigilance Excel- in new products areas. Even an lence Global Pharmacovigilance, old issue, such as carcinogenicity Abbott Laboratories, UK testing, can learn from this. Non-clinical safety assessment in Klaus Olejniczak, Non-Clinical There are five IMI Education and global pharmaceutical develop- Regulatory Consultant, Germany Training Projects. Three of them ment Gerd Bode, Consultant, Germany are dedicated to developing, Table cotinued on page 70 gf GLOBAL FORUM | REACH
testing and establishing research Subject Instructor(s) master programmes on medicines Interactions between regulatory Geneviève Michaux, Esq of Coun- safety. Hans Lindén, Leader and intellectual property, privacy sel, Covington and Burling LLP, European Projects , European and product liability Belgium Federation for Pharmaceutical Development Safety Update Re- Margreet Ockhorst-Besijn, Senior Sciences (EUFEPS), Sweden, is port (DSUR) kick-off in 2011; and Safety Executive, Vigilex B. V., the Chair for IMI Public-Private now in practice Netherlands Partnership in Medicines David Lewis, Global Head of Phar- Research Education and CHINA macovigilance Novartis Pharma, Training. This theme will provide Switzerland a bird’s eye view of key IMI projects in these pre-competitive Technology transfer for biophar- Richard Dennett, Director, Voisin maceuticals Consulting Life Sciences, France areas: Predicting safety, predicting efficacy, knowledge Pharmacovigilance audits and Leonardo Ebeling, Managing management, and education and inspections Director, Dr. Ebeling & Associates, training. These projects include Germany SafeSciMET, integrated medicines Patricia Moore, Operations development (PharmaTrain), Manager GCP/PV, Medicines and and on pharmacovigilance Healthcare products Regulatory and pharmacoepidemiology in Agency (MHRA), UK medicines usage (Eu2P). There is Coding with confidence Tomás Moraleda García, Inter- also (EMTRAIN); how to establish 70 national Medical Officer, MSSO a European education and training (Maintenance and Support Ser- network; and the European vices Organisation), Spain Patients’ Academy on Therapeutic Understanding translational medi- Aamir Shahzad, Director of Innovation (EUPATI). cine Administration and Secretary VOL 5 ISSUE 1 General, International Society for There is also a compelling Translational Medicine (ISTM), Antibiotics/Anti-infective Austria Treatments theme. This is headed up by Richard Bergström, Director Creating compliant Clinical Study Nancy Smerkanich, Vice President General, European Federation of Reports (CSRS) for the EU and US Regulatory Affairs Consulting, Oc- Pharmaceutical Industries and tagon Research Solutions, USA Associations (EFPIA), Belgium; Teresa Eastwood, DRA Operations together with Marco Cavaleri, Group Leader, Actelion Pharma- Head of Anti-infectives and ceuticals, Switzerland Vaccines, Safety & Efficacy Sector, GCP inspection and audit findings Beat Widler, Senior Partner, Widler European Medicines Agency, EU. & Schiemann, Switzerland This theme is currently under Fergus Sweeney, Head of Sector, development. Inspections, European Medicines Agency, EU For more information please go the EuroMeeting website at www.diahome. org/em2013. Contact DIA Europe Customer Services on +41 61 225 5151, or email us at euromeeting@ diaeurope.org. CHINA
Participants at the Pharmacovigilance Training held October 22-24 in Beijing.
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DIA China Successfully Held ICH Pharmacovigilance Training Aiming at providing better insight This training course, which was over pharmacovigilance and risk the first cooperation between management to professionals ICH and DIA China, was specially in China’s pharmaceutical designed for Chinese profession- sector, DIA China held the ICH als at which Dr. Jan Petracek, Pharmacovigilance Training an invited European experienced on October 22-24, 2012 in pharmacovigilance expert made Beijing with the assistance from an impressive presentation on ICH Information Center of the State E2 series of guidelines, imple- Food and Drug Administration. mentation details and practical This training was a success case studies in ICH countries. The as it not only furnished the Chinese experts from the Training professionals with latest Program Committee (see page pharmacovigilance knowledge, 73) also shared their accumulative principles and implementation rich experience in pharmacovigi- of relevant processes, but lance with participants on how to also promoted international apply the international rules and communication and cooperation. standards for the best practices in their work. There was atten- dance of newly 100 people to gf GLOBAL FORUM | REACH
the training, mainly coming from and became the 68th member. and is responsible for supervision health regulators of China, Hong Since the 1990s, China has been of national post-marketing drug Kong, Taiwan and Singapore experiencing the stages of first safety monitoring and technical and multinational pharmaceutical setting up adverse drug reaction evaluation. The construction of the companies, with their relevance monitoring system; then, the pilot system for drug adverse reaction in the field of phar- monitoring has really macovigilance, risk shown that China management and development of drug clinical work. All the supervision entered participants were into a new era after issued with ICH the State Food and Pharmacovigilance Drug Administration Training Certificates was establish in at the end of the 1998. Training. During the training, According to the participants gained definition by the comprehensive World Health knowledge of ICH Organization (WHO), guidelines, including pharmacovigilance international is a kind of scientific pharmacovigilance 72 research activity standards, rules which relates Jan Petracek, MD, MSc, Partner and CEO at PharmInvent, contract safety and regulatory science and process of their company, addresses the participants at the training. Dr. Petracek was a Head of Risk Management to exploration, Section of European Medicines Agency until 2010, where he was responsible for all operations of implementation as evaluation, the Agency in relation to risk management of medicinal products. well as pre- and understanding post- marketing
VOL 5 ISSUE 1 and prevention safety management of adverse drug reactions or and later the development which and safety update report. They any other possible drug-related have formulated a series of laws also learned about the origin problems. In 1974, France and regulations for drug safety of ICH establishment, the first proposed the concept of supervision, such as “Adverse purpose, forms of organization, “pharmacovigilance” (PV) which Drug Reaction Monitoring terms of reference and has given to a new meaning Management (tentative)” in workflow for implementation of and been generally understood 1999, “Drug Administration pharmacovigilance which can as surveillance, guard, ready to Law of the People’s Republic of be as a reference for making cope with possible harm from China” in 2001, “Adverse Drug further laws and regulations for drugs. Currently, under the Reaction Reporting & Monitoring China pharmacovigilance and coordination of the international Management Approach” in construction of adverse drug drug monitoring program of the 2004 and “Drug Recall” in 2007 reaction monitoring system. This World Health Organization, more as well as the newly revised training was a good opportunity than 80 member countries around “The Provision for Adverse for professionals to expand their the world have enrolled into the Drug Reaction Reporting and horizons, enrich their knowledge formation system. Monitoring” in 2011. The “National and learn from each other’s Adverse Drug Reaction Monitoring experiences. China officially joined the WHO Center“ was established in International Drug Monitoring 1999 on the basis of merger Cooperation Center in March 1998 of the Original Drugs Adverse Reactions Monitoring Center of Ministry of Health and Drug Evaluation Center of the State Food and Drug Administration, Pictured, from left: Ms Donglei Mao, executive Editor and Journalist of SFDA Southern Medicine Economic Research Institute, who interviewed Dr. Ho; Dr. David Ho; and Dr. Jessica Liu, who assisted with interpretation during this interview.
Program Committee for ICH Cocktail Therapy: 73 Pharmacovigilance Training Laurence Huang Victory in Just One Battle Executive Director, Regulatory This article was prepared by In preparation for this interview, Affairs, staff reporter Donglei Mao of the I reviewed two decades of AstraZeneca, China Medicine Economic News and coverage in Chinese and foreign is based on an interview with Dr. media, including RTHK’s TV series Xiaojun Guo, PhD David Ho, who is a Professor “Success Stories,” Time magazine Head of Clinical Safety and at The Rockefeller University 1996 Man of the Year cover story, Pharmacovigilance, and CEO of the Aaron Diamond CCTV celebrity interview, as well R&D China GlaxoSmithKline, AIDS Research Center, NY, NY, as Phoenix Satellite TV Century China and is on a journey to discover Forum, among others. Xiaoyao Conny MO a vaccine against AIDS following Dr. Ho was portrayed by the Director, Head of his renowned breakthrough of media as a real hero for reversing Pharmacovigilance and combination therapy. the hopeless situation in the fight Product Information (PPI) Dr. Ho was one of the honored against HIV/AIDS through his China R&D and Scientific guest speakers at DIA’s 4th China discovery of the cocktail therapy Affairs, Janssen-China, Annual Conference in Shanghai treatment. Dr. Ho, who is nearly Johnson & Johnson and this interview was conducted 60 years old, is relentless in his Xue Tang during this meeting. pursuit of an AIDS vaccine and Country Safety Lead for China, still works more than 10 hours a Global day. Pharmacovigiliance Office, Cocktail therapy was successfully WSRO, Pfizer, China developed and tested 16 years Tracy Zeng ago. Dr. Ho is one of the first Senior Manager of Patient scientists in the world who Safety, AstraZeneca, China recognized that AIDS is caused by a virus, and the AIDS diversity gf GLOBAL FORUM | REACH
is caused by the replication of AIDS Vaccine Research to the laboratories, but he has the AIDS virus. Based on this Shows Promise also expanded his AIDS research, knowledge and understanding, Dr. prevention and treatment efforts Ho and his colleagues worked on Several years after his famous all over the world, especially in developing and testing effective breakthrough, Dr. Ho and his China. combination antiretroviral therapy, Aaron Diamond AIDS Research which became known as cocktail Center faced a series of setbacks, Dr. Ho said that many other therapy. Dr. Ho has been at the searching for a new research scientists have brought cocktail forefront of AIDS research for the direction. These challenges made therapy to regions in the world past 26 years. The AIDS mortality some people concerned about with serious AIDS epidemics, rate has decreased by 60% in the future of the Aaron Diamond such as Africa and Thailand. “My developed countries since the AIDS Research Center. Some background naturally leads me introduction of cocktail therapy, questioned whether the Center to China,” he said. David Ho was and the international community and its famous leader would born in November 1952, Taichung has taken measures to provide ever see another breakthrough, City, Taiwan Province, and this therapy for millions of AIDS or remain stagnant in the fight immigrated to the United States patients in developing countries. against HIV/AIDS. with his family when he was 12 years old. In his view, to leave Dr. Ho was selected as “Person An effective vaccine against HIV is and then return to China, is a very of the Year” in 1996 by Time the only hope to completely defeat meaningful cycle. magazine, and received countless AIDS. Vaccine development has honors for his achievements. been the focus of Dr. Ho’s work When talking about vaccines, Dr. 74 These honors placed Dr. Ho at during this period. He believes, Ho is enthusiastic. I was excited the center of attention in the fight “The development of an AIDS to hear that Dr. Ho would discuss against AIDS. A lot of people think vaccine gave us the greatest his team’s latest progress in AIDS that AIDS was defeated by the challenge we have ever met. vaccine research at the recent DIA invention of cocktail therapy, “but We have to wait for a scientific China conference.
VOL 5 ISSUE 1 in fact, we just won one battle,” breakthrough, which may happen “In 20 years, you will be 80 years Dr. Ho said. tomorrow, or many years in the future.” old. Will you still be working in this Analyzing his past achievements area?” I asked. “I should be, I am and all the attention they Dr. Ho’s research team has optimistic by nature, otherwise I attracted, Dr. Ho said: “Some developed two deoxyribonucleic could not work in the field of HIV reports were true, some were acid (DNA) vaccines for the research,” Dr. Ho said. from reasonable guesses. This is prevention of HIV recently. Phase Below is the interview the media, they exaggerate, try to I clinical trials in humans of the between Dr. Ho and play things down, or somewhere two vaccines were started in the staff reporter, Donglei in between, isn’t it?” he asked me United States. However, it will Mao, of the Medicine in his reply. take at least five years before the vaccines can be introduced Economic News “Although my work had been into the market. Dr. Ho said that well-known in the scientific field, his research center has been What is the status of the praises from Time, The Wall Street cooperating with Chinese research research and development in Journal and other mainstream institutions, such as Chinese your AIDS vaccine project? media pushed me into the public Academy of Medical Sciences and We have a new HIV prevention eye, which allowed the general human trials of new HIV vaccines project, which is a passive public to learn more about AIDS may be carried out in China in the immunization method by prevention and treatment. I am future. using antibodies to block HIV grateful for this recognition,” says infection. A lot of research will be Dr. Ho has traveled between China Dr. Ho. developed, including one study to and the United States frequently promote a proof-of-concept test, in the last 20 years. As a scientist, aiming to show that this antibody Dr. Ho’s work is not only limited will have better pharmacokinetic and Technology, AIDS Fund, to see that the leading scientists in effect in its anti-HIV activity. and the Ministry of Health. Both AIDS research organizations such the Ministry of Science and as the Chinese Center for Disease We have conducted Phase I and Technology and the Ministry of Control and Prevention, Tsinghua will conduct Phase IIa clinical trials Health have agreed to give us University, the University of Hong in the U.S. We plan to conduct a support. In addition, we need to Kong, and other institutes, are my pivotal study, a Phase IIb clinical contact with the SFDA, which is former students. trial, in China in the next two the last step in the process. years. There are many reasons There are a lot of very talented to carry out these studies in Do you have a shortage in scientists in China. If you visit China. For example, China has a research funds? the laboratories in the Western large high-risk population; many countries, you will find a lot of scientists and research institutions Yes, very often. Sometimes I feel Chinese people working there. are interested to participate in like I am running on a treadmill Many Chinese professionals take the studies; Ministry of Science nonstop. The current economic new techniques and thinking back and Technology and Ministry of situation in the United States to China, so the gap between Health are eager to participate in has made the government cut Western scientists and Chinese innovative research now. Because a large amount of research scientists is narrowing constantly. it is a very new path, the process funds, whereas the situation is not easy, and will probably take is better in China. As a foreign The Chinese government’s at least two years. scientist, however, I can’t make investment is growing, which a direct application for funding really attracts scientists to come Which procedures should from the Ministry of Science back to China to work, or for 75 be modified after Phase IIb and Technology or the Ministry the establishment of cooperative clinical trials enroll Chinese of Health in China. However, research. Chinese government subjects? the establishment of a research leadership is also very far-sighted, institute in the School of Medicine, and they hope that China will I hope that China will be included Tsinghua University could be become a powerful country in in the study of the next Phase IIb a base for funding application. research and development, so clinical trial. However, it will be As this study is a multinational that more investments will be very difficult to carry out if there trial, we will make full use of the made. Certainly, it will take time to are many operation standards for international funding system change the deep-rooted culture in the study. to carry out this cooperative China. research. I do not think that Western As a team leader, how do you standards are golden standards. Nearly one-third of the see the failures and successes However, we should coordinate researchers at the Aaron of scientific research? with each other and establish Diamond AIDS Research a system, which is suitable not Center are from Asia, If you never fail, you will never only for the Western scientists especially from China,. be successful; if you do not and Western pharmaceutical However, more and more want to fail, you can only be companies. If the Chinese Chinese scholars have mediocre.. Failing requires making scientists want to participate in returned to China to do a correction and changing course. competition with the rest of the research. How do you evaluate This is prominent in the United world, we should recognize what the overall domestic academic States value system, which is is the path as a whole. environment? different from the oriental culture. The scientific progress is a self- It is a time-consuming marathon The scientists who have come correcting process to a great to carry out Phase IIb trial in out of our research center have extent. China, and it requires long- brought the latest developments term effort. We will discuss with in HIV/AIDS prevention and many relevant officials in China, treatment to the rest of the world, including the Ministry of Science including China. I am very pleased gf GLOBAL FORUM | REACH
JAPAN
The 2nd DIA China Ethics Training was successfully held at the Clinical Research Institute of Peking University Health Science Center with over 100 participants. DIA Worldwide Executive Director Mr. Paul Pomerantz, Deputy Executive Director Carlos Fulcher, the DIA China Office staff, Dr. Jinju Li, Director, Division of Drug Research Supervision, Department of Drug Registration from SFDA, Instructors and Program Committee Members, Dr. Beat Widler, Dr. Haiyan Li and Dr. Haitao Liu joined the participants for the photo. 76 HSA & MEB Sign MOU at Asia VOL 5 ISSUE 1 Regulatory Conference 2013
From January 28-30, the Health companies, and clinical research to risk management strategies, improved Sciences Authority (HSA) in Singapore meet and exchange views, discuss benefit/risk assessment of medicinal hosted Asia Regulatory Conference topics of interest and identify focus products, joint education and training 2013, the first time the HSA has areas for ongoing efforts to increase of regulators and postgraduate hosted this event. Developed on the patient access to new and improved students, and other initiatives. This theme “Regulatory Convergence medicines,” said Dr. Ling Su, DIA MOU was signed by Associate and Cooperation to Improve Access Board President, in his welcoming Professor John Lim, Chief Executive and Quality,” this conference remarks. “We are honored to have Dr. Officer of HSA and by Professor brought together experts from Amy Khor, Minister of State for Health Hubert Leufkens, Chairman of MEB, health authorities, pharmaceutical and Manpower in Singapore, speaking and witnessed by Dr. Khor. companies and academia across at the event as well as speakers almost 30 countries in the Americas, from top-level regulatory authorities In 2011, DIA co-presented the first Asia and Europe to identify specific in several Asian countries and Asia Regulatory Conference with the areas that will enhance patient access leading experts in the International IFPMA and Asia-Pacific Economic to new and improved medicines. It Conference of Harmonization Cooperation (APEC) Harmonization was co-organized by the HSA, DIA process.” Center (AHC) in Seoul, Korea. and the International Federation of Pharmaceutical Manufacturers During this conference, the Association (IFPMA). HSA signed a Memorandum of Look for more detailed coverage Understanding (MOU) with the from the Asia Regulatory “This three-day conference offers Medicines Evaluation Board (MEB) in your April a unique opportunity for key of the Netherlands to further Conference 2013 stakeholders from health authorities, collaboration between the two Global Forum. local and multinational pharmaceutical agencies on pharmacovigilance and JAPAN
Junichi Nishino
Program Vice- 77 Chair, the 9th DIA Japan An- nual Meeting
Chairman, Contents Com- 9th Japan Annual Meeting mittee
Novartis Reflections Pharma K.K. The 9th DIA Japan Annual attended as a speaker and Meeting was held at the Toshi panelist. We were also extremely Center Hotel (Tokyo, Japan) pleased and grateful to have over over three days from November 75 participants from PMDA in 19-21, 2012. The focus of this attendance. meeting was “Importance of Development Strategies for In this article, I would like to reflect Lifecycle Management of Safe upon the new improvements and Effective Medical Products– implemented at the 9th Japan What can we do from the start of Annual Meeting. development to benefit patients?” The State of New Drug Including speakers and exhibitors, Development in Japan the annual meeting was attended and the Japan Annual by around 800 participants Meeting from home and abroad, which was almost 300 more than last The strategies for new drug year. Among others, Dr. Peter development in Japan are Bachman (BfArM) attended as diversified, with three main a keynote speaker and panelist, approaches: domestic full and Dr. Gerald J. Dal Pan (FDA) gf GLOBAL FORUM | REACH
development, bridging strategy, regulations exclusively, but it is This Contents Committee and simultaneous global essential for us to comprehend was launched in 2012. Please development including global the regulations and trends in East see the June 2012 Global clinical trials. Furthermore, Asia and the West as well. Forum for an introduction to simultaneous global development the Contents Committee’s also includes Asian development The meeting theme and content organizational structure, roles strategy. Such strategies should was decided taking into and responsibilities. Composed be adopted in accordance consideration the current situation of members who work on the with what is optimal for the in Japan. forefront of new drug development therapeutic area and background and new drug review, the New Endeavors at the of each project. A current trend Contents Committee members Japan Annual Meeting is to develop in collaboration with have firsthand knowledge of PMDA from the early development This was the 9th Japan Annual areas that are going well and of phase, and move forward on Meeting, but a survey of problematic/improvement points. development together. On the participants at the 8th Japan Furthermore, they possess other hand, in terms of the Annual Meeting (2011) revealed solutions and prospects for review stage, in the country’s several comments that sessions the future. These members do effort to eliminate drug lag, a were primarily focused on not only have ideas on session development program and review strategies and policies and there content but also ideas for period are steadily shortened. were few operational matters candidates for chairpersons and These developments are a at the practical level. While speakers in each professional great leap forward in terms of area. Therefore, the Contents 78 some maintain that explicit delivering new drugs to Japanese discussions of practicalities are Committee concurrently worked patients more quickly. However, not of importance at a meeting of as Program Committee members it seems that something of great representatives from industries, for the 9th Japan Annual Meeting. importance has been forgotten government and academia, there A majority of the Contents as a result of being overly is also the opinion that precisely Committee members are relatively VOL 5 ISSUE 1 focused on development speed because it is a meeting of such young, so while Program and shortening review. The representatives, current issues Committee members had a great concept of “patient benefits” in each field should be shared responsibility, they were highly is once again being upheld by and as lessons are brought forth, motivated to manage such a big people in various positions which there should be discussions on event from the planning stage to transcend industry, government, problem resolution. As DIA is carrying out operations on the and academia, and it is important unaffiliated, it is a place of equality day of the meeting. The Contents to exchange opinions and to where opinions can be expressed Committee can claim a major role disseminate information regarding and debates can be held, so it in the 9th Japan Annual Meeting. the identification of issues and was decided that opinions from solutions, as well as future experts in each field would be 2. Changes to Meeting Scale expectations. It is also important included in the agenda for the 9th and Location to inquire directly with medical Japan Annual Meeting. Opinion at DIA Japan was unified staff and especially patients about expanding the Japan about their opinions on new 1. Involved Contents Annual Meeting to include experts drug development and to have Committee members and from specialties that were not the opportunity to fully process their Contribution formerly involved. Therefore, that information. Furthermore, although sessions in the past as mentioned above, this is At DIA Japan there is a committee were mainly focused on regulatory the time for simultaneous representing perspectives from affairs, clinical development, and global development, and as expert or operational staff that statisticians, at the 9th Japan development and review take proposes and coordinates Annual Meeting we decided to place simultaneously throughout innovative ideas about content hold sessions on post-market the world it is no longer the time of annual meeting, workshops safety, project management, for addressing pharmaceutical and training courses in Japan. CMC, patient groups, HTA (Health industries. We feel that having through the student sessions. Technology Assessment), and DIA incorporate not only science other important topics relevant but also work-life balance into the We intend to continue with to Japan. As session numbers Japan Annual Meeting helps to concepts of 9th Annual Meeting increased, the previous two- increase the value of DIA. at the 10th Annual Meeting as day, three-track program did not well. There are many improvement provide enough time slots. Thus, a Reflections on the points in 9th Japan Annual three-day, six-track program was Japan Annual Meeting Meeting, we think. We look decided upon. Furthermore, over forward to making the next Japan We would like to take this the past few years the meeting Annual Meeting even better opportunity to express our was held at a location which was based on opinions received gratitude towards the many who somewhat distant from the center from participants. Furthermore, participated and all of the parties of Tokyo, but for this meeting an we hope that the DIA Japan who cooperated with us. As the improvement was made which Annual Meeting becomes the planners of the meeting, we were took convenience for participants key conference in Japan on new extremely pleased with the great into consideration and the drug development and that it will turn-out at the newly incorporated conference was held at a hotel in continue to be notable as a place student sessions, tutorials, and the heart of Tokyo with convenient where industry, government, and sessions on project management, access to public transportation. academia unite to have heated CMC, and patient groups. What discussions for medicine and 3. Introduction of Tutorials, kind of development strategy patients in Japan. Student Sessions, and the should be the best scenario to PMDA Town Hall deliver safe drugs to patients 79 quickly and effectively, and what Although tutorials, student to pay attention to in order to sessions and the PMDA Town encourage manufacturing and Hall have been established in supply of drugs and safe use even programs at the DIA EuroMeeting in the post-market phase were UPCOMING and the DIA Annual Meeting, realized through each session. In EVEJAPANNTS they were not previously held general we feel that the goal of 16th Annual Workshop in Japan. However, in addition this conference was achieved. in Japan for Clinical Data to increasing the scale of the Management meeting, it was decided that There were many first-time Feb. 7-8 these programs would be participants at the DIA Japan 3rd Labeling Workshop in introduced at this meeting. While Annual Meeting. This is attributable to the fact that our Japan cooperation was necessary from March 16 PMDA officials for the PMDA new programs, which targeted Town Hall, academic professors people in the field, differed from 7th Annual Conference in for the student sessions, and the past and the fact that the Japan for Asian New Drug lecturers and panelists offered a Development lecturers for the tutorials, everyone April 15-16 graciously undertook the task balance of industry, government, for us, furthering the impression and academia. It was clear that 2nd CMC Forum in Japan that DIA is an organization expectations are ripe for the June 10 which helps realize cooperation Japan Annual Meeting to offer 2nd FDA IND/NDA Training in between industry, government programs in the future which serve the interests of many and take into Japan and academia. Also, in addition June 12 -13 to these sessions, we held a consideration a balance between workshop entitled “Diversity policy and practice. Furthermore, 4th Cardiac Safety Workshop Forum” on the second night to in terms of the future, it is in Japan July 11-12 exchange opinions on career important and meaningful for paths and women working students to develop an interest in Japan in pharmaceutical in and have a hand in DIA Japan gf GLOBAL FORUM | REACH
INDIA
Arun D. Bhatt
80 MD (Med), FICA (India), FICR (UK)
President, VOL 5 ISSUE 1 Clininvent Research Pvt Gene Based Therapies: Ltd Ethical Challenges in India Over last 2 decades, gene based of gene therapy raised several therapy has evolved from bench challenging ethical issues. In India, to bedside. As of June 2012, the field of gene therapy is still in over 1800 trials were registered infancy. But Indian researchers in Journal of Gene Medicine are developing gene therapy for Database.1 In 1982, the US conditions such as thalassemia President’s Commission for the and hemophilia, which affect Study of Ethical Problems in Indians in large numbers.3 Medicine and Biomedical and Therefore an understanding of Behavioral Research concluded ethical issues in clinical trials of that the therapeutic applications gene therapy is vital for Indian of gene based therapy were regulators, ethics committees, analogous to other forms of investigators, and sponsors. therapy. Hence, general ethical standards and procedures were Gene therapy considered adequate to assess Gene therapy, more precisely the usefulness of gene based called gene transfer, is a form therapy.2 However, death of Jesse of experimental treatment that Gelsinger in an early phase trial involves transferring genetic material into the cells of a patient with a disease caused treat infants suffering from Social value of intervention by a missing or mutated gene.4 severe OTC deficiency, selection The goal is to cure the disease of patients such as Gelsinger • The main justification for gene by modifying the genetic with mild OTC might have transfer therapy is that it offers a information of the patient‘s cells, placed them at unnecessary true cure or a useful remedy for thereby inducing normal protein risk. patients who are suffering from expression to replace the mutated a serious illness, and for whom or lost gene. • Informed consent – Researchers there are very few therapeutic failed to inform the subjects options available.4 Gene transfer research includes about the risks involved in somatic (non-reproductive) cell the study e.g. rhesus monkey Scientific validity gene therapy and the germ deaths in preclinical studies, the • Most of the gene transfer (reproductive) line therapy.4,5 primitive nature of gene therapy studies in early stage are The somatic gene transfer only research. conducted to assess feasibility affects the treated individual. In and safety of the therapy. contrast, in germ cell transfer the • Conflict of interest – The Hence, most such studies are modified gene is incorporated in clinical trial was conducted uncontrolled in nature. Also the the genome of the individual and at University of Pennsylvania available preclinical information can be transmitted to subsequent School of Medicine, which had is still primitive. So one of the generations. As of now, germ line financial links with Genovo, most difficult questions is: transfer therapy is considered the company holding licensing when to begin human studies. ethically unacceptable due to its rights to the study intervention, Information from early clinical unknown risks. Hence, research and the lead investigator James 81 studies often leads to new on this therapy is prohibited. Wilson. The US Food and Drug Administration inspections animal studies, which in turn The complexities in the biology of revealed many protocol suggests the need for additional 2 gene transfer make the therapy violations, fudging of eligibility human studies. a scientifically and ethically criteria, and failure to report the • As gene transfer has challenging procedure.4 up-to-date safety information, potential for exposing the trial and failure to report the findings participants to the possibility Gelsinger Case : A from preclinical studies that of serious, unforeseeable, and Milestone in Ethical suggested a significant risk for latent harms, the objective of Evolution human subjects, and failure trials should be to generate to update informed consent The death of Jesse Gelsinger, information that can be applied document with reports of an 18-year-old patient, in a to the development of gene significant adverse reactions Phase I study involving ornithine transfer rather than to support – grade III liver enzyme transcarbamylase (OTC) commercialization.8 elevations.2,6 deficiency, has become a major • As some of the serious adverse milestone in the debate on The Gelsinger case has become a events (AE) may be latent or ethical issues surrounding gene watershed event in the evolution 2,4 delayed, a long term and robust therapy. This event raised of ethical policies for gene transfer surveillance of safety issues is several ethical questions, which research. include: a must during clinical trials. The Global Ethical uncertainty of risks mandates • Selection of subjects – Concerns a high scientific standard for all Gelsinger had mild OTC gene therapy trials.8 deficiency, controlled with The ethical challenges of gene medication and diet. Although transfer therapy could be Fair selection of study the adenoviral-based discussed in relation to general population intervention was designed to ethical principles and benchmarks, • One major dilemma has been applicable to all research.7: whether to select adults with mild disease, who can give gf GLOBAL FORUM | REACH
consent or patients with serious competent for review of human Respect for recruited life threatening conditions, protocols. Indian Council Of participants who may benefit from the Medical Research (ICMR) gene transfer, but who are Ethical guidelines recommend • The issues of privacy and vulnerable.8 that the ethics committee confidentiality are paramount (EC) reviewing research in genetic research. The high • As the risks of gene therapy proposals related to research risk and potential for late are latent and uncertain, the on human genetics should have development of serious AE selection of healthy volunteers necessary expertise, which brings into focus the problems is not usually favored. 8 For includes knowledge of latest of compensation for medical similar reasons, the hazards developments in the field of injury and death. of conducting gene transfer human genetics.5 trials are enhanced in children • A major issue concerns 2 who have non-life threatening • The inherent uncertainties of cost and access of therapy. diseases.8 risks require central ethical Gene therapy involves high review of all trial protocols.8 development cost. Most of the Favorable Risk-benefit Ratio In most countries use of two gene therapy targets are orphan ethics committees (EC) - a diseases, with an expensive • Death of two subjects in early central EC and an institutional treatment cost. As gene therapy phase studies - Gelsinger and EC is mandatory. The would be usually a onetime a child with X linked severe responsibility of central EC intervention, the cost of therapy combined immunodeficiency is to review genetic research has to be borne upfront. These (X-SCID) succumbing to 82 from a national perspective, factors are likely to limit the leukemia, highlight the enhance the capacity of local potential of global access to challenges and uncertainties of EC, and organize conferences gene therapy. assessing the risk. In X-SCID to discuss/debate on safety study, three more children had Indian situation or study design to evolve developed leukemia. Such VOL 5 ISSUE 1 consensus.2,5 serious AEs have been difficult In India, there is a growing interest in genomic research. Hence, to anticipate, because the Informed consent animal models and toxicology Department of Biotechnology assays used in preclinical safety • The uncertainty of risk profile, (DBT) has issued ethical policies 9 assessment were primitive. The potential for future serious AES for genetic research and services. levels of uncertainty are likely e.g. malignancy, availability The policies emphasize basic to be greater in the context of limited preclinical data and principles – autonomy, privacy, of gene transfer, compared to high expectations of potential justice and integrity and provide other novel medicinal entities.2 benefit amongst desperately ill general guidance to researchers, subjects, makes the consent ECs, institutions and organizations Independent review process demanding. Such for conduct and design of severely ill subjects are liable to research and services. Similarly • The ECs have to face formidable therapeutic misconception and ICMR ethical guidelines cover challenges in the evaluation could misunderstand the clinical issues related to genetic research of the risk: benefit ratio of the 5 trial as a “therapy” rather than a and gene therapy. However, both gene therapy and oversight of scientific experiment.6 DBT and ICMR guidelines do not disclosure of risk in consent discuss issues of gene therapy in 8 documents and process. • The investigators should detail. Hence, one has to apply spend enough time and effort global principles to local research • The scientific complexity and in discussing the possibility of on human gene therapy. ethical concerns require that the unforeseen consequences and independent review should be should provide full disclosure of Some genetic disorders – done by an ethics committee serious AE which have occurred thalassemia, hemophilia, sickle which has members who are in previous human gene transfer cell diseases – prevalent in Indian trials.8 population have serious medical be experimental, with uncertain to be a special focus on long consequences. For thalassemia risk profile, the investigator term surveillance of adverse the treatment is lifelong and would like to include a few events beyond the trial period. expensive. For such a condition, patients in the trial. However, gene therapy may provide a good the potential for cure in a • The cost is a major issue medical option. It would be worth condition, which has serious for thalassemia treatment. discussing ethical challenges of health impact, would make the The treatment costs for gene therapy, using thalassemia family / community demand that thalassemic child of 4 years is as a model. Beta thalassemia all patients be included in the around Rs. 90,000-100,000 is one of the most common trial. annually in a private set- single-gene defect condition, up. Hence, not more than which poses a severe health and • The independent review by EC 5-10% of thalassemic children economic burden to patients suffers because of deficiencies receive optimal treatment. and families.10,11 Every year in EC functioning. A recent Stem cell transplantation, approximately 10,000 children survey of approval letters of which is a curative treatment, with Thalassemia Major are born Indian ECs revealed deficiencies costs between Rs 600,000 – in India. India has 65,000-67,000 in composition, quorum, 1,600,000.11 Gene therapy Beta thalassemia patients, with and review of insurance, and has a high cost in development. around 9,000-10,000 cases being clinical trial agreement.12 Hence, the access to this added every year. The treatment, Hence, as recommended by promising therapy would be which is lifelong, includes regular ICMR, the EC reviewing gene restricted to the most affluent cell transfusions, and chelation therapy proposals should have Indian patients. therapy for iron overload. The necessary expertise, which 83 Conclusions iron overload, associated chronic includes knowledge of latest developments in the field of blood transfusions, is the most The potential benefits of 10 human genetics.5 Also, we important cause of death. Gene gene therapy bring hope to a need to have a National EC to therapy offers the promise of patient suffering from a serious provide a robust review and cure by transferring the normal genetic condition. However, the guide institutional ECs. B-globin or y-globin gene into uncertainty of risk profile, the hematopoietic stem cells to • The population of patients vulnerability of subjects, and the permanently produce normal red – mostly children – and the commercial potential of gene blood cells.10 However, gene cost/complications of therapy, therapy pose significant ethical therapy for thalassemia in India, is vulnerable and requires challenges, which the investigator is fraught with several ethical adequate safe guards to and ethics committees have to dilemmas, such as: protect their rights, safety and deal with proactively. • For a genetic condition such as wellbeing. thalassemia, prevalent in certain Arun D. Bhatt • The consent process would communities e.g. Sindhis and need a thorough disclosure of MD, FICA, FICR, is President Punjabis from Northern India, of Clininvent Research Pvt all risks and transparency to and Bhanushalis, Kutchis, Ltd. He has extensive experi- protect the vulnerable subjects ence over 3 decades in the Lohanas from Gujarat, the from therapeutic misconception. pharmaceutical industry, in- issue of selection of subjects cluding serving as CEO of CMI (India) PVT Ltd and as Medi- becomes quite complex. The • Indian framework for cal Director of Novartis India selection of subjects for clinical surveillance of adverse Limited, and as a consultant trial would pose a challenge of in Pharmaceutical Medicine & reactions during trials and in Clinical Pharmacology. He is a enrolling an individual subject postmarketing setting is not former president of the Indian vs. enrolling several children well developed enough to pick Society for Clinical Research from one family or from a close (ISCR) and the recipient of up early signals for important DIA’s Outstanding Service nit community. As the gene adverse drug reactions. As the Award 2012. therapy in a trial setting would nature of risk of gene therapy is uncertain and latent, there has References/citations from this article are available upon request. gf GLOBAL FORUM | REACH
Upcoming DIA India Meetings 2013
e-CTD Hands on Technological Training Workshop Advancements to Meet February 4-5 | MuMbai Regulatory Challenges april 25 | MuMbai Pharmacovigilance MarCH 8-9 | bangalore Generics/APIs July | Hyderabad
6th Regulatory Conference 8th Annual Conference april 5-6 | aHMedabad oCtober 24-26 | MuMbai
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6th Regulatory 7th Annual 5th Annual Meeting Conference Conference in Japan DIA CHINA 2013 for Asian New Drug May 12-15, 2013 April 5-6, 2013 Development Beijing, China Ahmedabad, India April 15-16, 2013 Tokyo, Japan
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DIA HEADQUARTERS 800 Enterprise Road, Suite 200 Horsham, PA 19044, USA Tel. +1.215.442.6100 | Fax +1.215.442.6199 www.diahome.org EDUCATEEDUCATE ggff
EDUCATE does just that: Keeps
you abreast of the association,
membership, regulatory, and
legislative news while including
features such as career advice,
book reviews, patient perspectives
and more. PROFILE Beatriz Vicén Banzo
Beatriz Vicén Banzo, who will serve as Co-Chair of our 25th EuroMeeting: Amsterdam 2013, March 4-6 at RAI in Amsterdam, is a Pharmacist by training with specialization in Biochemistry by the University of Barcelona. She also holds a Master in Business Administration of the Pharmaceutical Industry from the University of Barcelona (1992), a Master in European Regulatory Procedures from the Autonomous University of Barcelona (1999), and a Master in Business Administration from the Business School ESADE (2005) Barcelona – Spain. 87
With a regulatory background of more than 20 years, she currently holds the position of Head of Public Affairs and Technical Department at Bayer in Spain. Prior to joining Bayer as Head of Regulatory Affairs and Quality Assurance in June 2008, she held the position of Head of Regulatory Affairs in Novartis Beatriz Pharma, Spain. Vicén Banzo
In your current position as with the best possible quality as Head of Regulatory Affairs and soon as possible is one of my Technical Department, what main priorities. The current crisis are your main responsibilities? environment in European countries like Spain makes the market My responsibilities include access policies of particular those related to the company importance, making my daily interactions with Central and work a continuous challenge. We Regional Spanish Health should also try and get experience Authorities with regard to in different ways of public and Regulatory Affairs, Quality private partnerships that allow Assurance, Pricing, Market the public health systems to Access and Government Affairs. overcome the current critical Making sure that innovative scenarios and also permit the Q medicines are available to patients industry to continue investments to get better medicines for the &A global population. gf GLOBAL FORUM | EDUCATE
What was your first career company in your country. Getting Agency – have been pioneers in step in this pharmaceutical experience through work and the implementation of these new development and regulation knowledge will undoubtedly make systems, namely the electronic arena and what advice would you a valuable professional in the submissions. This has also you give to people who want to field. influenced the training of people begin a career in this industry? who want to work today in a What is the single most Regulatory Affairs department, When I started in Regulatory amazing change you have where the knowledge as a Affairs, only one year after I witnessed while working in this pharmacist is best combined concluded my University degree industry? with a variety of other different in Pharmacy, and after some disciplines in this field. months of working in a pharmacy The most amazing change I (also a great experience, I must have experienced was when I You have found time in your say) the daily work in this field was able to participate in the professional life to contribute was mostly bureaucratic and first Mutual Recognition and in a major way to DIA. Why? not very appealing for young Centralized procedures at people, at least in an affiliate. In Novartis, and see that there was I have attended most DIA contrast, Marketing was at that a common agreement among the EuroMeetings since I started in time much more interesting for participating countries, fulfilling the regulatory arena. DIA forums most of the recently graduated the pre-established procedural always provide productive people. Regulatory Affairs timelines and achieving the discussions on a number of departments were seen as like a market access within a reasonable current regulatory and scientific 88 post office without possibilities timeframe in countries otherwise topics that most have on our to influence on the development considered ‘slow.’ In Spain, this office desks every day. I am of the products. Nowadays, it has meant at least one year of delighted with this opportunity involves a continuous update in earlier approval when compared to devote a part of my time to the understanding of scientific/ to the old national procedures. contribute to the forthcoming
VOL 5 ISSUE 1 technological innovations and Nowadays, however, it is not a DIA EuroMeeting. It has been, knowledge of regulations; from matter of just filing submissions and is still, a challenge and an this starting point, the regulatory and getting approvals; those excellent opportunity to network strategy has to be thoroughly involved in Regulatory Affairs with colleagues from different prepared in order to overcome require continuous training not pharmaceutical disciplines. the barriers in the currently only on the new regulations restrictive economic environment. but also on the associated If you had to do it over again, The regulatory input is nowadays technicalities, which translates would you take the same path? into a deep involvement in an of crucial importance to better Although I am totally satisfied ever-challenging arena. develop the product in the with my career, I often think that way that companies fulfill the On the other hand, the new studying law in addition to my requirements to avoid hurdles and Information Technologies have current studies would be really accelerate as much as possible also been notably important in interesting, as I feel very attracted the approvals that will eventually the development of this area and to law and it is very much linked to permit an earlier access to the have contributed to improvement my current profession. market. in the daily management of My advice to those who want any regulatory procedure. In to begin a career in Regulatory this context, some European Affairs, having all these important Agencies – and, I am proud to items in mind, would be to say, among them the Spanish keep studying and, if possible, try starting by applying for an internship in the Regulatory Affairs department of a pharmaceutical PATIENT PERSPECTIVE Patient Advocate Fellows Reflect on DIA 2012
Cynthia Ryan, Vestibular Disorders Association (VEDA) Cynthia Ryan, MBA, serves as Executive Director of the Vestibular Disorders Association. She also served as a Patient Advocate Fellow for our DIA 2012 Annual Meeting. While DIA continues to prepare for our DIA 2013 49th Annual Meeting: Advancing 89 Therapeutic Innovation & Regulatory Science, Cynthia shared these thoughts about her DIA 2012 experience and how that experience can benefit the patients this organization represents.
What are some of your of a vestibular disorder that constituents’ biggest fears accompany unsteadiness include about participating in a clinical dizziness, vertigo, hearing and Cynthia trial and did you see or hear vision problems, and difficulty with Ryan anything at DIA 2012 that could concentration or memory. help alleviate or overcome those fears? People with vestibular (inner ear balance) disorders suffer from a Most people don’t find it difficult chronic, invisible illness. They may to walk across a gravel driveway, not look sick, but their capacity transition from walking on a to function normally is severely sidewalk to grass, or get out of impaired, often to the extent bed in the middle of the night that they can no longer work, without stumbling. However, with drive, or participate in social impaired balance such activities activities. Vestibular disorders are can be extremely fatiguing and misdiagnosed and under-treated, sometimes dangerous. Symptoms resulting in extended periods of discomfort and frustration on the part of the vestibular patient, and Q often a resulting distrust of the &A medical community. gf GLOBAL FORUM | EDUCATE
Surprisingly, in a recent website role in their own medical care, Many vestibular patients find the poll, 72% of respondents said which in turn strengthens patient strain of travel difficult, and/or may that they would be willing to confidence in the medical system. be negatively impacted by the participate in research to help Establishing relationships with bright lights and busy atmosphere develop better treatments patients through transparent two- of the typical hospital or research for vestibular disorders. This way communication is essential setting. Seven percent of our represents the desperation that to building trust and increasing survey respondents indicated that many vestibular patients feel in patient participation in the clinical they would participate in a clinical the lack of current understanding trial process. trial if they could do so remotely. about these conditions, and During DIA 2012, I learned the limited treatment options Finally, nine percent of survey about a growing movement to available. The good news is that respondents indicated that they enable patients to enter data it signals an open door for the don’t know enough about the electronically, thereby removing a medical community to engage clinical trial process or how to potential barrier to participation in the so-called ‘dizzy patient’ in find out about an appropriate research. research designed to reduce clinical trial in their area. So how diagnosis times and improve do vestibular patients become May we ask you to please treatment outcomes for people informed about research and get explain and describe your with vestibular disorders. connected with clinical trials that work with VEDA, and how can are actively recruiting participants? organizations such as DIA Ten percent of survey respondents collaborate to help? said that they were concerned VEDA is a resource hub for 90 about unknown side effects the people suffering from vestibular VEDA’s mission is to serve people treatment they received during disorders. We provide information with vestibular disorders by a clinical trial could cause. on symptoms, treatments, and providing access to information, Considering the sensitivity of coping strategies, and serve offering a support network, vestibular patients to motion, as a conduit for researchers and elevating awareness of the
VOL 5 ISSUE 1 sound, and visual stimuli, in to recruit study participants challenges associated with these addition to dietary restrictions, through our website. We also disorders. We provide patients their concern is understandable. provide information about why with information and tools to However, to them I would quote research studies are important, educate themselves about what the first goal of any clinical trial, and explain the benefits/ it means to live with a vestibular which is to do no harm. Safety consequences a research disorder, how and where to get first was a consistent theme participant can reasonably help, and what they can do to give throughout the workshops I expect. By partnering with patient back to the vestibular community. attended during DIA 2012, advocacy organizations like VEDA, VEDA welcomes partnerships reinforced by the FDA’s diligent the medical community can reach with organizations like DIA that oversight policies. The stringent out to targeted patient groups promote patient-centered issues screening process for clinical while educating them about the and increase awareness about trial participants assures that research process. the value of patient participation only appropriate subjects will be Multiple DIA 2012 sessions in the diagnostic and treatment selected, and patient monitoring is discussed pharma’s increasing process. We consider it a service built into study protocol. use of social media to manage to our members – patients and Another theme repeated during and inform relationships and build professionals – to provide relevant DIA 2012 was that patients who patient engagement. There is a news and information on current live with a disease have a direct recognition that patients are going research therapies, clinical trials stake in the drug review process online to look for information recruiting patients, and other and are in a unique position to about their medical concerns, and topics that affect patient care, and provide input. Validating the that patients need support tools invite individuals and organizations patient perspective empowers to facilitate their participation in to submit such articles for patients to take a more active the research process, and enable publication in our newsletter and/ them to make informed decisions. or on our website. VEDA’s website receives hundreds Similarly, VEDA needs accurate Americans – have experienced of thousands of visitors annually. and scientifically validated data some form of vestibular Our Facebook page has an to continue and strengthen our dysfunction. As VEDA continues active following. Our audience advocacy efforts. Such data is a to reach out to people affected can be your audience. We invite valuable tool when approaching by inner ear balance disorders, researchers to post clinical trials medical schools to encourage we look forward to developing on our website, submit research better training for primary relationships within the medical findings for publication in our care physicians in vestibular community that help us achieve newsletter, and distribute other dysfunction, or lobbying insurance our goal to reduce diagnosis information to help vestibular companies for better coverage of times and improve treatment patients learn about how new vestibular conditions. effectiveness for vestibular treatments are developed and patients. decrease the stigma often As many as 35% of adults aged associated with being a research 40 years or older in the United ‘guinea pig.’ States – approximately 69 million
To learn more about the Vestibular Disorders Association, please visit www.vestibular.org.
91
Karen Ball, Sturge-Weber Foundation (SWF) Karen L. Ball serves as President and CEO of the Sturge-Weber Foundation. She also served as a Patient Advocate Fellow for our DIA 2012 Annual Meeting. While DIA continues to prepare for our DIA 2013 49th Annual Meeting: Advancing Therapeutic Innovation & Regulatory Science, Karen shared these thoughts about her DIA 2012 experience and how that experience can benefit the patients this organization represents.
Q: What are some of your Approximately 10,000 individuals constituents’ biggest fears in the US live with Sturge-Weber about participating in a clinical syndrome (SWS) and its hallmark trial and did you see or hear features of a port wine birthmark Karen Ball anything at DIA 2012 that could on the face, glaucoma, and help alleviate or overcome seizures with varying degrees of those fears? cognitive and physical impairment. Over one million people have a port wine birthmark on one particular body part. Because the vascular malformation involves gf GLOBAL FORUM | EDUCATE
so many parts of the body, these involved?’ The SWF always Wine Birthmark conditions through individuals (depending on the includes this information on collaborative education, advocacy, degree of involvement) must the risks as well as potential research and friendly support.” juggle medical appointments with benefits of involvement but this neurologists, dermatologists, question comes up often. DIA I founded the SWF 25 years ophthalmologists and therapists. 2012 was beneficial in helping ago when my daughter was So far, there are less than a me understand even more about diagnosed at birth with Sturge- handful of SWS clinical trials for the clinical trials process and Weber syndrome. Following our members. The SWF’s goal the benefits of telemedicine, three eye surgeries in the first is to increase the number of and getting to meet vendors three months of her life, I began these clinical trials through our in this area. Another bonus of a crash course on the lifelong ten Centers of Excellence across DIA 2012 was the information medical and other implications the country. The SWF helped shared amongst all the patient she would face with this rare facilitate an Office of Rare Disease advocate fellows. Since many of disease. It was a short course: A Research (ORDR), National us don’t have dedicated staffers handful of medical articles on the Center for Advancing Translational for public policy or clinical trials, syndrome, demeaning photos Sciences (NCATS), Brain Vascular these exchanges solidified what associated with the articles, and Malformation Consortium (BVMC) we are doing well and identified less than a handful of ‘experts’ grant to study three diseases, other channels of distribution that on the syndrome, most of them one of which included SWS we could employ across sectors neurologists who dispensed and the genetics of SWS, and to maintain communication and palliative care but with none our members have been very participation. focused on finding a cure. 92 responsive to participating at The SWF started out as a these Centers. SWF’s participation in umbrella and industry events such as DIA clearinghouse of information on One of the first questions 2012 provides global access the syndrome. In those days, members typically ask about to key opinion leaders, industry support was dispensed by phone
VOL 5 ISSUE 1 clinical trials is, ‘How much more leaders and scientists. The or with the postmaster’s help. time out of my schedule will it plethora of DIA 2012 sessions Slowly, word reached the medical take?’ They know the SWF has allowed me to choose tracks that community that there was a been at the forefront of engaging benefit our learning curve for trials central point to refer patients researchers and funding small today and will guide us to lay the to for support and awareness. research seed grants to obtain proper groundwork for a program Former First Lady Betty Ford and baseline data necessary for larger to utilize compounds that might Anne Landers helped boost the investigations. The SWF and our benefit the members in the future. organization’s profile and we were members have been in dialogue Our members are excited to off and running! The SWF was an through printed publications and know we are engaging individuals all volunteer organization for the online endeavors to become in dialogue and awareness first four years. When we added better versed on clinical trial opportunities at every turn. staff positions, our outreach and terms, the value of clinical trials fundraising efforts to support the and the need for participants May we ask you to please mission and infrastructure across which will increase the pace of explain and describe your the country grew. A research discovery. They understand the work with the SWF, and how fund was established with a Betty SWF has done our due diligence can organizations such as DIA Ford Award stipend and the on the trials before we notify them collaborate to help? National Organization for Rare for participation. Our members Disorders (NORD) provided an The Sturge-Weber Foundation are ready for more clinical trials opportunity to share and learn (SWF) is an international 501c3 because there have been so few with other emerging not-for -profit organization with a mission to on the syndrome. organizational leaders. improve the quality of life and care Another question that inevitably for people with Sturge-Weber It’s been a very humbling and comes up is, ‘What are the risks syndrome and associated Port exciting 25 years. The SWF has been responsible for increasing online, I am eager to engage a more from each other when we the number of health care new generation of clinicians and come together! Thankfully, DIA providers who know more investigators as we work together has been at the vanguard of about the natural history of the to improve the quality of life and advocacy inclusion and launched syndrome and now care for care for the members with more the Patient Advocates Fellows our more than 4,300 members. tailored treatment options. program. The SWF has become Increased internet communication a richer and even more exciting allows the SWF to assist families When I was invited by a physician community through our DIA and care providers in over 96 to speak at the DIA 39th Annual partnership and our patients countries. NIH co-sponsored Meeting in San Antonio (2003), I will ultimately benefit sooner workshops and member was looked upon as an anomaly rather than later. I look forward participation in studies through the and people kept asking me why to highlighting our blossoming informal Natural History database I was there. I found it odd then – partnership with DIA during the have confirmed the involvement and I still do now – that individuals Sturge-Weber Foundation’s annual of angiogenesis, immune who represent the patient would Month of Awareness events in suppression, growth hormone not be seen as a valued resource May. We welcome volunteers and abnormalities and more. As the and an equal partner at the supporters for a reason, a season SWF launches this database table. We can learn so much or a lifetime!
To learn more about the Sturge-Weber Foundation, please visit www.sturge-weber.org. 93
Be Smart – Sign Up for SmartBrief
Kathleen Doyle
Subject: FW: January 31, 2013 - Glaxo refuses settlement in U.K. Avandia diabetes cases
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Glaxo refuses settlement in U.K. Avandia diabetes cases Despite settling U.S. lawsuits over claims that the diabetes drug Avandia causes heart attacks email publication that gives quick, and strokes, GlaxoSmithKline has decided to fight similar claims in the U.K. It has become “increasingly difficult in the U.K. to challenge large corporations such as pharmaceutical companies," said Liz Thomas, policy manager at the patient-safety charity Action against Medical Accidents. Pharmalot.com (1/30) easy-to-read summaries of articles Europe
EMA to consider blood clot risks of newer contraceptives The European Medicines Agency will review third- and fourth-generation combined oral contraceptives' safety to decide if restricting their use is necessary. The move was requested by France, which wants to limit the newer contraceptives' use due to blood clots. "There is no relevant to you. Get the latest news reason for any woman to stop taking her contraceptive," the EMA said. "If a woman has concerns, she can discuss this with her doctor." Reuters (1/28) , PharmaTimes (U.K.) (1/29)
U.K. drugmakers and physicians call for transparency in financial ties in drug development innovations A coalition of British physicians and pharmaceutical companies have created a template for disclosing payments and other financial ties between drugmakers and doctors as the U.K. works toward greater transparency. Similar efforts are under way in the U.S. and France. The effort is to find "whether there is, in principle, support for a publicly available, single, searchable system for disclosure of payments," said Richard Thompson, president of the Royal College of Physicians. Pharmalot.com (1/29) worldwide wherever you are on Drug industry group disputes lowering cost-effectiveness thresholds Lowering the current threshold of quality-adjusted life years, or QALYs, for assessing the cost effectiveness of new drugs and medicines would deny such medications to thousands and lead to a decline in U.K. health standards, according to the Association of the British Pharmaceutical Industry. The group was responding to a study by University of York health economists who your laptop or smart phone with said the current standard "is not based on evidence" and should be reduced. PharmaTimes (U.K.) (1/30)
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SIAC Corner New SIAC for Medical Science Liaisons
david Cram
Corcept 94 Therapeutics
VOL 5 ISSUE 1 DIA’s mission to provide community is to raise awareness educational and networking of the MSL profession; to forums that anticipate and meet encourage dialogue about that the needs of our professional profession among regulators, constituencies requires the academic institutions, industry constant reevaluation and personnel and others; and assessment of these forums in the to promote the professional context of continual and dynamic development of MSL SIAC scientific, industry, regulatory members. and market change. As progress marches forward, some forums Rebecca Vermeulen, RPh are no longer necessary and may (Genentech) serves as MSL be left behind while new forums SIAC Chair. Dr. David L. Cram are created as other emerging (Corcept Therapeutics) serves needs are identified. on MSL SIAC Core Committee and shared his thoughts on the DIA is proud to announce the concerns of the MSL professional formation of a new Medical community, and how this SIAC Science Liaison (MSL) Special hopes to address them through Interest Area Community (SIAC). programming and networking, The goals of creating this new in the following Q&A. “I have to commend the leadership of DIA. Q DIA has done worlds of good for &A the MSL community,” David says. When was the MSL SIAC committee is led globally by proactively counter that without formed and what needs within Rebecca Vermeulen and in the some level of risk.” the healthcare community was US by Ramineh Zoka. I serve it formed to address? as a co-chair for our regional Another is the environment around development. Geoff Brockway compliance. We want to do the This SIAC has been in serves as co-chair for Canada. right thing but the rules can be development for a long time and Amy Gomez serves as European very hazy. You only know that you a very positive environment at lead. J. Lynn Bass is our break them when they call you. DIA over the past five years or so communications coordinator I started in this industry in 1991 has made this possible. Prior to and Ken Massey is our strategic and I can tell you that there were that, the MSL world was basically development co-chair.” things the industry considered relegated to a subgroup of the acceptable in 1991 that would put Drug Information SIAC. As the Do MSL professionals across us all in striped jumpsuits today. number of MSL teams grew over the pharmaceutical, medical Although the FDA really hasn’t the years, we felt it was important device, and biotechnology changed many rules, they enforce to have our own SIAC. At this industries have essentially them more heavily now than they point in time, there are probably the same needs, or are they did. Nasty letters from the FDA more MSLs in the pharmaceutical different? have evolved into fines. We ask world than drug information government regulators to fill in professionals. MSL organizations have become the rules for us, a double-edged a lot more uniform over the years sword because they become Some history: Back in the late but there is clearly still a difference even more conservative. Right 1980s/early 1990s, there were if you work for the device industry now, the general rules are: You do 95 probably three or four MSL versus the pharmaceutical not promote off-label indications teams. That number just exploded industry. The pharmaceutical in your drug. You can address throughout the ‘90s. By the year industry has a lot more restrictions questions associated with that. 2000, almost every company had than the device industry in You can review things with people an MSL team, and now every general, but that’s tightening up when they ask you. But you startup company has an MSL really fast. The device world has cannot proactively go out there team, of some sort – even small always had a lot more flexibility, and promote. That’s as simple as companies such as my own but I think that’s tightened up it gets. where we have one half-time quite a bit. So we are becoming drug information person and nine a lot more uniform in the things Other issues would include: MSLs. There’s this huge group we worry about. That’s the best How do you evaluate MSL now, and we felt like we needed way to put it: We worry about the performance? What are the career more representation than what same things. tracks that MSLs might have? How we’d been getting. Not that the is this environment changing, and Drug Information SIAC was not What are some of these if this environment is changing, paying attention to us or not giving concerns? how do we change along with it? us what we needed – they did a What is the measured value of an Where are the limits of ‘clinical really good job of that – but we MSL team? The main thing for exchange’? That’s the number were getting to that point where this DIA group is that we create a one concern right now: Where our needs started overwhelming forum for networking to talk about does ‘clinical exchange’ end and their needs. So we officially these issues. become ‘off label promotion’? Or, established the MSL SIAC core where does ‘clinical exchange’ team in May 2012. end and you’re no longer able Please visit “Networking & Communities” on our We started looking around at to provide a balanced argument www.diahome.org website global MSL leadership and against somebody who’s established a core group mislabeling your drug? For to learn more about the and communication group of example, if managed care says MSL SIAC and other DIA international as well as national something that mislabels your Special Interest Area experts in this field. Our core product, we are in no position to Communities. gf GLOBAL FORUM | EDUCATE
SIAC Corner Don’t Be Caught off Guard: Meeting Tips for DIA’s Annual Meeting
Lisa Palladino 96 Kim
VOL 5 ISSUE 1 Lisa Palladino Kim The DIA 2013 49th Annual 1. Set objectives and narrow just completed a masters Meeting: Advancing Therapeutic your focus before attending. degree in Clinical Sciences Innovation & Regulatory Science is The DIA Annual Meeting offers from University of Medicine and Dentistry of New Jersey. “the largest multidisciplinary event over 19 different interest In addition, she has 13 that brings together a community tracks, across three levels years of experience in the of professionals from all levels of experience. You will feel pharmaceutical industry and across all disciplines in the like a child in a candy store, across both Clinical Sciences discovery, development, and life so approach your choices and Clinical Operations. cycle management of medical systematically. She is currently employed products.” at PharmaNet/I3, dedicated a. Select one or two tracks to Merck & Co., as a Global As in years past, DIA 2013 you are currently working Trial Optimization Specialist. will feature 280 sessions with on or learning about, a Most recently, Lisa developed comprehensive coverage of track you would like to work a process for engaging today’s hottest medical topics. on in the future, or a track worldwide investigators in early program and protocol If this is your first DIA Annual of personal interest. design with the expectation Meeting with over 200 speakers of improving operational and 450 plus exhibitors, you may b. Determine your level of execution. feel overwhelmed initially, but knowledge per track and relax, take a deep breath, and choose accordingly. invest some time into planning. Also remember, sessions you do This is key for an excellent not attend in person are offered experience and pre-conference online after the conference. planning is best. To enhance this experience, here are some tips to consider: 2. What to wear: Business casual be overwhelming. Therefore, the follow-up e-mail, attach with comfortable shoes and when you receive one, the photo of the two of layers. You may be tempted record a few distinguishing you. This provides a unique to wear a pretty new pair of notes about the person or opportunity to remind the high-heeled shoes, but keep conversation on the back: person who you are and in mind the conference space location (exhibit hall near DIA when you met them. is vast; envision football field booth), appearance (bright size in length with sessions at pink suit), or topic (2 yr old 7. Ensure your badge has the both ends. You will do A LOT son, school for pharmacy bar code facing out to make it of walking, and do it all over degree, etc.). Save time spent easier for scanning. DIA tracks again the next day, so save sorting these: Pick one pocket attendees at each session; the stylish shoes for another for “follow-up” and one pocket they will scan your badge prior event. In addition, wear layers, for the “future reference” to entering a meeting session. because it is impossible business cards. 8. Volunteer! DIA is always to make the temperature looking for people to help comfortable for everyone who 6. Network! Network! Network! with the annual conference. attends. There are countless opportunities to meet diverse Contact the volunteer group, 3. There are many, many and potential colleagues! offer to take photos, or exhibitors; therefore, be perform informal surveys. strategic with your choices a. If you’re reserved, you may 9. Give your opinion. DIA before navigating the exhibit be uncomfortable meeting improves each annual meeting hall. Prior to the meeting, new people. Take a risk 97 based on feedback from plan your visit to this area, and introduce yourself, attendees. and create a targeted list you’ll have a high chance of meeting somebody who of top exhibitors. If you are 10. Trim your conference carry will change your life. If you looking for employment, visit items, bring only what is want to practice, be sure to the booths dedicated to “job necessary, and of course stop by the DIA Advocate hunter.” If you are looking for don’t leave your personal Patient Fellow booth for a new ways to recruit patients items unattended. What feels very interesting and diverse for your clinical study, focus light at 7 am will feel heavy group! on the recruitment vendors, by 5 pm. Utilize the DIA app etc. b. Scope out lunch tables and or pull out the schedule-at- a-glance sheet from the DIA 4. Leave space in your suitcase use this as a networking program. Plan the best time to for conference and exhibitor opportunity to meet new pick up exhibitor information items! On top of receiving people with different so you are not carrying around conference materials, background or expertise. extra weight for 8 hours. business brochures and fact Everyone has something to share. sheets, exhibitors may offer Enjoy DIA’s 2013 49th Annual reminder gifts, which can be c. Keep an ear out for non- Meeting! In addition to this really fun for you and others in DIA sponsored informal get- amazing learning opportunity, your office. They often request togethers after the meeting. it provides an excellent a business card or will scan These provide another great opportunity to meet people your badge. opportunity to network and from around the globe, share your views and knowledge, and 5. Bring tons of business can be tons of fun! establish new relationships! cards, and then bring more. d. T ake a picture of yourself Exchange business cards next to the key person you with attendees and exhibitors. want to follow-up with at a With XX amount of attendees, later date. Prior to sending your collection of these can gf GLOBAL FORUM | EDUCATE
SIAC Name Change
DIA is a global member Below is a listing of association that provides all the active SIACs and knowledge resources across the their Chairpersons full spectrum of medical product development. In the last few years SIACs and Chairpersons the organization has expanded Anti-doping that global reach and will continue Chairperson to grow to meet the needs for Barbara Leishman health care knowledge exchange F. Hoffmann-La Roche Ltd. around the world. As DIA’s reach 98 Switzerland expands globally, our volunteers have suggested that the term Biotechnology and Innovative SIAC does not resonate globally in Preclinical Sciences its explanation of what the special Chairperson interest groups represent. VOL 5 ISSUE 1 Joy Cavagnaro The CLC (Community Leadership Access BIO Council) has discussed this topic United States and asked for feedback from the SIACs. The consensus is that Clinical Data Management changing the name “SIAC” to Chairperson “Community” will enhance the Kristin Neff visibility and purpose of these TARIS Biomedical, Inc. groups. The CLC has also agreed United States that if a region wishes to use a local language term other than Chemistry Manufacturing and Community that will be more Controls and Quality Systems acceptable to their membership, Chairperson they are free to use that regional Georges France term. Novartis Consumer Health S.A. Switzerland
Clinical Pharmacology Chairperson Royce Morrison Quorum Review, Inc. United States Clinical Research Good Clinical Practices and Professional Education, Training Chairperson Quality Assurance and Development Susan Nunchuck Chairperson Chairperson Actelion Clinical Research Leslie Sam Daniel Mudgett United States Eli Lilly and Company Medidata Solutions Worldwide United States United States Clinical Safety and Pharmacovigilance Investigator and Investigative Project Management Chairperson Sites Chairperson Michael Bui Chairperson John Sun Bayer Healthcare Pharmaceuticals Ana Marquez Novartis Pharmaceuticals Inc. Marquez Clinical Site Partners, Corporation United States LLC United States United States Clinical Trial Disclosure Quality Risk Management Chairperson Information Technology Chairperson Robert Paarlberg Chairperson Peter Schiemann Paarlberg & Associates LLC Pamela Campbell Widler & Schiemann Ltd United States EMC Corporation Switzerland United States Devices & Diagnostics Regulatory Affairs Chairperson Medical Communications Chairperson Shayesteh Fuerst-Ladani Chairperson Sarah Powell 99 SFL Regulatory Affairs and Stacey Fung Liquent, Inc. Scientific Communication Genentech, Inc. United States Switzerland United States Statistics Documents and Records Medical Science Liaison Chairperson Management Chairperson Joan Buenconsejo Chairperson Rebecca Vermeulen FDA Lisa Mulcahy Genentech, Inc. United States Mulcahy Consulting, LLC United States United States Study Endpoints Medical Writing Chairperson eClinical Chairperson Keith Wenzel Chairperson David Clemow Perceptive Informatics Munish Mehra Eli Lilly and Company United States Global Alliance of Indian United States Biomedical Professionals Translational Medicine United States Natural Health Products Chairperson Chairperson Aamir Shahzad Emerging Professionals Pradip Paul European Society For Translational Chairperson Strategic Pharmacovigilance and Medicine (EUSTM) Yasmin de Faria Krim Risk Management Consultant Austria Janssen Pharmaceutical United States Companies of Johnson & Johnson Validation Belgium Pediatric Chairperson Chairperson Richard Chamberlain Gesine Bejeuhr Executive Consultant Services vfa Research-based United States Pharmaceutical Companies Germany ASSOCIATIONASSOCIATION ggff NEWSNEWS
ASSOCIATION NEWS compiles the
latest updates from DIA plus news
from and about our global network
of members and volunteers in one
comprehensive section. DIA is pleased to announce ASSOCIATION Stephen P. Spielberg, MD, PhD, former FDA Deputy Commissioner for Medical Products & Tobacco, NEWS as TIRS Editor-in-Chief effective March 1
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Dr. Spielberg, a pediatrician who – more than 17,000 around is the former dean of Dartmouth the world – and to interpret Medical School, has deep ties that information, to ensure that to the pharmaceutical industry. the public has access to safe, Until he was selected by Margaret effective and high quality medical Hamburg to serve in the Office of products. We see an opportunity the FDA Commissioner, he was here, with the rapidity of change in Director of Personalized Medicine science, the rapid accrual of new at Children’s Mercy Hospital in understanding of the mechanisms Kansas City. He also served for of human disease, and converting six years as Johnson & Johnson’s that understanding of mechanisms Vice President for Pediatric Drug into new products that I could not Development, after tenure in have even imagined when I began senior positions at Merck & Co’s medical school more than 35 Research Laboratories. He is also years ago.” a former member of PhRMA’s pediatric task force. “With that rate of accrual of new knowledge, we have a challenge “I’m incredibly excited to have of how to inform each other joined the DIA at this time,” said about science – everything from Dr. Spielberg. “Medical science is basic science and genomics, advancing at an ever-increasing rapidly understanding how the rate, and the journal is part of human genome impacts health DIA’s efforts to ensure the flow of and disease – and how that information to all our members converts into the discovery, the 102 VOL 5 ISSUE 1 drug development,everything consequences ofinternational about theimplicationsof as wellthinkingmore broadly with theacademiccommunity; regulatory community, together medicines, togetherwiththe of industry, ofdiscoverers ofnew of scientists,drugdevelopers, idea ofbringingnewgenerations I’m extremely excitedabout the interventions forseriousillness.So of improved diagnostics and on behalfofallpatientsinneed new informationisinfactused a majorstakeinensuringthatthe through regulatory science,have in academiathrough industry, involved, from basicscientists products. Allofuswho are and theuseofnewmedical development, theregulation, g g Proposed 2013 Topics Upcoming SpecialFocus Sections INFORM specialfocus sections. for ourADVISE best practices section andour section editors to write and/or solicitarticles The GlobalForum islookingfor authors and Would you like to beapartoftheGlobalForum? Call for Contributions f • Scientific Regulatory DecisionMaking • Pharmacovigilance intheEuropean Union • Integrity oftheSupply ChainManagement • AIDS Retrospective f GLOBAL FORUM |ASSOCIATION NEWS It certainlyisaboutinnovation, our thinkingaboutthefuture. synthesizes“The titleofthejournal health,” Dr. Spielbergsaid. a stakeinthefuture ofhuman together allofthosewhohave – inanintegratedwaytobring activities throughout theworld oureducational– ourjournal, be abletouseallofourresources DIA isunusuallywell-situatedto discovery intoreal products. And To converthumanbrillianceand “All oftheseare hugechallenges: medicines.” the availabilityofcomplexnew to thinkabouttheeconomicsof medicines; through how we need of medicinesandconstituents from supplychains, international deadlines. article topics and/or submission diahome.org withquestions on Judy Connors, at judy.connors@ Contact theManagingEditor, GLOBAL GLOBAL H 8-10 O SPECIAL REPORT: C THE IN THIS ISSUE IN THIS WOrLd REGISTER LINICAL EALTH 6 T EMA TH HE N PA CTOBE 2012 r ETHErL A H GE & F & C NNUAL F A F dA, ANAdA, 83 GUE Or NOW Or dA ANdS UM UM ,
INFORM ADVISE
forum VOL 4•ISSUE AUGUST 2012 REACH
EDUCATE g g f f
medicines inclinicalpractice.” more rationalandeffective useof distribution processes, through manufacturing through new molecular biologythrough new everything from understanding and innovationinallrespects, under our“News&Publications.” find theminthe“Newsroom” www.diahome.org website.You’ll “Publications &Research” onour & RegulatoryScienceunder visit TherapeuticInnovation please and ournewjournal, share histhoughtsonDIA To seeandhearDr. Spielberg Cover andFrontend.indd1 GLOBAL GLOBAL REIMBURSEMENT IN THIS ISSUE IN THIS DIA 2013CHAIR NEW SECTION: HEALTHCARE ANNOUNCED PAYERS AND WOMEN IN PART 2 PAGE 116 PAGE 107 PAGE 10 PAGE NOMAVS EC EDUCATE REACH INFORM ADVISE forum VOL 4•ISSUE5 OCTOBER 2012 IN2013 DIGITAL GOING in December sneak peak g g f f 9/26/12 5:04PM FDLI Honors DIA Board Member Minnie Baylor-Henry
DIA President-elect Minnie Chair further demonstrate her Baylor-Henry, JD, RPh (Johnson commitment and leadership.” & Johnson Medical Devices & Diagnostics) received the FDLI, founded in 1949, is a non- Food & Drug Law Institute profit organization that provides (FDLI) Distinguished Service & a marketplace for discussing Leadership Award at FDLI’s annual food and drug law issues through Holiday & Leadership Award conferences, publications and Reception held at the Westin member interaction. The scope of Georgetown in Washington (DC) FDLI includes food, drugs, animal this past December 12. The drugs, biologics, cosmetics, 103 FDLI Distinguished Service & diagnostics, dietary supplements, Leadership Award, established medical devices and tobacco. in 1993, annually recognizes Upon receipt of her award, Minnie up to four individuals who have Geoffrey M. Levitt, Immediate Past Chair and Susan commented, “I have worked a record of sustained service, C. Winckler, RPh, President & CEO, FDLI, presents for 20+ years as a regulatory leadership and contribution to Minnie Baylor-Henry with the FDLI Distinguished professional in pharmaceuticals, Service & Leadership Award. the food and drug law community consumer products and medical and/or FDLI; or provided an devices. To be recognizedwith exceptional contribution to FDLI such an honor by FDLI is one and/or the food and drug law of the greatest moments of my community, for example, through career.” outstanding writing or scholarship, by developing new legal theories Alan Bennett (Ropes & Grey, and precedents or engaging in DC Office), Thomas O. Henteleff significant activities on behalf of (Kleinfeld, Kaplan and Becker) and the public interest. John M. Taylor III (Counselor to the Commissioner, Office of the “Minnie’s service to the food Commissioner, FDA) also received and drug law community – as a this award. regulator with FDA, consultant with Deloitte and executive with Johnson and Johnson – has been extraordinary,” stated Susan C. Winckler, FDLI President and CEO. “Her contributions to the FDLI Board of Directors as Q 104 & VOL 5 ISSUE 1 g g f f A GLOBAL FORUM |ASSOCIATION NEWS Gardineer Bayard American Institute ofCertified as wellamember ofthe (Certified PublicAccountant) York atAlbanyandisaCPA from theStateUniversityofNew master’s degrees inaccounting hisundergraduateandHe earned Director/Senior FinancialAuditor). Auditof America(InterimInternal Operations) andSonyCorporation (Corporate Controller/Director of of Finance),HeraeusInstruments PresidentGeneral Manager/Vice Vocaltec Communications(as executive financialleadershipto Bayard hasalsocontributed Hurricane Katrina. tsunami, Haitiearthquakeand response totheIndianOcean the Philadelphiaarea alone,in million ofhumanitarianrelief, from that generatedwellover$50 oversaw fundraisingoperations and $50millioninassets, than $50millioninannualrevenue responsibleVirginia), formore National CapitalArea andWest Pennsylvania, Maryland,the for UpperDivision6(Delaware, and provided financialoversight strategies forfinancialstability chapters, where hedeveloped regional AmericanRedCross (CFO) ofonethenation’s largest served asChiefFinancialOfficer Officer forDIA.Bayard previously Director ofFinance/ChiefFinancial welcomed Bayard Gardineer as headquarters inHorsham(PA) In January2013,DIAworldwide Director ofFinance/CFO DIA Welcomes New What mostattractedyouto Forum. these thoughtswiththeGlobal technology efforts andshared finance operationsandinformation Bayard willoverseeDIA’s global patient care.” products andimprovements in of safeandeffective medical the developmentandregulation allow DIAtocontinuesupport wealth offinancialknowledgewill Director, PaulPomerantz.“His said DIAWorldwide Executive experience toourassociation,” Bayard bringsexceptional profile worldwidecorporations, acareer spanninghigh- “With the Year. of BusinessasNonprofit CFOof Drexel University’s LeBowCollege andPhiladelphia BusinessJournal Bayard wasrecognized bythe Public Accountants.In2009, communities? serve theworld’s healthcare which youcouldcontinueto Johnson and Johnson’s ‘Special the leadmechanical engineerfor health care. In1979,myfatherwas I liveinNewJerseyisbecause of Interestingly enough,thereason been interested inhealthcare. medical devices,butIhave always other groups, from biomedicalto works withaswellvarious many ofthemarketsthatDIA I haveanextensivehistorywith DIA asanorganization through
Research Group’ that helped to India, Japan and Switzerland, and I of Princeton currently working make ultrasound an everyday love that international component. toward his PhD at Stanford; my diagnostic tool. He was an MIT Now the mission for me isn’t just daughter is at Syracuse, looking graduate who loved the field, regional – it’s global. I worked to pursue her graduate degree and so do I. In fact, I worked for various types of international in psychology; and my youngest several summers with the Special companies earlier in my career, son is going to Centenary College, Research Group. It was new so I feel comfortable in this looking to be a business major. and really exciting. In addition, environment. It’s even better when The simple fact that they’re I feel comfortable in the non- you’re looking at many of those attending college leaves my wife profit as well as in the for-profit regions or locations as potential Mary and I a lot of extra time, environment. That said, non- growth opportunities: It allows me at least on weekends, that we profits typically require action that to get in on the ground floor as have never had before. We love is very often above and beyond part of that growth as well as to to do cultural things. We love the call of duty. It’s not unusual help manage the cost associated Philadelphia and New York. We – it’s expected. Many who work with it. It’s a chance to really make love cultural events and we also in this type of environment are a difference. love theatre. One of my other extremely caring and dedicated passions is reading US history, As you join a new company in to excellence. It’s something I especially American history an environment undergoing personally strive for each and every with a particular love for our such profound and extensive day. To sum up, it’s a logical next Founding Fathers and the Civil change, what’s your primary step in my professional career. War. According to family lore, I focus? am a descendant of President What previous professional Stewardship of the non- John Adams, of Vice President 105 experiences will most benefit profit dollar and providing the Aaron Burr, and of Nathan Hale. your service with DIA, and necessary back-office support Last but certainly not least, I love how? which inevitably allows others baseball and my team is the New Most certainly, my recent to be more proactive as well as York Yankees. experience serving eight years effective. We are a team and we with the American Red Cross, must all work together to provide What message would you an organization with a strong our members with support and like to share with DIA’s global and extensive volunteer network, services. It’s critical to provide network of members and highlights the fact that when you’re the organization with efficient volunteers? working with others you can really and cost-effective back-office I’m thrilled to manage DIA’s accomplish a lot with very little. financial support to help it not only financial operations as the You also learn that it’s not all about provide the necessary services association continues to expand you, but about the mission. My but the flexibility to grow, despite globally and bring high-quality ‘value add’ was stewarding each a challenging environment. When programs and services to our dollar donated to the organization. the non-profit dollar is tough members and stakeholders I think it highlighted the fact that to come by, the reality is that around the world. I have every dollar is precious, and that you’ve got to provide enhanced already seen our members, our it’s very important to challenge services with greater quality. volunteers, our Board and our everyone involved with being With the worldwide web, the staff all go above and beyond the effective and financially cost instantaneous transmission of real call of duty whenever possible. efficient. It’s important to note that, time information, and competition One thing is for sure: I’ve already despite moving on professionally, being what it is, it’s extremely seen at DIA a tremendous I remain a dedicated Red Cross important to make sure that energy and a true willingness on donor and volunteer. diligence prevails. everyone’s part to deliver the best to our members. What opportunities with DIA What do you enjoy when you’re are you looking forward to the away from the office? most? I am the proud father of three. My Working in and around health care. children are in various colleges: Currently, we have offices in China, My oldest son is a graduate 106 VOL 5 ISSUE 1 Q & g g f f GLOBAL FORUM |ASSOCIATION NEWS A Lyn Jytte g vi g
DIA Welcomes New Director, Environment andtheTechnical City ofCopenhagen,Ministry Transportation, Mercuri Urvala/s, with Greater Copenhagen Director orConsultantpositions Prior tothis,Jytteservedin Copenhagen BusinessSchool. on individualassignmentsatthe participates asaSeniorAdviser Group. Sheadditionally as ChairoftheManagement and HeadsofMedicinesAgencies Management Board asVice-chair the European MedicinesAgency Jytte participatedasamemberof the DanishMedicinesAgency, In additiontoservingasCEOof Europe, theMiddle EastandAfrica. for oversight ofDIAprograms andoperationsin Agency. In position,shewillberesponsible hernew Chief Executive Officerofthe Danish Medicines especially inEurope, asthe andmostrecently served considerable breadth anddepthofexperiencetoDIA, asDirector,service DIA Europe. Dr. Lyngvig brings On January 10,2013,Dr. Jytte Lyngvig began challenges. she spokeaboutitspromises and she prepared forthisnewposition, and sixregional staff leaders.As consists ofDIA’s globalexecutives global managementteam,which and willalsoserveonDIA’s our staff inBasel,Switzerland, Council ofEurope, supportedby collaboration withtheAdvisory She willworkinclose University ofDenmark. Research attheTechnical Statistics andOperations from theInstituteofMathematical a PhDinSocioeconomicPlanning Jytte isaChemicalEngineerwith University ofDenmark. DIA Europe
What most attracted you to empowered citizens’ because some areas that we would have DIA as an organization through we’re all ‘patients to be.’ I have tolerated fifteen years ago. It’s a which you could serve the always preferred to talk about moving target because what is health care communities in citizens and not patients. moving is society. And society is Europe? citizens. And therefore we need to In some ways, this concept is be involved. It’s really complex. What first attracted me was DIA’s contradictory. In general, or on obvious role in the development of average, we have more access Q: What unique challenges health. At the moment, the health to knowledge. We have the and benefits does the agenda is changing all over the internet, but we also have much European Union (EU) world, including Europe. And to be crap in the internet. And so, this legislative framework present part of that, with the platform of empowerment is nearly two-faced: to the science of therapeutic DIA as a non-profit, neutral global There is much more knowledge out innovation and regulatory organization, is a gift. there, but there is much more non- approval? validated knowledge out there. What opportunities to serve I have been part of that system, in this new position are you In some way, people have to through my service with the looking forward to the most? be taken seriously. We are the Danish Medicines Agency, and citizens. It is our lives, and we I can see all the advantage The challenge in Europe, as in need to have influence in that of it. Normally, it is seen as a nearly the rest of the world, is way. Demands and understanding disadvantage. The US with the the financial challenges of the are a part of the society that we FDA, for example, and everybody society. You have citizens who are are living in nowadays. There’s in Europe says, ‘Oh, if we just had 107 used to a well-functioning health no way back. Instead of being an FDA and then everybody could system and democracy also puts frustrated – because I know be settled in one minute.’ That’s a on challenges. We will have to, many are frustrated, that patients part of it. all together, find our way through, complicate things and they don’t because if we just go on like we know things and so on – I think But the strength of the European are now, in five or ten years we will that we should see how it can add system – and I prefer to talk go bankrupt. Nobody wants that. to the decision. about its strengths instead of its weaknesses – is that we On the other hand, you have the Benefit/risk is a key discussion have managed to create a challenge of innovation. You have in this area: Benefit/risk seen European medicines network. more empowered patients and from a regulatory point of view We have the European Medicines citizens, and much more large is one thing; benefit/risk seen Agency, which has their roles demand for quality of life. I’m from a scientific point of view is to play. We have the European in a generation where we had something else; benefit/risk seen Commission. And we also have everything, always, and we will from a patient point of view is national agencies, and you cannot always be demanding. And, of something third; benefit/risk from understand Europe without course, its European background, a future patient is something else understanding that these are a very social health system. again. In some way, if we continue working together. And this has There needs some sort of radical this example, benefit/risk should change. Again, the possibility to be balanced out and the decision be part of that is a privilege. should level out the state of the art and the understanding of Why is the concept of “the society. Could we tolerate that? empowered patient” so Today we will not tolerate risks in important? ‘The empowered patient’ is a very strange expression. If I could express that in another way, just to challenge that, I think it’s ‘the 108 VOL 5 ISSUE 1 academia in from industry, regulatory, and is harmonizinghistory. harmonizing healthorhealthcare historical differences. Because has advantagesincopingwith that thiscomplexsystemalso never work.We sometimesforget just say‘Onesizefitsall,’itwould Europe.than Northern Andifyou Europe iscompletelydifferent the useofantibiotics:Southern to health.Theeasiestexampleis historical andculturalapproaches take intoaccounttheverydifferent Europe? Itaddsthepossibility to What doesthatadd,atleastto together inascientificnetwork. staff atalltheagencies working the EMA)inLondon,itisall is notonlythepeoplesitting(at way. Butthebaseofregulators differences goinginthesame been veryhard, tohave all these mention statistics,andalsosome computer science,youcan science. You canmention pharmaceutical ormedical science. It’s muchmore than or whateveryoucallitisa The newhealthcare agenda This questionisveryrelevant. help themostpr disciplines orareas can I n whatspecificprofessional g g f f E urope? GLOBAL FORUM |ASSOCIATION NEWS ofessionals DIA
synergy ofreally working together. scientific participantstoseethe will require theeducationofall fully integratedfrom the start. This some statistics’butthattheyare pharmaceutical science‘adding rely notonmedicalscience or in innovationhealthcare will sciences andsoon.Development of themore advancedbiological pleasant. Buthowwouldyouever creates thingsthatare notso war technology, whichofcourse improved tocreate muchmore military technology, andthey mathematics andsoon,within efficient useofbiostatistics, group whichpilotedthe was combined,amultidisciplinary the USorUKperhapsit There wasputtogether, eitherin during theSecondWorld War. know, wassotosay‘invented’ Operations research, asyoumight you anexamplefrom myworld. other scientificareas. Icangive Another wouldbetoinclude research. let’s dare toaskforefficiency in to makeitmore efficient but don’t havetheanswersofhow efficiency ofclinicalresearch. I in research. Iwouldfocusonthe multidisciplinarity andefficiency It wouldbetoarriveattrue would youfocusonfirst? enterprise in about theclinicalresearch I f youcouldchangeonething E urope, what very muchtoinnovation. paradigms. Thatwillalsomean will alsohaveashiftofthese based understanding,thenyou understanding toamolecular- a symptom-baseddisease is adisease?’Ifyougofrom our understandingof,‘What that way, whichwillchallenge discussion, ifyoucouldputit The otheristheemerging together inaglobalizedworld. for communication–getting not onlyforefficiency butalso there are lotsofopportunities strangers inthatworld.Ofcourse, that worldandotherswhoare the peoplewhocansurvivein have thisdivisionbetween that wesimplygiveup,soyou so complexandcomplicated opportunities butsometimesit’s the people.There are many and servethebusiness it trulyasatooltosupport information technology, tosee One oftheseopportunitiesis meets allstakeholderneeds. multidisciplinary approach that efficiency inresearch andatruly goes,’ more quickly. Focuson get ridoftheothers,‘non- spot thepotential‘goes’and Efficiency isalsotobeable to improve thetimeandefficiency. too long.Thegoalisofcourseto do itthetraditionalway, ittakes have tothinkoutofthebox.Ifyou platform forinnovation,thenyou If yousaywehaveaburning ‘traditional development’? War ifyouhadputthatinto France duringtheSecondWorld have theinvasioninNorthern But I don’t think any of these are What message would you like quick and they will be very difficult to share with DIA’s network because you will challenge the of members and volunteers in traditional system and therefore Europe? will also challenge the people who are the spokespersons for this I would first say hello! I am very system, and that’s always a little happy and challenged to work difficult. with you, and I really appreciate the contributions from our What message would you like volunteers, members, individuals to share about DIA’s upcoming from industry, regulators, and so 25th Annual EuroMeeting: on. Because, at the end of the Amsterdam 2013? day, this area is so complex but also so important. If we don’t go It’s a very important meeting. It is this way together, we will not be a great opportunity to strengthen able to achieve what we all want your network and listen and at the end of the day: Not eternal contribute to the very important life, but better quality of life. discussions in the sessions. We have a great program. What topics can you get? You can get everything! It’s a very easy way to be in one spot and be completely 109 up to date in very attractive surroundings in Amsterdam, meeting all your good friends and making new friends.
New Director on New Pharmacovigilance Legislation in Europe
In an exclusive November 2012 interview with Scrip Regulatory Affairs, Jytte also commented on the new pharmacovigilance legislation in Europe, which will bring tremendous change to the drug regulation system in the EU.
It establishes a new EMA pharmacovigilance committee (PRAC) to contribute specific assessments or reports that will be in the public domain and must be taken into account by the Committee for Medicinal Products for Human Use (CHMP), the EMA’s key scientific committee. The legislation also allows the PRAC to hold public hearings. “I think it’s very important that we firstly find out what should they be used for, where will they give added value to the process,” she explained.
This new legislation has to work regardless of its challenges, she said. There will be problems – “but it’s our duty to overcome.”
To read the complete interview, visit www.scripregulatoryaffairs.com. gf GLOBAL FORUM | ASSOCIATION NEWS
GOVERNANCE Corner DIA 2012-2013 Philanthropy Grant Program Announcement
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VOL 5 ISSUE 1 2012-2013 was a landmark year for the DIA Philanthropy Grant Program. A record-breaking 62 eligible applications were received. The Philanthropy Committee had a very difficult task of selecting winners to receive the limited funding.
It was not an easy choice, as each Rare Genomics Institute and every application showed Derwood, Maryland, USA compassion for patients and a determination to improve quality Arthritis Foundation, of life. On behalf of Chairman Northeast Region, Inc. Tatsuo Kurokawa and the DIA New York, New York, USA Philanthropy Committee, we are proud to announce the awardees Fundacion Huesped of the 2012-2013 Philanthropy Buenos Aires, Argentina Grant Program: Congratulations to these three fine organizations! We also announce at this time programs – including fellowships that, upon the awarding of and education – beginning in We extend heartfelt this year’s funding, the DIA January 2013. “The DIA patient thanks to the DIA Philanthropy Grant Program will initiative includes programs in five Philanthropy Committee sunset in its current form and will events, including the EuroMeeting, for their time and be transformed to provide direct European Rare Disease dedication: support to DIA’s Patient Advocate Conference, North America Dr. Tatsuo Kurokawa, Chair Fellowship program. The DIA Annual Meeting, North America Keio University School of Philanthropy Committee will be Rare Disease Conference, and Pharmacy dissolved. EuroMeeting Clinical Forum. The Karen Arts bottom line here is that patients Ontario Institute for Cancer The DIA Philanthropy Program will continue to be at the center Research began about 15 years ago with of the funding,” stated DIA an annual budget of $150,000. Worldwide Executive Director Paul Kelley Hill It helped to promote individual Pomerantz. Dr. Tatsuo Kurokawa Shire Human Genetic Therapies, Inc. and group activities which met added, “At the same time that DIA the mission and vision of DIA. was promoting grant funding for Truus Janse-de Hoog However, while DIA is still rooted others, we have been developing Medicines Evaluation Board in supporting causes that benefit several initiatives that also serve a the public, some important Larisa Nagra Singh philanthropic purpose. For seven Quintiles changes have taken place over years, DIA has had various Patient recent years. As patients and Fellowship programs in Europe Per Spindler their organizations play a role and North America, and there is BioPeople; University of 111 of increasing importance in interest in expanding this globally.” Copenhagen research, regulatory and policy For additional information about matters, the DIA Board of DIA’s patient initiatives, please Directors (BOD) agreed in 2012 contact Donna Mayer at donna. that the philanthropic resources [email protected]. should be directed to support DIA’s growing portfolio of patient
DIA 2013 Board of Directors Election Just Around the Corner It’s never too early to start thinking of the upcoming DIA Board of Directors election. Even though the election is still a month away, please plan ahead and ensure that your member profile includes your current email address. In mid-March, look for an email that provides your personal link to the 2013 Election Ballot and secure electronic voting. Don’t miss your chance to vote in the annual DIA Board of Directors election!
With the disbanding of the Philanthropy Committee and the merging of two other committees, Bylaws revisions will be necessary. Your next issue of the Global Forum will provide more detailed information on these Bylaws revisions for 2013. 112 VOL 5 ISSUE 1 Administration Institute ofExecutive the ChineseState FoodandDrug Inc. HealsoservesasProfessor at Principal, EWHulihan&Associates, Alumni Award. Prof. Hulihanservesas Alumni Association’s Distinguished received theEastStroudsburg University P Harvard MedicalSchool. conducted herpost-doctoraltrainingat from PennsylvaniaStateUniversity. She in biochemistryandmolecularbiology Bloomsburg (PA) University, andherPhD in biologyandBAchemistryfrom Boston. Dr. herBS Dallabridaearned Hospital, andChildren’s Hospitalof Cancer Institute,Brigham&Women’s Anders Scientific,Zafgen,DanaFarber strategic advisorforBiogenIdec,Rubin previously servedasconsultant and PHT ConsultingServices.Dr. Dallabrida appointed SeniorScientificAdvisorfor D medicineandendocrinology.in internal Stanford University. Heisboard certified sciences andbiologicalfrom University, and aBASinpolitical General Hospital,hisMDfrom Columbia residency trainingatMassachusetts Pharmaceuticals. Dr. Chenreceived his Corporate DevelopmentforAmylin Director ofClinicalResearch and Regulus Therapeutics,andasSenior President ofTranslational Medicinefor Dr. Chenpreviously servedasVice Disease, forAileron Therapeutics, Inc. Development, Endocrine&Metabolic Presidentappointed Vice ofClinical D accomplishments. members fortheirrecent professional us incongratulatingthefollowingDIA and networkingforums.Pleasejoin and volunteersthrough oureducational professional practiceofourmembers DIA iscommittedtoimproving the rofessor r r . S . H g usan g ubert f f D C.C E allabrida arl hen GLOBAL FORUM |ASSOCIATION NEWS W. H Members ontheMove was ulihan was
LLC. Dr. holdshis doctoratein Ryan as Chairmanof theBoard forLevare, Research, LLC.Hepreviously served Consulting &Submissions,for INC President,Vice RegulatoryStrategy, D (CA). and herMBAfrom St.Mary’s College from theStateUniversityofNewYork herBSinbiochemistry Cindy earned Organization) PharmacovigilanceGroup. of theBIO(BiotechnologyIndustry Genentech. ShealsoservesasChair Safety/Documentation, forRoche/ Global RegulatoryOperations&Clinical previously President, servedasVice Cannon Research Institute.Cindy President, SCRIServices,fortheSarah C London, UniversityofLondon(UK). in pharmacologyfrom King’s College herBScwithHonors Alison earned Regulatory Affairs Professional Society. President andChairofthe Board ofthe and MassMEDIC,isapast Director ofCubistPharmaceuticals of Directors. Shealsoserves asa appointed totheVerastem, Inc.,Board Biosurgery). Inaddition,Alisonwas Sanofi Biosurgery(formerlyGenzyme President andGeneralManager of Alison previously servedas SeniorVice Chief OperatingOfficerofOvaScience. A DIA FoundersServiceAward. Hulihan isalsoapastrecipient ofthe Council ofNorthAmerica.Professor oftheDIAAdvisory and Vice-chair Steering Committee,andasamember a memberoftheDIAAnnualMeeting as ChairoftheDIAValidation SIAC, Chinese Medicine.Hehasalsoserved at ShanghaiUniversityofTraditional Professorof NewJerseyandVisiting at UniversityofMedicineandDentistry Development, asAssociateProfessor lison r indy . R obert P L erettie awton R y an hasbeennamed wasappointed , wasappointed D Toxicology. is adiplomateoftheAmericanBoard of in pharmacokineticsandgenetics, Carolina atChapelHill,master’s degrees toxicology from theUniversityofNorth of California, Berkeley.of California, molecular cell biology from the University herBAin Development. Dianeearned Alzheimer ImmunotherapyResearch & Clinical QualityAssuranceforJanssen served asAssociateDirector of Research, Inc.Dianepreviously of QualityAssuranceforProTrials D the UniversityofKentucky. microbiology andPhDintoxicology from Sciences. Dr. herBSin Thomasearned President, RegulatoryAffairs, forBiPar prior toSunesis,sheservedasVice Executive Director, RegulatoryAffairs; Inc. Dr. Thomas previously servedas Affairs, forSunesisPharmaceuticals, President,appointed Vice Regulatory iane r or emailaddress, too. your newjobtitle,employer contact informationtoreflect “MyDIA” andupdatingyour profile current bylogging into to keepyourDIAmember approval. Pleaseremember to DIAeditorialreview and submissions are subject [email protected]. All if youhaveone)toChris. resolution digitalphotograph, announcement (andhigh- Forum, pleasesendyour members inourGlobal or career newswithother to share yourprofessional DIA memberandwouldlike On t Kn . D eborah W ow h ong e M –Ifyou’re anactive wasappointedDirector A.T o ve? LetU homas , was s
DIA EuropE TrAInIng progrAMME 2013
Chemistry, Manufacturing and Controls (CMC) / Quality Safety and Pharmacovigilance
global CTD Dossier – regulatory aspects and focus on quality documentation Benefit/risk Management including concepts of Quality by Design 13-14 May 2013 | Zurich, Switzerland | ID 13523 1-3 December 2013 | Dubai, United Arab Emirates | ID 13562 26-27 September 2013 | Prague, Czech Republic | ID 13524 Quality by Design for Chemical and Biotech products – A hands-on course for the Diagnosis and Management of Drug-Induced Liver Injury (DILI) pharmaceutical industry and regulators 19-20 September 2013 | Paris, France | ID 13563 11-13 September 2013 | Vienna, Austria | ID 13559 How to prepare for pharmacovigilance Audits and Inspections 11-12 June 2013 | Nice, France | ID 13555 Clinical Research 7–8 November 2013 | Paris, France | ID 13556 pre-Marketing Clinical Safety Advanced gCp Study Monitoring 18-19 April 2013 | Vienna, Austria | ID 13526 5-6 June 2013 | Basel, Switzerland | ID 13549 Signal Management in pharmacovigilance Clinical Aspects of Quality risk Management and Quality by Design 10-11 June 2013 | Nice, France | ID 13557 19-20 September 2013 | Basel, Switzerland | ID 13560 6–7 November 2013 | Paris, France| ID 13558 Clinical project Management – part I 18-20 September 2013 | Basel, Switzerland European Medicines Agency Information Days and Courses Clinical project Management – part II EudraVigilance Information Day Dates and location to be confirmed 17 May 2013 | London, United Kingdom | ID 13529 Clinical Statistics for non-Statisticians 22 October 2013 | London, United Kingdom | ID 13530 24-25 October 2013 | London, United Kingdom | ID 13551 Excellence in pharmacovigilance: Clinical trials and post-marketing Essentials of Clinical Study Management 18-22 November 2013 | London, United Kingdom | ID 13522 17-19 April 2013 | Vienna, Austria | ID 13527 IDMp International Standards ICH M5/M2 and the Implementation of eSubmission of 20-22 November 2013 | Paris, France | ID 13554 MpIs in the Eu, Article 57(2) Information Day practical gCp Compliance Auditing of Trials and Systems 20 November 2013 | London, United Kingdom | ID 13531 23-25 October 2013 | London, United Kingdom | ID 13548 EudraVigilance courses: EudraVigilance – Electronic reporting of ICSR Non-Clinical Safety Sciences eXtended EudraVigilance Medicinal Product Dictionary Introduction to Pharmacovigilance and Electronic Transmission of Individual Case Safety non-Clinical Safety Sciences and Their regulatory Aspects Reports (ICSR) for the Use of Eudravigilance at the European Medicines Agency November 2013 | Lisbon, Portugal Courses throughout the year | European Medicines Agency, London, United Kingdom and Regulatory Affairs selected European cities.
Authorisation of Biopharmaceuticals, Biosimilars and Advanced Therapies in Europe For course details on EV, please visit www.diahome.org > Training > EudraVigilance > Click 18-20 September 2013 | Basel, Switzerland | ID 13546 on > Related Courses. European regulatory Affairs: In-depth review of current registration procedures in the European union 21-22 February 2013 | Berlin, Germany | ID 13525 DIA Europe Tailored Training 6-7 June 2013 | Basel, Switzerland | ID 13550 DIA Europe Tailored Training is a highly flexible, efficient and cost-effective way to get the 21-22 November 2013 | Paris, France | ID 13553 maximum return on your training investment. Schedule your training course when it suits you good Management of Medical Devices including In Vitro Diagnostics and Companion best, at the venue of your choice. You can even adapt the content to include areas specific to Diagnostics: Legal and practical aspects of devices your environment, and to match the level of expertise of the audience. 10-12 June 2013 | Amsterdam, the Netherlands | ID 13547 Health Authority Interactions – preparation, consultation and implementation DIA Tailored Training is available to both public and private institutions and is delivered by 15-16 October 2013 | Location to be confirmed instructors with no conflict of interest. Health Technology Assessment (HTA) 26-27 November 2013 | Zurich, Switzerland | ID 13561 The DIA Tailored Training programmes in Europe make the most of a selection of world- class expert faculty who are experienced professionals in the pharmaceutical and related paediatric Investigation plans (pIp) industries. 15-16 April 2013 | Amsterdam, the Netherlands | ID 13503 The Impact of regulatory Affairs on Chemistry, Manufacturing & Controls (CMC) Contact DIA Europe to discuss your organisation’s requirements. 2-4 October 2013 | Basel, Switzerland | ID 13532 uS regulatory Affairs: A comprehensive review of regulatory procedures for InDs and nDAs in the uS 6-8 November 2013 | Paris, France | ID 13552
For more information and a complete listing of all DIA conferences and training courses, please visit: www.diahome.org > click on Meetings & Training Call DIA Europe on +41 61 225 51 51 or email: [email protected] DIA 2013 Advancing Therapeutic Innovation and Regulatory Science
49th Annual Meeting | June 23-27, 2013 | Boston, MA | Boston Convention and Exhibition Center Play Your Part Foster Innovation to Promote the Development of Safe and Effective Medical Products and Therapies to Patients.
JUST Online Searchable Program RELEASED Now Available!
KEYnoTE SPEAKER
Daniel Kraft, MD Executive Director FutureMed
Start Playing Your Part Register Today! diahome.org/DIA2013
Submit your Abstract! Call for Professional Posters Deadline: February 28, 2013
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