GLOBAL FORUM | ISSUE 5 ƫĂĀāāƫđƫ ƫăƫ 10 Best Practices 22 Special Section: Comparative Effectiveness 100 Patient Fellowship Program IN EACH ISSUE President’s Message | 3 Regulatory Updates | 92 GFCovers and Spines.oct.indd 1 9/20/11 3:10 PM 0197V01GlobalForumAd212x277:Layout 1 27/4/11 22:28 Page 1 Reducing the Cost and Complexity of Safety & Risk Management Need more good reasons to switch to a hosted Total Safety™ solution? ARIS GLOBAL understands that by reducing the cost and Agrowing list of life complexity of safety, you can focus on what you do best. science companies and CROs, from small to large, agOnDemand™ eliminates the need to invest in new hardware, have chosen agOnDemand. software or support staff by giving you a ready-to-use solution on Request the agOnDemand a subscription basis. white paper. Fast deployment—typically in a matter of a few weeks—lets you easily ensure compliance and manage risk. 24x7 availability and end- Visit www.arisglobal.com/WP_agOD to-end support give you total peace of mind. Visit Booth #415 at the DIA 47th Annual Meeting GFCovers and Spines.oct.indd 2 9/20/11 3:10 PM OCTOBER 2011, VOL 3 ISSUE 5 GLOBAL FORUM Medicines’ Effectiveness, Role of a Patient, and Innovations ithin a rather short space of time (see our April 2010 W Global Forum), we have come back to the important topic of the real value of medicines. The question if a new Global Forum Editorial Board drug is better than any of those already available on the market has been gaining importance over time. Attempts to Andrzej Czarnecki, MD, PhD, DSc scrutinize the add-on benefit of a drug are Editor-in-Chief EDITOR-IN-CHIEF already in place, be it on national level (e.g., UK, Australia, Canada) or a local level (e.g., Richard Chamberlain, PhD hospital formularies). Therapeutic value is ECS, Inc. a very fair question; specifically for the Ronald D. Fitzmartin, PhD, MBA individual patient that wants to be healthy Managing Partner again, and even more so if he or she has Decision Analytics, LLC already been treated with other available drugs, with limited or no success. Alberto Grignolo, PhD PAREXEL From the provider’s perspective, everyone is aware that there were never Betty R. Kuhnert, PhD, MBA unlimited funds to cover treatment PharmaNet costs. Therefore, it seems that debating comparative effectiveness research (CER) Sarah Powell and putting existing and developing new Thomson Reuters methodologies into practice for assessment of the therapeutic and cost value of Nancy D. Smith, PhD Potomac, MD ANDRZEJ CZARNECKI ANDRZEJ CZARNECKI treatments to support decision-making is the right thing to do. Jean H. Soul-Lawton, DPhil GlaxoSmithKline CER should be distinguished from cost effectiveness. It is important therefore to J. Rick Turner, PhD factor in two other aspects of the issue Quintiles OPEN FORUM that should not be forgotten in the drive towards increased effectiveness in the Qingshan Zheng, PhD environment of limited resources – the Shanghai University of individual patient and the public health. Chinese Medicine, China Any practicing physician knows from experience that patients respond differently to different drugs, including those 1 OF-Open Forum.indd 1 9/19/11 11:04 AM from the same pharmacological individual patient. The overall also raise concerns that innovation class. Comparative effectiveness impact of drug costs in a health in medicine can be affected. If is assessed on larger groups of budget is large, but not as substantial this were the case, careful thought patients (public health perspective), as we frequently are persuaded to ought to be given to such a potential sometimes larger than the one think. More specifically, drug costs impact. in which efficacy and safety has account for about 10% of health been established in pivotal clinical care spending, so a substantial These and related points are the trials. We may, therefore, face two 10% savings on drug costs would focus topic of this issue. We also concepts that are not necessarily amount to only a 1% saving in present further developments in aligned – a lack of superiority spending on the health care budget. health technology assessment/ of a tested treatment but, at the Despite the size of these budgets, CER and some practical aspects same time, a proven efficacy in the one cannot regard the 1% overall of methodologies used for these tested indication. Such treatment saving as great on a percentage basis studies. With a growing interest in should, without any doubt, find its unless it is truly justified. Looking the area, additionally stimulated by way to an individual patient as an from this perspective, therefore, substantial federal funds allocated alternative, as personalized care it is effectiveness and not cost by the US government, it is worth a is one of the outcomes of CER. effectiveness that should drive the closer look at these developments. Subsequently, if the product is CER studies. clearly inferior to others being used, In the “Best Practices” section, both the physician and the patient We should do all the necessary work there are several interesting articles; will recognize its true value and the to establish “real life” effectiveness, however, I would like to draw market will “regulate” itself. The and not concentrate excessively on special attention to the information landscape of current approaches to the cost outcomes of CER that may provided by “Nanotechnology, medicines is briefly set out in one achieve a relatively small overall Nanomedicine, Implications for of our articles (“CER: International cut in spending. This is not to say Drug Safety, Pharmacovigilance Developments and Impact”) when that CER and the understanding and Risk Management.” We do not comparing definitions of Evidence- of the cost should not be a top frequently get the opportunity to based Medicine, CER and Patients- priority. By all means, physicians learn something different about centered Outcomes Research. and patients have a great interest a topic that is perceived as an in knowing which of the available important step to the future. I would Increasing populations and products delivers the best health like to extend my thanks to Dr. budgetary constraints pave the way value for them and allows for savings Sougato Das for serving as section for the development of CER, which that should ultimately translate chair for this special topic and his should serve public health purposes into better health service overall. excellent work on the CER section of well. But, it should also serve the Implementing CER/HTA widely may this issue. ■ OPEN FORUM 2 GLOBAL FORUM OCTOBER 2011 OF-Open Forum.indd 2 9/19/11 11:04 AM OCTOBER 2011, VOL 3 ISSUE 5 GLOBAL FORUM CER & HTA: NEW DAYS FOR DIA his issue presents a special focus on CER/HTA related initiatives from an on the issue of Comparative international array of pertinent organizations, T Effectiveness Research and Health such as Health Technology Assessments TechnologyTechnolo Assessment. International (HTAI), the European Federation of Pharmaceutical Industries & Associations Current discussion around CER, as it is known (EFPIA), the National Institute for Health & in the US, and HTA, its European counterpart, Clinical Excellence (NICE, UK), ARCS Australia can sometimes bring to mind George Bernard Ltd. (previously the Australian Association Shaw’s famous quote that, “England and of Regulatory and Clinical Scientists), the America are two countries separated by a International Society for Pharmacoepidemioloy common language.” CER and HTA employ (ISPE), the US FDA, and others. different terms, but their concepts are very similar and their goals are close: To identify and These conversations illustrate how DIA can evaluate objective evidence to determine the be a key player, because these conversations optimum health care treatment options, at both are precisely what DIA does best: Create the patient and population levels, at the best opportunities for stakeholders from the YVES JUILLET cost. Increasingly, regulatory approval no longer different sides of an issue – in this case, means simultaneous, instant market assess; patients, researchers, clinicians, regulators, CER and HTA assessments are becoming new payers, and legislators – to share their gatekeepers responsible for recommendations knowledge, clarify concepts, and facilitate that public and/or private payers fund the cost common terminology, for us to take back and of new health technologies after regulatory use in our respective workplaces to benefit approval. the world’s health. We specifically identified the need for more collaboration between the At the EuroMeeting in Geneva this past March, different groups engaged in this activity, and and the DIA 2011 Annual Meeting in Chicago to facilitate the bridge between the regulatory this past June, DIA convened two special and the HTA perspectives, particularly at two executive consultation forums on CER/HTA. specific points in the life of a product. Discussions at these forums centered on the concepts of CER and HTA in both regions, The first point is during product development. PRESIDENT’S MESSAGE their practical implications on drug and device In the US, the applicant and Agency interact development, and, just as importantly, where during the IND/NDA process; in Europe, and how DIA can advance these discussions the applicant and Agency interact through from theoretical scenarios to practical actions voluntary Scientific Advice meetings. This early and operations for the various stakeholders and stage in both processes is also an opportunity our