Conference Day One Thursday, April 23rd 2015 – Main Conference Room
8.55 Chairperson’s Opening Remarks Hans Schikan, Former Chief Executive Officer, Prosensa
9.00 Keynote: Data driving diagnostics - Clinically relevant information from ordinary photos, and an algorithm to aid in diagnoses of rare diseases Christoffer Nellaker, Research Fellow, Medical Research Foundation’s Functional Genomics Unit, Oxford University
9.30 Keynote: Is the market sustainable? Henri Termeer, Former Chairman, President and CEO, Genzyme (on video)
10.00 Speed Networking & Networking coffee break
11.00 Good medicine is good business: Transforming lives and meeting unmet needs Stephanie Okey, Senior Vice President and Head of North America, Genetic Diseases and US General Manager, Genzyme
11.20 The Great Debate: “The Price is Too Damn High!”…or is it? FOR: The orphan population is so small the rest of the population can more than cover the cost Ram Selvaraju, Managing Director, Equity Research – Healthcare, MLV & Co. AGAINST: First there was rare, now there’s ultra-rare. And it seems every other disease is rare and every other drug orphan. WE won’t be able to afford it – period. Ed Pazalla, VP, National Medical Director for Pharmacy Policy and Strategy, Aetna
12.00 Networking Lunch
1:00 Lunch session: Pediatric vouchers to expedite rare disease treatments in children Gayatri Rao, Director, Office of Orphan Product Development, FDA
1.30 2 x 40-minute roundtables with a 10 minute break in-between. Please sign up in the exhibition hall lounge. 1: How does miscommunication or incomplete communication about orphan drug pricing rationale affect the future of Orphan Programs? Kristine Dorward, Director, Marketing & Business Development, ProMetic
2: Effective engagement with the financial community for orphan drug stakeholders Celia Economides, Director, Patient Advocacy, Biomarin
3: Early access to orphan drugs – more practical consideration Tom Watson, Business Development Director, Clinigen Global Access Programs (GAP)
4: Strategies and collaborations to accelerate rare disease clinical drug development Scott Schliebner, Vice President of Scientific Affairs, Rare Diseases, Federal Work, PRA Health Sciences
5: How to develop a registry for successfully meeting multi-stakeholder evidence requirements Eric Gemmen, Senior Director, Scientific Affairs, Biostatistics & Outcomes Research, Quintiles
6: Helping patients afford the high cost of orphan drugs Gerald Lauria, Director of Business Development, The Assistance Fund
7: How to gain more control of your products success: key strategies for patient access and distribution planning Jan Nielsen, Division President, Access & Patient Support, Sonexus Health
8: The Executive, Legislative and Judicial powers and physicians: ensuring rights for rare diseases in Brazil Paolo Tagliaferri, Consultant, M3 Pharma
9: How to take orphan drugs from manufacturer to patient – commercialization strategy Denise Von Dohren, Vice President, Specialty Care Group, Omnicare
10: Biopharma manufacturer strategies: integrated vs. non-integrated program models Mark Vineis, Vice President, Specialty Pharmacy, OncoSource Rx, Cardinal Health
11: Gathering patient insights in rare diseases
Malcolm Mackenzie, SVP, Strategic Planning/New Business Development, Juice Pharma Worldwide
3.00 Networking coffee break
3.40 Gaining regulatory special protocol assessment (aka expedited review) approvals or agreements for Phase 3 orphan drug development programs Emily Stube, Director, Medical Writing Product Development and Client Relations, Premier Research
4.00 Launching the 1st therapy for DMD – a 16-year journey! Mark Rothera, Chief Commercial Officer, PTC Therapeutics
4.20 Reality TV: Grassroots method of achieving expanded disease awareness and commercial success without a patient group Kate Holland, VP, Commercial, Vanda Pharmaceuticals
4.40 1 x 40-minute roundtable. Please sign up in the exhibition hall lounge. 1: Endpoint validation and clinical trial design: overcoming issues and challenges Tuyen Ong, Senior Vice President, Head of Clinical Development and Translational Research, PTC Therapeutics
2: Tailored biologic development programs for orphan drugs Rekha Patel, Director, Large Molecule Analytical Chemistry, Catalent Pharma Solutions Rob Guistines, Director, Biologics, Catalent Pharma Solutions
3: Pharmacovigilance for orphan drug companies – what are the challenges? Joerg Seebeck, Chief Medical Officer, PrimeVigilance
4: How might a smartphone app (and other new technology applications) advance patient outcomes in rare diseases? Greg Gorgas, Senior Vice President, Commercial, Mast Therapeutics
5: Optimizing patient engagement with digital strategies and tools May Orfali, Senior Director at Global Innovative Pharma Business Unit, Pfizer Mary Ellen Carroll, Director, Global Commercial Development, Rare Diseases, Pfizer
6: When is the right time to on-board external expertise in orphan drug development? Thomas Ogorka, President and Chief Executive Officer, Orphan Reach
7: The NIH/NCATS GRDRSM Program (Global Rare Diseases Patient Registry Data Repository) - A model to accelerate drugs and therapeutics for rare diseases Yaffa Rubenstein, Director of Patient Resources for Clinical and Translational Research, Office of Rare Disease Research, NIH
8: Is there a case for open-label extensions to early development studies? – Balancing the need for early access to treatments for rare disorders and protecting patients’ safety and the viability of promising novel treatments Leone Atkinson, Senior Medical Director, Covance Neuroscience
9: ‘R’ you collaborating? The 5-R approach to real-world collaboration in rare disease Thomas Gegeny, Stakeholder Engagement Leader, Clear Pharma
10: How to run successful rare disease studies Angi Robinson, Executive Director, Pediatrics and Rare Diseases, Premier Research
11: A 2020 vision: Orphan drugs in 5 years’ time Nigel Livessy, Consultant, Comradis Jonathan Morton, Consultant, Comradis
12: Demystifying managed access for orphan drugs: obstacles and opportunities Keith Watts, Executive Vice President Strategic Services, One-World, Inc.
5.20 Chairperson’s recap of the day
5.25 Roaring ‘20s-themed Cocktail Party sponsored by 7.00 Gala Dinner* sponsored by: This requires separate registration.
Conference Day Two Friday, April 24th 2015 – Main Conference Room
8.55 Chairperson’s opening remarks Kristine Dorward, Director, Marketing & Business Development, ProMetic
9.00 Keynote: Building the cures of the future – FDA’s programs to boost orphan drug development Richard Moscicki, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, FDA
9.30 Keynote: RNA as an orphan drug: turning genes on or off, and correcting them to cure genetic disorders Daniel Anderson, Scientific Founder, CRISPR Therapeutics
10.00 Morning Refreshments P&R AND MARKET ACCESS
10.40 Access: the key to successful European commercialisation – rising to the challenge Wills Hughes-Wilson, Senior Vice President, Chief Patient Access Officer, SOBI
11.00 Early access to orphan drugs – the rationale, challenges and options available Mark Corbett, Senior Vice President, Clinigen Global Access Programs (GAP)
11.20 Perceiving value and sustainability in the orphan drug market through the eyes of the payers Julie Stoss, Vice President of Government Relations, Kaiser Permanente
11.40 Payers’ Panel: Not against progress but against expensive drugs – understanding the payers’ perspective Ed Pezalla, VP, National Medical Director for Pharmacy Policy and Strategy, Aetna Julie Stoss, Vice President of Government Relations, Kaiser Permanente Jeff Myers, President and Chief Executive Officer, Medicaid Health Plans of America (MHPA)
12.20 Networking Lunch
1.50 1 x 40-minute roundtable. Please sign up in the exhibition hall lounge.
1: Developing a high-touch rare disease business model for companies bringing their first orphan drug to market Eric Pauwels, Senior Vice President and General Manager, Americas, PTC Therapeutics
2: Co-development model in orphan drug development Miroslav Reljanović, CEO, Ergomed PLC
3: Leveraging integrated offerings for drug products with orphan and or expedited designations Matthew Mollan, General Manager, Development & Analytical Solutions, Catalent
4: Can you make a sustainable biotech out of orphan development? Mark Varney, Chief Executive Officer, Neurolixis
5: Rare disease medicines: the complexities of healthcare, innovation and societal value Elizabeth White, Assistant Vice President, Rare Disease Commercial Development, Global Innovative Pharma Business, Pfizer Danielle Rollmann, Head, International Policy within Pfizer’s Corporate Affairs, Pfizer
6: What to do if patient recruitment in orphan trials falls behind? Peggy Schorr, VP, US Operations, Orphan Reach
7: Obtaining multiple orphan designations, and the commercial benefits Dr. David Drutz, Chief Medical Officer, DARA Biosciences
8: Advance the use of regulatory science to aid clinical trial design and performance for PRD Nach Dave, Director, Regulatory Affairs, Premier Research
9: Successful strategies for patient registries that meet the needs of Pharma, research and advocacy communities Kyle Brown, Founder & CEO, PatientCrossroads
10: The value of Named Patient Programs (NPP): market insights from ethical early access to innovative products Wendi LeVigne, Vice President Manufacturer Relations, One-World, Inc.
GLOBAL ACCESS
2:30 From clinical development to market access: the MENA region opportunity Alaa Assem, President & CEO, Clinart MENA
2:50 Brazilian scenario for rare diseases: Interfarma’s and ANVISA’S initiatives Renato Porto, Director of the National Agency for Sanitary Surveillance, ANVISA Maria Jose Delgado Fagundes, Director, Interfarma
3:10 Speeding up orphan drugs to patients by harmonizing global regulatory and market access Chander Sehgal, Director, Common Drug Review (CDR) and Optimal Use, CADTH
3.40 Networking coffee break
4.10 International Markets: 1 x 40-minute roundtable. Please sign up in the exhibition hall lounge.
1: Indian Organization for Rare Diseases and launching orphan treatments in India Ramaiah Muthyala, Associate Director, Center for Orphan Drug Development, University of Minnesota
2: Canada’s new patient involvement pilot as a model, and the Canadian orphan drug framework Chander Seghal, Director, Common Drug Review (CDR) and Optimal Use of Drugs, CADTH
3: The role of patient organizations in setting the public agenda in behalf of people with rare diseases Renato Porto, Director, ANVISA Antonie Daher, President, Casa Hunter Maria José Delgado Fagundes, Director, Interfarma
4: Local market experience of rewards & challenges in Korea Keya Chiang, Corporate Sales Director, Giddi Pharma
5: Prioritizing Taiwan for launch: low hanging fruit or tough cookie? Ben Tsai, VP of Business Development, Giddi Pharma
4.50 Chairperson’s closing remarks
4.55 End of Conference