The Proceedings of the World Federation of Hemophilia’s Eighth Global Forum on the Safety and Supply of Treatment for Bleeding Disorders © World Federation of Hemophilia, 2013 The WFH encourages redistribution of its publications for educational purposes by not-for-profit hemophilia organizations. For permission to reproduce or translate this document, please contact the Education and Public Policy Department at the address below. This publication is accessible from the World Federation of Hemophilia’s Web site at www.wfh.org. Please note: This material is intended for general information only. The World Federation of Hemophilia does not endorse particular treatment products or manufacturers; any reference to a product name is not an endorsement by the WFH. The WFH is not a regulatory agency and cannot make recommendations relating to safety of manufacturing of specific blood products. For recommendations of a particular product, the regulatory authority in a particular country must make these judgments based on domestic legislation, national health policies, and clinical best practices. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. Any treatment must be designed according to the needs of the individual and the resources available. Published by the World Federation of Hemophilia World Federation of Hemophilia 1425 René Lévesque Boulevard West, Suite 1010 Montreal, Quebec H3G 1T7 CANADA Tel.: (514) 875-7944 Fax: (514) 875-8916 E-mail: [email protected] Internet: www.wfh.org Acknowledgements The World Federation of Hemophilia gratefully acknowledges the financial contribution of Baxter, the Foundation for America’s Blood Centers, Héma-Québec, Ministère de la Santé et des Services sociaux du Québec (Québec Ministry of Health and Social Services), Novo Nordisk, Pfizer, and Swedish Orphan Biovitrum (SOBI) towards the 2013 Global Forum. Only through such support and partnerships can the WFH continue to provide important global interchange on issues related to blood product safety and supply. 8TH WFH GLOBAL FORUM ON THE SAFETY AND SUPPLY OF TREATMENT PRODUCTS FOR BLEEDING DISORDERS Montreal, Canada September 26-27, 2013 PROCEEDINGS The World Federation of Hemophilia gratefully acknowledges the sponsors of the 2013 WFH Global Forum: Baxter Foundation for America’s Blood Centers Héma-Québec Ministère de la Santé et des Services sociaux du Québec (Quebec Ministry of Health and Social Services) Novo Nordisk Pfizer Swedish Orphan Biovitrum (SOBI) TABLE OF CONTENTS Executive Summary ........................................................................................................................................... 1 Day 1: September 26, 2013 ................................................................................................................................. 7 Welcome and Opening Remarks ..................................................................................................................... 7 Session 1: The Safety and Sustainability of Plasma Products ..................................................................... 8 Safety and Supply – A WFH Focus ................................................................................................................. 9 New Technologies For Virus Testing: Where Donor Screening Is Going ............................................... 10 Transmission of B19 and Hepatitis E by Plasma Products ........................................................................ 12 Inactivation of Non-Enveloped Viruses such as Parvovirus B19 and Hepatitis E Virus ...................... 16 Project Recovery ............................................................................................................................................... 19 Point/Counterpoint: The Future of Plasma vs. Recombinant .................................................................. 24 Future of Plasma Fractionation: A Pessimistic View .................................................................................. 24 The Global Plasma Industry: An Optimistic Future ................................................................................... 27 Session 2: Clinical and Economic Aspects of Novel Treatment Products ............................................... 31 Is the Annual Bleed Rate Enough? The Micropathology of Joints ........................................................... 32 Changing Treatment Paradigms – What Should Personalized Care Look Like? .................................. 36 Economic Considerations: Pricing, Sustainability, Access, Increased Supply ....................................... 39 Rise of the Patient Voice: Patient Perspectives on Clinical and Economic Aspects of Novel Products .. 43 Day 2: September 27, 2013 ............................................................................................................................... 47 Session 3: Product Choice – What Does It Mean? ....................................................................................... 47 Panel Discussion: What Do We Mean By “Product Choice”? ................................................................... 48 Session 4: The Impact of Regulatory Factors on Product Access .............................................................. 55 Orphan Designation and Market Exclusivity .............................................................................................. 55 Market Exclusivity for Hemophilia Products: The New Extended Half-Life Factors .......................... 58 Interchangeability of Coagulations Proteins: The Treaters Perspective .................................................. 60 Session 5: Manufacturer Updates .................................................................................................................. 63 Development of Recombinant FVIII Fc Fusion Protein and Recombinant FIX Fc Fusion Protein ..... 63 Baxter Healthcare – Innovation in Hemophilia? ......................................................................................... 66 Manufacturing Process for the New Recombinant Factor VIII Turoctocog Alfa ................................... 67 CSL Behring: Committed to the Bleeding Disorders Community ........................................................... 69 Closing Remarks ............................................................................................................................................... 71 NOTES EXECUTIVE SUMMARY Introduction The 8th WFH Global Forum on the Safety and Supply of Treatment Products for Bleeding Disorders, held September 26-27, 2013, in Montreal, Canada, focused on current issues and developments related to the safety and sustainability of plasma products, clinical and economic aspects of novel treatment products for hemophilia and other inherited bleeding disorders, product choice, and the impact of regulatory factors on product access. More than 120 people from around the world attended this Global Forum, including leading hematologists and clinicians, multidisciplinary health professionals, patient organization representatives, blood services officials, and industry experts. The objectives were to present the latest knowledge and encourage discussion, consensus building, and networking towards addressing global challenges related to treatment safety, supply, and access. The meeting also highlighted the ever-present issue of access to safe treatment in developing countries. “About 75% of people in the world who have bleeding disorders are not diagnosed and are without access to proper care,” noted John Bournas, WFH CEO and Executive Director. At this Global Forum, the WFH officially launched Project Recovery, a groundbreaking initiative in partnership with Canadian Blood Services and plasma product manufacturers Biotest AG and Grifols, which will turn unused blood components from Canadian blood donations into medicines for people with hemophilia in developing countries with little or no access to life- and limb-saving treatment. In addition, the WFH’s Close the Gap Campaign is underway to raise funds for the development of sustainable care in areas of the world with the greatest need. To inspire support, WFH patron Jan Willem André de la Porte committed to match and double all contributions made during this Global Forum. The global bleeding disorders community is on the cusp of great change which will revolutionize treatment and care. New treatment products are about to enter the market as soon as early 2014, and will change the landscape and management of care for people with inherited bleeding disorders. Longer-acting factor concentrates based on new strategies such as pegylation, fusion technologies, and amino acid modification will offer substantially enhanced efficacy and extended half-life. This 8th WFH Global Forum explored the implications of the new therapies in terms of treatment, clinical practice, product choice, and product access, and regulatory requirements and challenges related to orphan drug designation, market exclusivity, and marketing authorization of biosimilars. The Safety and Sustainability of Plasma Products David Page of the WFH Treatment Product Safety, Supply and Access Committee opened the first session with a retrospective overview of the WFH Global Forum. First held in Montreal in 2000, the Global Forum has become an important meeting for scientific exchange and international dialogue on important issues related to treatment, blood safety, supply, and