Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary (2015)

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B Workshop Agenda Financial Incentives to Support Unmet Medical Needs for Nervous System Disorders: A Workshop January 20–21, 2015 Institute of Medicine Keck Center 500 Fifth Street, NW Room 100 Washington, DC 20001 Background: The global burden of nervous system disorders is projected to signifi- cantly increase over time and is estimated to cost society more than $6 trillion per year by 2030 (World Economic Forum and Harvard School of Public Health, 2011). Although there have been recent international initia- tives to better understand the human brain in order to develop new therapeu- tics, several large pharmaceutical companies have decreased investment or even withdrawn from their neuroscience research programs. The perceived high risk and low probability of success has made the neuroscience sector less attractive than other therapeutics areas for research and development (R&D), despite the large market potential. As a result, patients are often left with few if any options for treatment and thus there is a need to con- sider policy options to increase private-sector investment in R&D for nervous system disorders. With this context this public workshop will explore opportunities to foster private-sector innovation by supporting 65 

66 FINANCIAL INCENTIVES new investments directed toward the development of novel therapeutics to meet unmet needs for nervous system disorders. Meeting Objectives: The workshop will bring together key stakeholders to explore opportuni- ties to increase private-sector investments directed toward the develop- ment of novel therapeutics to meet unmet needs for nervous system disorders. Presentations and discussions will be designed to: • Examine opportunities and barriers to increasing investments for the development of novel therapeutics to support unmet medical needs for nervous system disorders. o Discuss specific considerations for combination therapies and disease-modifying treatments that may require extensive long-term prevention trials. • Explore potential incentives that might lead to a significant rein- vestment in R&D within the neuroscience sector, while consider- ing the resources needed for implementation. For example, o Discuss regulatory changes that may help decrease the time it takes for a new central nervous system (CNS) drug to be approved. o Consider the impact of potential policy changes on patients. SESSION I: OVERVIEW AND BACKGROUND Session Objectives: • Introduce the workshop objectives. • Examine the current unmet medical needs for nervous system disorders. • Provide a context for the current level of investment that CNS gets in comparison with other therapeutic areas. 

APPENDIX B 67 January 20, 2015 8:30 a.m. Welcome and Workshop Objectives DENNIS CHOI, Workshop Co-Chair Professor and Chair, Department of Neurology, School of Medicine Director, Neurosciences Institute Stony Brook University TIMOTHY COETZEE, Workshop Co-Chair Chief Advocacy, Services, and Research Officer National Multiple Sclerosis Society 8:45 a.m. Overview of Unmet Medical Needs for Nervous System Disorders STEVEN HYMAN Professor of Stem Cell and Regenerative Biology Director, Stanley Center for Psychiatric Research Broad Institute, MIT and Harvard University 9:15 a.m. Policy-Based “Pull” Incentives for Creating Break- through CNS Drugs: Background Neuron Paper DENNIS CHOI, Workshop Co-Chair Professor and Chair, Department of Neurology, School of Medicine Director, Neurosciences Institute Stony Brook University 9:45 a.m. CNS Incentives in the Context of Other Therapeutic Areas DAVID MEEKER President and Chief Executive Officer Genzyme, A Sanofi Company 10:05 a.m. Discussion with Speakers and Participants Moderators: Dennis Choi and Timothy Coetzee 

68 FINANCIAL INCENTIVES 10:30 a.m. BREAK SESSION II: MARKET PROTECTIONS Session Objectives: • Consider the impact that increased intellectual property (IP) pro- tections, including both enhanced data package protection and longer patent life, might have on private-sector investment in R&D for CNS disorders. • Discuss the duration for enhanced IP protection that would be necessary to attract increased investment in the large-market CNS space. • Examine the specific potential benefits and other impacts that enhanced IP protection could have on those with or at risk for CNS disorders. 10:45 a.m. Overview of Current Intellectual Property Protections: Patents and Data Package Protection ROBERT ARMITAGE, Session Chair IP Strategy and Policy Consultant Former Senior Vice President & General Counsel, Eli Lilly and Company 11:05 a.m. Panel Discussion: How Might New Market Protections Impact R&D Investment Decisions? Moderator: Robert Armitage Discussion Questions: • How do IP and technical issues interrelate to de- cide where both short- and long-term decision making affect allocation of resources? • What are the current IP and market protections and why are they not working to incentivize CNS investments? • What factors or policies might increase equity investments into this sector? 

APPENDIX B 69 Panelists: • Bonnie Weiss McLeod, Partner, Cooley, LLP • Steven Paul, Chief Executive Officer and Board Member, Voyager Therapeutics; Weill Cornell Medical College • Arti Rai, Professor of Law and Co-Director, Duke Law Center for Innovation Policy • Kiran Reddy, Senior Director, Corporate Strate- gy, Biogen Idec 11:45 a.m. Discussion with Panelists and Workshop Participants 12:15 p.m. LUNCH 12:45 p.m. Panel Discussion: Potential Policy Pathways and Their Implications Moderator: Ben Roin, Assistant Professor, MIT Sloan School of Management Discussion Questions: • What can be learned from other efforts to in- crease market exclusivity (e.g., Orphan Drug Act, MODDERN Cures Act, GAIN Act, and the Biosimilars Act)? Have they been successful? • Is there a role for orphan drug-like registration exclusivity, priority review vouchers, or similar policies? • What are the comparative benefits and potential drawbacks of enhancing patent protection versus greater data package protection as they relate to the CNS space? • Should industry be expected to provide “give backs” in return for enhanced IP incentives and, if so, what might be appropriate (e.g., data shar- ing, publication of negative data)? Panelists: • Marc Boutin, Executive Vice President and Chief Operating Officer, National Health Council 

70 FINANCIAL INCENTIVES • Alfred B. Engelberg, Trustee, The Engelberg Foundation • William (Terry) Fisher, Wilmer Hale Professor of Intellectual Property Law, Faculty Director, Berkman Center for Internet and Society, Harvard Law School • Nicholas Manetto, Director, FaegreBD Consulting 1:15 p.m. Discussion with Panelists and Workshop Participants 1:45 p.m. Response Panel and Discussion with Participants Moderator: Robert Armitage Discussion Question: • What IP-related incentives would make a real and substantial difference in how biopharma en- terprises evaluate potential investments in CNS? Panelists: • Marc Boutin, Executive Vice President and Chief Operating Officer, National Health Council • Alfred B. Engelberg, Trustee, The Engelberg Foundation • William (Terry) Fisher, Wilmer Hale Professor of Intellectual Property Law, Faculty Director, Berkman Center for Internet and Society, Harvard Law School • Nicholas Manetto, Director, FaegreBD Consulting • Bonnie Weiss McLeod, Partner, Cooley, LLP • Steven Paul, Chief Executive Officer and Board Member, Voyager Therapeutics; Weill Cornell Medical College • Arti Rai, Professor of Law and Co-Director, Duke Law Center for Innovation Policy • Kiran Reddy, Senior Director, Corporate Strategy, Biogen Idec 2:45 p.m. BREAK 

APPENDIX B 71 SESSION III: INCENTIVES THROUGH INNOVATIVE REGULATORY PATHWAYS Session Objectives: • Discuss opportunities to incentivize CNS R&D by using existing Food and Drug Administration authorities or through new regula- tory mechanisms. • Explore innovative applications of existing clinical development regulatory pathways and how they may be adopted for CNS drugs to decrease the length of clinical trials and the time it takes for a new drug to be approved. • Consider the risks, benefits, and trade-offs of establishing accel- erated and conditional approval pathways. 3:00 p.m. Session Overview JANET WOODCOCK, Session Chair Director, Center for Drug Evaluation and Research Food and Drug Administration 3:10 p.m. The Promise and Pitfalls of Changing Regulatory Standards to Spur CNS Drug Discovery AARON KESSELHEIM Associate Professor of Medicine Harvard Medical School and Brigham and Women’s Hospital 3:25 p.m. Six Opportunities for Improving Pathways to Market: A Global Perspective RAJ LONG Senior Regulatory Officer-Integrated Development, Global Health The Bill & Melinda Gates Foundation 3:40 p.m. Panel Discussion: New or Existing Regulatory Approval Pathways Moderator: Janet Woodcock Discussion Topics: 

72 FINANCIAL INCENTIVES • Discuss whether and how existing regulatory pathways can be used by CNS drug developers. • Discuss new or modified accelerated approval pathways to facilitate CNS drug development and how these innovations might alter risk and other ethical considerations. • Explore innovations in clinical trials that could help reduce time, cost, and risk to expedite pathway to market. Panelists: • Jeff Allen, Executive Director, Friends of Cancer Research • Lauren Chiarello, Senior Director, Federal Government Relations at National Multiple Sclerosis Society • Jeffrey Jonas, Chief Executive Officer, SAGE Therapeutics • Aaron Kesselheim, Harvard Medical School and Brigham and Women’s Hospital • Alex London, Professor of Philosophy and Director, The Center for Ethics and Policy, Carnegie Mellon University • Raj Long, The Bill & Melinda Gates Foundation 4:20 p.m. Discussion with Panelists and Workshop Participants 5:00 p.m. Adjourn Day 1 

APPENDIX B 73 January 21, 2015 SESSION IV: IMPACT OF FINANCIAL INNOVATION ON THE PATIENTS Session Objectives: • Identify issues that will need to be addressed in further depth re- lated to how proposed incentives could potentially impact patient access to new treatments. • Consider how innovation-friendly reimbursement and payment policies can ensure patient access to new medicines. • Examine how the costs associated with increased financial incen- tives, including longer IP protection or data exclusivity, would impact patient access to innovative and generic medicines. • Consider how access to new medicines may impact overall health care costs and other potential economic benefits. 8:30 a.m. Session Overview GEORGE VRADENBURG, Session Chair Chairman, Founding Board Member USAgainstAlzheimer’s 8:40 a.m. Potential Impact of New Treatments on Health Care Costs Defining Value for Innovative Therapeutics to Meet Unmet Medical Needs for Nervous System Disorders ROGER LONGMAN Chief Executive Officer Real Endpoints Economic Cost and Impact of Nervous System Disorder Prevention and Treatment Strategies ADELINA COMAS-HERRERA Research Fellow London School of Economics and Political Science 9:10 a.m. Balancing Access, Value, and CNS Drug Risks: 

74 FINANCIAL INCENTIVES Societal Impact Tension and Trade-Offs for Incentivizing Innovative Therapeutics PETER UBEL Professor of Business, Public Policy and Medicine Duke University Value and Costs of Innovative Therapies to Patients GAIL MADERIS President and Chief Executive Officer BayBio Practical Considerations with the Implementation of Innovative Medicines into Generally Accepted Practice That Is Reimbursable RHONDA ROBINSON BEALE Senior Vice President and Chief Medical Officer Blue Cross of Idaho 9:55 a.m. Discussion with Attendees 10:30 a.m. BREAK SESSION V: MEETING RECAP AND OPPORTUNITIES FOR IMPACTING CHANGES TO U.S. POLICY Session Objectives: • Recap the key themes presented and discussed during each session. • Consider how the ideas discussed at the workshop can be im- plemented into U.S. policy. • Discuss the role of each stakeholder (patients, academic societies, and the private sector) in helping to implement potential policy changes to incentivize CNS drug discovery and development. Session Chairs: Dennis Choi and Timothy Coetzee 

APPENDIX B 75 10:45 a.m. Mobilizing a Path Forward: Translating Ideas into Policy HONORABLE PATRICK KENNEDY Co-Founder, One Mind The Kennedy Forum 11:00 a.m. Discussion with Workshop Participants 11:15 a.m. Session Chairs II-IV: Presentation of Key Themes • Presentation by session chairs on key themes pre- sented and discussed. • What actions are needed to advance CNS drug dis- covery and development at a policy level? ROBERT ARMITAGE IP Strategy and Policy Consultant Former Senior Vice President & General Counsel, Eli Lilly and Company JANET WOODCOCK Director, Center for Drug Evaluation and Research Food and Drug Administration GEORGE VRADENBURG Chairman, Founding Board Member USAgainstAlzheimer’s 11:55 a.m. Discussion with Workshop Participants 12:30 p.m. LUNCH 1:00 p.m. Next Step Panels Discussion Questions: • Who else needs to be brought into the conversation? • What are practical steps individual groups can follow to advance the dialogue? 

76 FINANCIAL INCENTIVES • What are sector-specific challenges and oppor- tunities to advance policy? 1:00 p.m. Next Steps: The Potential Role of Academic Societies to Advance Policy-Based Incentives for CNS Drug Discovery and Development Moderator: Walter Koroshetz, Acting Director, National Institute of Neurological Disorders and Stroke Panelists: • William Z. Potter, American College of Neuropsy- chopharmacology, National Institute of Mental Health • Michael Rogawski, President, American Society for Experimental NeuroTherapeutics; University of California, Davis • Edward F. Rover, Chairman and President, Dana Alliance for Brain Initiatives; Charles A. Dana Foundation • Katie Sale, Executive Director, American Brain Coalition • Paul Summergrad, President, American Psychiatric Association; Tufts University School of Medicine 2:00 p.m. Next Steps: The Potential Role of Patient or Disease Advocacy Groups to Advance Policy-Based Incen- tives for CNS Drug Discovery and Development Moderator: Margaret Anderson, Executive Director, FasterCures Panelists: • Brian Fiske, Vice President, Research Programs, The Michael J. Fox Foundation for Parkinson’s Research • Stephen Johnson, Chief Policy Officer, One Mind • Robert Ring, Chief Science Officer, Autism Speaks • Andrew Sperling, Director of Federal Legislative Advocacy, National Alliance on Mental Illness 

APPENDIX B 77 • William H. Thies, Senior Scientist in Residence, Medical and Scientific Relations, Alzheimer’s Association • George Vradenburg, Chair, Founding Board Member, USAgainstAlzheimer’s 3:00 p.m. Next Steps: The Potential Role of the Private Sector (Industry and Foundations) to Advance Policy-Based Incentives for CNS Drug Discovery and Development Moderator: Bernard H. Munos, Founder, InnoThink Center for Research in Biomedical Innovation; Faster- Cures Panelists: • Cartier Esham, Executive Vice President, Emerging Companies, Biotechnology Industry Organization • Bruce Kinon, U.S. Therapeutic Head, Psychosis, Lundbeck LLC, USA • Michele M. Oshman, Director, Federal Alliance De- velopment, Corporate Affairs, Eli Lilly and Company • Maike Stenull, Senior Director, Strategic Projects and Transformational Leadership, Office of the Chief Medical Officer, Johnson & Johnson • David Wholley, Director, Research Partnerships, Foundation for the National Institutes of Health 4:00 p.m. Discussion with Workshop Participants 4:30 p.m. Closing Remarks DENNIS CHOI, Workshop Co-Chair Professor and Chair, Department of Neurology, School of Medicine Director, Neurosciences Institute Stony Brook University TIMOTHY COETZEE, Workshop Co-Chair Chief Advocacy, Services, and Research Officer National Multiple Sclerosis Society 4:45 p.m. ADJOURN