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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

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 significantly 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 initiatives to better understand the human brain in order to develop new therapeutics, several large pharmaceutical companies have decreased investment or even withdrawn from their neuroscience research programs.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 consider 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

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

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 opportunities to increase private-sector investments directed toward the development 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.
- 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 reinvestment in R&D within the neuroscience sector, while considering the resources needed for implementation. For example,
- Discuss regulatory changes that may help decrease the time it takes for a new central nervous system (CNS) drug to be approved.
- 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.

Page 67 Cite

Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	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 Breakthrough 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

Page 68 Cite

Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

10:30 a.m.	BREAK

SESSION II: MARKET PROTECTIONS

Session Objectives:

Consider the impact that increased intellectual property (IP) protections, 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 decide 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?

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	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 Strategy, 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 increase 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 sharing, publication of negative data)?
	Panelists: Marc Boutin, Executive Vice President and Chief Operating Officer, National Health Council

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	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 enterprises 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

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

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 regulatory 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 accelerated 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:

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	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

Page 73 Cite

Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

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 related 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 incentives, 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:

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	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 implemented 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

Page 75 Cite

Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

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 presented and discussed. What actions are needed to advance CNS drug discovery 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?

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Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	What are sector-specific challenges and opportunities 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 Neuropsychopharmacology, 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 Incentives 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

Page 77 Cite

Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.

	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; FasterCures
	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 Development, 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

Page 78 Cite

Suggested Citation: "Appendix B: Workshop Agenda." Institute of Medicine. 2015. Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/21732.