B
Workshop Agenda

Day 1

February 22, 2010

House of Sweden

Anna Lindh Hall

2900 K Street, NW

Washington, DC 20007


Background:

This workshop will examine federal policies and activities that relate to discovery through approval of medical countermeasures (e.g., vaccines, drugs, and diagnostics) for responding to public health emergencies with the potential to rapidly overwhelm the public health and medical systems (e.g., terrorism and pandemic flu). The workshop will explore potential opportunities to enhance this enterprise by evaluating other models or systems that have similar goals of developing medical products with low commercial viability. As charged by the Secretary of Health and Human Services, the Office of the Assistant Secretary for Preparedness and Response is leading a review of the entire public health emergency medical countermeasures enterprise, to be completed in the first quarter of this year. Because there is a limited commercial market for most medical countermeasures, the government has had to create incentives to encourage private-sector pharmaceutical and biotech companies to develop the needed products. The countermeasures research and development (R&D) enterprise encompasses many partners from across the federal government, states, and industry, which need to function together to develop the medical countermeasures necessary to sustain



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B Workshop Agenda Day 1 February 22, 2010 House of Sweden Anna Lindh Hall 2900 K Street, NW Washington, DC 20007 Background: This workshop will examine federal policies and activities that relate to discovery through approval of medical countermeasures (e.g., vaccines, drugs, and diagnostics) for responding to public health emergencies with the potential to rapidly overwhelm the public health and medical systems (e.g., terrorism and pandemic flu). The workshop will explore potential opportunities to enhance this enterprise by evaluating other models or systems that have similar goals of developing medical products with low commercial viability. As charged by the Secretary of Health and Human Services, the Office of the Assistant Secretary for Preparedness and Response is leading a review of the entire public health emergency medical countermeasures enterprise, to be completed in the first quarter of this year. Because there is a limited commercial market for most medical countermeasures, the government has had to create incentives to encourage private-sector pharmaceutical and biotech companies to develop the needed products. The countermeasures research and development (R&D) enterprise encompasses many partners from across the federal government, states, and industry, which need to function together to develop the medical countermeasures necessary to sustain 59

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60 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE national health security. However, certain structural, strategic, and technical elements of the enterprise continue to impede research, development, and production of medical countermeasures. The presentations and discussions at this workshop are intended to assist federal officials in conducting a thorough review of the “pipeline through approval” spectrum of our national programs and to assist in the ultimate goal of improving the efficiency and effectiveness of the countermeasures enterprise. Meeting Objectives: • Identify and discuss strategies to optimize the federal public health emergency medical countermeasures enterprise, and explore resources and/or other supporting components needed for accomplishing goals of countermeasure discovery, development, approval, and production. • Examine strategies to further enhance the translation of early phase investments in basic science into potential public health interventions. • Identify and discuss models for enhancing current partnerships and establishing new ones among federal programs, innovators, and the commercial marketplace to enhance our nation’s capabilities to meet public health emergency preparedness goals. • Consider market forces acting on the advanced development biodefense community (pharma/biotech) that incentivize/ disincentivize efforts to develop and license products in support of the national response. • Examine ways the regulatory oversight process for public health emergency medical countermeasures might evolve and identify ways to enable more efficient approval and use. • Review the innovative approaches being used to advance drug development for orphan diseases (i.e., rare, neglected, or tropical diseases) or any other area that does not have a ready and sustainable commercial market (e.g., oncology therapeutics), and identify the shared challenges and opportunities for strategies that might be adopted by the Enterprise.

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APPENDIX B 61 Working Dinner 6:00 p.m. Welcome, Introductions, and Charge to Workshop Participants GAIL CASSELL, Workshop Chair Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company 6:15 p.m. Needs and Opportunities to Advance the Countermeasures Enterprise RANDY LARSEN Executive Director, Commission on the Prevention of Weapons of Mass Destruction Proliferation and Terrorism Presentation of White Papers 6:35 p.m. Case Studies of the HHS Medical Countermeasure Programs (Paper 1) ROBERT KADLEC Vice President Global Public Sector PRTM 6:55 p.m. Optimizing the Medical Countermeasure Product Pipeline from the Science Base Through Advanced Development (Paper 2) GEORGE KORCH Senior Science Advisor Principal Deputy Assistant Secretary for Preparedness and Response Office of the Assistant Secretary for Preparedness and Response Department of Health and Human Services

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62 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE 7:15 p.m. Synthesis of Business Models and Economic and Market Incentives for Vaccines and Therapeutics (Paper 3) JAMES GUYTON Principal Public Health and Biodefense Practice PRTM 7:35 p.m. National Biodefense Science Board (NBSB): Medical Countermeasure Markets and Sustainability JOHN GRABENSTEIN Senior Medical Director for Merck Vaccines Member, National Biodefense Science Board 7:50 p.m. Discussion with Attendees 8:15 p.m. ADJOURN Day 2 February 23, 2010 House of Sweden Alfred Nobel Hall 2900 K Street, NW Washington, DC 20007 8:00 a.m. Welcome and Introductions GAIL CASSELL, Workshop Chair Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company

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APPENDIX B 63 8:10 a.m. Charge to Workshop Participants and Overview of the Federal Public Health Countermeasures Enterprise: Challenges and Opportunities NICOLE LURIE Assistant Secretary for Preparedness and Response Office of the Assistant Secretary for Preparedness and Response Department of Health and Human Services 8:30 a.m. The Public Health Perspective on Medical Countermeasure Development, Acquisition, and Use THOMAS FRIEDEN Director Centers for Disease Control and Prevention SESSION I: THE COUNTERMEASURES ENTERPRISE: OVERVIEW OF THE CHALLENGES AND OPPORTUNITIES 8:50 a.m. Past and Present Enterprise Efforts: Why Are We Where We Are and What Models Are Most Likely to Succeed? PHILIP RUSSELL Board of Trustees Sabin Vaccine Institute 9:05 a.m. International Approaches to Countermeasure Research, Development, and Approval MARIA JULIA MARINISSEN Team Leader, International Partnerships and Initiatives Office of the Assistant Secretary for Preparedness and Response Department of Health and Human Services

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64 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE 9:20 a.m. Defining the Steps of the Critical Pathway: Strategies to Move Forward MARIETTA ANTHONY Associate Director Arizona Center for Education & Research on Therapeutics Critical Path Institute 9:35 a.m. Commercial Challenges: Perspectives from the Biotech Industry ERIC ROSE CEO and Chair, Board of Directors Siga Technologies, Inc. 9:50 a.m. Commercial Challenges: Perspectives from Big Pharma JOHN REX Infection Clinical Vice President Oncology & Infection Therapy Area AstraZeneca 10:05 a.m. Discussion with Attendees 10:30 a.m. BREAK SESSION II: MEDICAL COUNTERMEASURE EXPERIENCES FROM PANDEMIC INFLUENZA AND ANTHRAX PLANNING: IDENTIFYING LATE-STAGE ENTERPRISE ISSUES THAT IMPACT EARLY STAGE DECISION MAKING Session Objectives: • Provide context for the countermeasures enterprise given the ultimate use of medical countermeasures. • Explore how the considerations of various end users should inform the design of the Enterprise and developmental product profiles.

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APPENDIX B 65 • Provide CDC and state public health practice experiences and lessons regarding H1N1 pandemic medical countermeasure policies, distribution, and uses. • Provide examples of challenges and opportunities faced in developing an anthrax medical countermeasures distribution model. • Identify the issues from H1N1 experiences that can be generalized or transferred to questions about the broad range of medical countermeasure programs. 10:45 a.m. Session Introduction MONIQUE MANSOURA, Session Chair Director for Medical Countermeasure Policy, Planning and Requirements Office of the Biomedical Advanced Research and Development Authority, Office of the Assistant Secretary for Preparedness and Response Department of Health and Human Services 10:50 a.m. Panel Presentations DANIEL JERNIGAN Deputy Influenza Director Centers for Disease Control and Prevention SUSAN COOPER Commissioner Tennessee Department of Public Health DAMON ARNOLD (via telecon) Director Illinois Department of Public Health ANDREW PAVIA George and Esther Gross Presidential Professor Division of Pediatric Infectious Diseases University of Utah School of Medicine

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66 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE 11:10 a.m. Discussion with Attendees 11:40 a.m. LUNCH: Atrium Lounge SESSION III: OPTIMIZING THE RESEARCH AND DEVELOPMENT ELEMENTS OF THE ENTERPRISE Session Objectives: • Review the structural and strategic elements of current efforts by the relevant federal agencies to support the research, development, and production of emergency medical countermeasures, including consideration of the underlying infrastructure that supports these efforts. • Identify and discuss the critical paths and systems approaches needed to optimize the research, development, and approval elements of the medical countermeasures development enterprise. • Propose strategies to optimize the federal public health countermeasures enterprise. • Identify and discuss models for enhancing collaboration and coordination among relevant federal programs. Examine the enabling technologies and infrastructures that will be necessary. 12:30 p.m. Session Introduction and Objectives ROBERT KADLEC, Session Chair Vice President Global Public Sector PRTM 12:35 p.m. Panel Discussion: The Countermeasures Enterprise: Current Constraints and Opportunities to Move Forward • Review the structural and strategic elements of current efforts by the relevant federal agencies to support the research, development, and production of emergency medical countermeasures, including consideration of the underlying infrastructure that supports these efforts. • Discuss the current constraints of the structure and organization of the countermeasure enterprise.

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APPENDIX B 67 • Explore opportunities to optimize the public health emergency medical countermeasures enterprise. o Propose strategies for enhancing collaboration and coordination within the enterprise, taking into account constraints related to ongoing program- matic activities, budget cycles, overlapping goals, and limitations due to existing federal regulations and departmental policies. NIH ANTHONY FAUCI Director National Institute of Allergy and Infectious Diseases, NIH Department of Health and Human Services BARDA ROBIN ROBINSON Director Office of the Biomedical Advanced Research and Development Authority, Office of the Assistant Secretary for Preparedness and Response Department of Health and Human Services DoD KENNETH COLE Medical Director Nuclear and Chemical and Biological Defense Programs Office of the Deputy Assistant to the Secretary of Defense Department of Defense

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68 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE Perspective of Prior Leadership D. A. HENDERSON Former Director, Office of Public Health Emergency Preparedness Director of WHO Smallpox Eradication Program Distinguished Scholar, Center for Biosecurity of UPMC 1:30 p.m. Discussion with Attendees 2:00 p.m. BREAK SESSION IV: OPPORTUNITIES FOR ENHANCING TRANSLATION OF BASIC SCIENCE: MODELS TO IMPROVE INNOVATION THAT RESPONDS TO NATIONAL PRIORITIES Session Objectives: • Explore how our investment in basic research is currently “procured” and “exploited” to yield products for the advanced development pipeline. • Identify potential other models to improve innovation based on national priorities. • Examine whether the structure and nature of current investments in science lead to the intended result of potential products or product candidates. • Discuss how the Enterprise can be the most collaborative and constructive partners with industry to ensure efficient product development. • Discuss how the current investment in basic science can be better exploited to improve the MCM research infrastructure to ensure more coordinated and collaborative research that effectively advances the Enterprise.

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APPENDIX B 69 2:15 p.m. Panel Discussion: Models for Procuring Science MICHAEL KURILLA, Panel Chair Director, Office of BioDefense Research Affairs Associate Director for BioDefense Product Development NIAID MICHAEL GOLDBLATT President and CEO Functional Genetics DAVID WURTMAN VP, Corporate Development NexBio BRETT GIROIR Vice Chancellor for Research Texas A&M PAUL OWENS Senior Director, Chorus CMC Lilly Research Laboratories 2:50 p.m. Discussion with Attendees 3:20 p.m. Panel Discussion: Strategies to Improve Portfolio Management: Translating Basic Science PHYLLIS ARTHUR, Panel Chair Director Health & Regulatory Affairs BIO

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70 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE Overview: Current Process for Making Go/No- Go Decisions MICHAEL KURILLA Director, Office of BioDefense Research Affairs Associate Director for BioDefense Product Development NIAID Panel Discussion DAVID PERRYMAN President & CEO Zirus GEORGE PAINTER CEO Chimerix TYLER MARTIN Chief Medical Officer Dynavax PATRICK IVERSEN Senior Vice President Strategic Alliances AVI BioPharma JOHN REX Infection Clinical Vice President Oncology & Infection Therapy Area AstraZeneca 4:00 p.m. Discussion with Attendees 5:00 p.m. ADJOURN

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APPENDIX B 71 Day 3 February 24, 2010 House of Sweden Alfred Nobel Hall 2900 K Street, NW Washington, DC 20007 KEYNOTE PRESENTATION: FDA CONTRIBUTIONS TO THE ENTERPRISE: CURRENT AND FUTURE STRATEGIES 8:00 a.m. Welcome and Introduction GAIL CASSELL, Workshop Chair Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company 8:05 a.m. Keynote Presentation JESSE L. GOODMAN Chief Scientist and Deputy Commissioner Science and Public Health (Acting) Food and Drug Administration SESSION V: OPPORTUNITIES FOR ACCELERATING APPROVAL OF MEDICAL COUNTERMEASURES: EVOLVING THE REGULATORY FRAMEWORK Session Objectives: Review current regulatory authority and discuss current barriers to approval of products emerging from the countermeasures enterprise. Identify and discuss innovative approaches to facilitate effective regulation of countermeasures for rapidly emerging and/or rare public health threats, while still ensuring appropriate review of safety and efficacy data. Examine the current scientific infrastructure at the FDA and opportunities for the agency to be better prepared for the needs of the countermeasure community. Discuss whether FDA approval should be the standard for all medical countermeasures.

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72 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE 8:35 a.m. Session Objectives and Introduction BORIS LUSHNIAK, Session Chair Assistant Commissioner for Counterterrorism Policy Food and Drug Administration 8:40 a.m. Panel Discussion GERALD PARKER Principal Deputy Assistant Secretary Office of the Assistant Secretary for Preparedness and Response Department of Health and Human Services MARY PENDERGAST Founder, Pendergast Consulting Former Deputy Commissioner of the FDA JEANNE NOVAK CEO and President CBR International Corp. LUCIANA BORIO Medical Reviewer Office of Vaccine Research and Review Center for Biologics Evaluation and Research Food and Drug Administration GAIL CASSELL Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company 9:40 a.m. Discussion with Attendees 10:10 a.m. BREAK

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APPENDIX B 73 SESSION VI: MARKET INCENTIVES IN THE DEVELOPMENT OF MEDICAL COUNTERMEASURES: IDENTIFYING MARKET OPPORTUNITIES AND ELIMINATING DISINCENTIVES Session Objectives: How can the federal government better ensure that the emerging basic science concepts are translated into candidate countermeasures? What market issues exist, and what financial incentives are needed to overcome these issues? Explore the impact of the animal efficacy rule and opportunities to decrease its impact on the product development time frame while still ensuring appropriate review of safety and efficacy data. Discuss the advantages and disadvantages of establishing a consolidated federal intramural program to support translational and preclinical studies. Consider the impact of contract requirements and use policy on the private sector. Explore strategies to offset the limited commercial marketplace for emergency medical countermeasures. Examine the impact of existing liability provisions. 10:25 a.m. Session Objectives and Introduction JOHN GRABENSTEIN, Session Chair Senior Medical Director for Adult Vaccines Merck & Co., Inc. 10:30 a.m. Panel Discussion: Other Models, Lessons Learned, and Success Stories from the World of “Orphan Drugs” and Challenging Commercial Products CHANTAL MOREL (via telecon) Department of Social Policy London School of Economics MARGARET ANDERSON Executive Director FasterCures MELINDA MOREE President and CEO BIO Ventures for Global Health

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74 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE MARLENE HAFFNER (via telecon) President Haffner Associates, LLC VICTORIA SUTTON Robert H. Bean Professor of Law Texas Tech University School of Law RAJEEV VENKAYYA Director, Global Health Delivery Bill & Melinda Gates Foundation 11:00 a.m. Discussion with Attendees 11:30 a.m. Panel Discussion: Market Incentives WESLEY YIN Assistant Professor Department of Economics Boston University THOMAS MONATH Partner Pandemic and Biodefense Fund Kleiner Perkins Caufield & Byers DAVID GILBERT Director Infectious Diseases Providence Health & Services DAVID RIDLEY Assistant Professor The Fuqua School of Business Duke University

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APPENDIX B 75 BRUCE ARTIM Director Federal Affairs Eli Lilly and Company BRADLEY SMITH Senior Associate Center for Biosecurity, UPMC 12:15 p.m. Discussion with Attendees 12:45 a.m. LUNCH: Atrium Lounge SESSION VII: THE ROLE OF PARTNERSHIPS AND ALTERNATIVE BUSINESS MODELS Session Objectives: Examine how partnerships (public–private, private– private, and public–public) and alternative business models can be established to mitigate the risk for each sector. Discuss opportunities to leverage the expertise of pharma while retaining the innovation from biotech. Explore the role that public–private and private–private partnerships may have in supporting the development of multiproduct technologies and facilities. Identify opportunities for partnerships to help establish smoother transition from discovery to development and testing to regulatory approval for MCMs. 1:30 p.m. Session Objectives and Introduction RONALD SALDARINI, Session Chair President Biological Initiatives 1:35 p.m. Panel Discussion: Partnerships: Opportunities to Leverage the Expertise of Pharma While Retaining the Innovation from Biotech PHILLIP GOMEZ Director Global Public Sector PRTM

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76 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE TERI MELESE Director of Research Technologies and Alliances UCSF School of Medicine ROBERT HOUSE President DynPort Vaccine Company, LLC DOUGLAS PON Assistant Vice President Licensing in Global Business Development Pfizer Inc. 2:05 p.m. Discussion with Attendees SESSION VIII: “BLUE SKY” SESSION Session Objectives: Explore options to bring about a paradigm shift to the public health countermeasures enterprise from research, development, and approval. Discuss strategies to implement change, including legal, statutory, and regulatory authorities. 2:40 p.m. Session Objectives and Introduction GAIL CASSELL, Workshop Chair Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company 2:45 p.m. Panel Discussion: Proposing Paradigm Shifts GILLIAN WOOLLETT Chief Scientist Engel & Novitt, LLP

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APPENDIX B 77 FRANK GOTTRON Specialist in Science and Technology Policy Congressional Research Service U.S. Library of Congress THOMAS MONATH Partner Pandemic and Biodefense Fund Kleiner Perkins Caufield & Byers BRETT GIROIR Vice Chancellor for Research Texas A&M ERIC ROSE CEO and Chair, Board of Directors Siga Technologies, Inc. BRUCE ARTIM Director Federal Affairs Eli Lilly and Company CHUCK LUDLAM Former Counsel, Senator Joseph Lieberman Former Principal Lobbyist for The Biotechnology Industry Organization PHILLIP GOMEZ Director Biodefense and Public Health Practice PRTM

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78 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE 3:40 p.m. Discussion with Attendees: What Options Rise to the Top? What Have We Not Considered? GAIL CASSELL, Workshop Chair Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases Eli Lilly and Company 4:30 p.m. ADJOURN