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OCR for page 59
The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
Explore opportunities to optimize the public health emergency medical countermeasures enterprise.
Propose strategies for enhancing collaboration and coordination within the enterprise, taking into account constraints related to ongoing programmatic 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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary
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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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