Excerpt

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 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.

Contents

• The National Academies
• Workshop Planning Committee
• Forum on Medical and Public Health Preparedness for Catastrophic Events
• Forum on Drug Discovery, Development, and Translation
• Reviewers
• Introduction
  • About This Summary
  • Charge to Workshop Participants
• Background
  • The Public Health Perspective on Medical Countermeasure Development, Acquisition, and Use
  • The FDA Perspective on the Countermeasures Enterprise: Moving Forward
  • Government Procurement of Science
• Partners in a Single Mission, Diverse Concerns and Challenges
  • The Growing Threat of Bioweapons
  • Gaps and Barriers to International Collaboration
  • Issues for Federal Agencies Engaged in Countermeasures Development
  • Challenges Facing the Innovative Biopharmaceutical Industry
  • Research Infrastructure and Resources
  • Liability
  • End Users: Challenges for Public Health and Providers
• Examples of Successful Countermeasures Development and Deployment
  • Features of Successful Government Countermeasures Efforts
  • Countermeasures Development in Industry
• Partnerships and Alternative Business Models
  • Venture Philanthropy and Orphan Product Development Models
  • Pharmaceutical Shared-Risk Approaches
  • Planning for Failure
  • Open Innovation Business Strategies
  • Public–Private Partnerships
  • Independent Third-Party Facilitation of Collaboration
  • Strategic Investor Model
• Engaging Industry
  • Incentives: Push vs. Pull
  • Incentives Not Needed?: Making a Strong Business Case
• New Paradigms, Strategies, and Tactics for Enhancing the Countermeasures Development Enterprise
  • Outsourcing Program Management
  • Government as a Strategic Partner
  • Platform Technologies
  • Revised PHEMCE Implementation Plan
• Existing Regulatory Tools and Approaches That Can Be Applied to Advance Countermeasures Development
  • Opportunities for Accelerating Approval of Medical Countermeasures: Evolving the Regulatory Framework
  • The Way Forward: Themes from the Workshop
• Conclusion
• Appendixes
  • A References
  • B Workshop Agenda
  • C Registered Workshop Attendees
  • D Case Studies of HHS Chemical, Biological, Radiological, and Nuclear Medical Countermeasure Development Programs, Executive Summary
  • E Synthesis of Business Models and Economic and Market Incentives for Vaccines and Therapeutics

This project was supported by contracts between the National Academy of Sciences and the American College of Emergency Physicians, the American Hospital Association, the American Medical Association, the American Nurses Association, the Association of State and Territorial Health Officials, the Centers for Disease Control and Prevention (Contract No. 200-2005-13434 TO #6), the Department of the Army (Contract No. W81XWH-08-P-0934), the Department of Health and Human Services’ Agency for Healthcare Research and Quality (Contract No. HHSP233200800498P), the Department of Health and Human Services’ National Institutes of Health (Contract No. N01-OD-4-2139 TO #198), the Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response (Contract Nos. HHSP233200900680P, HH5P23320042509X1), the Department of Homeland Security’s Office of Health Affairs (Contract No. HSHQDC-07-C-00097), the Department of Homeland Security’s Federal Emergency Management Agency (Contract No. HSFEHQ-08-P-1800), the Department of Veterans Affairs (Contract No. V101(93)P-2136 TO #10), the Emergency Nurses Association, the National Association of Chain Drug Stores, the National Association of County and City Health Officials, the National Association of Emergency Medical Technicians, the Pharmaceutical Research and Manufacturers of America, The Robert Wood Johnson Foundation, and the United Health Foundation.

Suggested citation:

IOM (Institute of Medicine). 2010. The Public Health Emergency Medical Countermeasures Enterprise: Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary. Washington, DC: The National Academies Press.

The views presented in this publication are those of the editors and attributing authors and do not necessarily reflect the views of the organizations or agencies that provided support for this project.

NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance.