The Public Health Emergency Medical Countermeasures Enterprise

Innovative Strategies to Enhance Products from Discovery Through Approval

Workshop Summary

Theresa Wizemann, Clare Stroud, and Bruce M. Altevogt, Rapporteurs

Forum on Medical and Public Health Preparedness for Catastrophic Events

Forum on Drug Discovery, Development, and Translation

Board on Health Sciences Policy

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.
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The Public Health Emergency Medical Countermeasures Enterprise Innovative Strategies to Enhance Products from Discovery Through Approval Workshop Summary Theresa Wizemann, Clare Stroud, and Bruce M. Altevogt, Rapporteurs Forum on Medical and Public Health Preparedness for Catastrophic Events Forum on Drug Discovery, Development, and Translation Board on Health Sciences Policy

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THE NATIONAL ACADEMIES PRESS • 500 Fifth Street, N.W. • Washington, DC 20001 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. 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. 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. International Standard Book Number-13: 978-0-309-15024-8 International Standard Book Number-10: 0-309-15024-8 Additional copies of this report are available from The National Academies Press, 500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334- 3313 (in the Washington metropolitan area); Internet, http://www.nap.edu. For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. Copyright 2010 by the National Academy of Sciences. All rights reserved. Printed in the United States of America 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.

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The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Academy has a mandate that requires it to advise the federal government on scientific and technical matters. Dr. Ralph J. Cicerone is president of the National Academy of Sciences. The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding engineers. It is autonomous in its administration and in the selection of its members, sharing with the National Academy of Sciences the responsibility for advising the federal government. The National Academy of Engineering also sponsors engineering programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Charles M. Vest is president of the National Academy of Engineering. The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Institute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Harvey V. Fineberg is president of the Institute of Medicine. The National Research Council was organized by the National Academy of Sciences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and Dr. Charles M. Vest are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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Workshop Planning Committee* GAIL CASSELL (Chair), Eli Lilly and Company, Indianapolis, IN PHYLLIS ARTHUR, Biotechnology Industry Organization, Washington, DC CAPTAIN KENNETH COLE, Office of Assistant Secretary of Defense for Health Affairs, Department of Defense, Washington, DC ALEXANDER GARZA, Department of Homeland Security, Washington, DC JOHN GRABENSTEIN, Merck & Co., Inc., Whitehouse Station, NJ ELIN GURSKY, Analytic Services Inc., Arlington, VA TIL JOLLY, Department of Homeland Security, Washington, DC ROBERT KADLEC, PRTM Management Consultants, Washington, DC GEORGE KORCH, Department of Health and Human Services, Washington, DC MICHAEL KURILLA, National Institute of Allergy and Infectious Diseases, Bethesda, MD NICOLE LURIE, Department of Health and Human Services, Washington, DC BORIS LUSHNIAK, Food and Drug Administration, Rockville, MD MONIQUE MANSOURA, Department of Health and Human Services, Washington, DC PETER PALESE, Mount Sinai School of Medicine, New York RON SALDARINI, Biological Initiatives, Mahwah, NJ DANIEL SOSIN, Centers for Disease Control and Prevention, Atlanta, GA JANET TOBIAS, Ikana Media, New York ERIC TONER, University of Pittsburgh, Pittsburgh, PA STEPHANIE ZAZA, Centers for Disease Control and Prevention, Atlanta, GA ________________________ ∗ IOM planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution. v

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IOM Staff BRUCE ALTEVOGT, Preparedness Forum Director ROBERT GIFFIN, Drug Forum Director (until March 2010) CLARE STROUD, Program Officer ANDREW POPE, Director, Board on Health Sciences Policy AMY PACKMAN, Administrative Assistant ALEX REPACE, Senior Program Assistant vi

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Forum on Medical and Public Health Preparedness for Catastrophic Events* LEWIS GOLDFRANK (Chair), New York University Medical Center, New York DAMON ARNOLD, Association of State and Territorial Health Officials, Arlington, VA GEORGES BENJAMIN, American Public Health Association, Washington, DC D. W. CHEN, Office of Assistant Secretary of Defense for Health Affairs, Department of Defense, Washington, DC ROBERT DARLING, Uniformed Services University, Bethesda, MD VICTORIA DAVEY, Department of Veterans Affairs, Washington, DC JEFFREY DUCHIN, Seattle & King County and University of Washington, Seattle ALEXANDER GARZA, Department of Homeland Security, Washington, DC LYNN GOLDMAN, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD DAVID HENRY, National Governors Association, Washington, DC JACK HERRMANN, National Association of County and City Health Officials, Washington, DC KEITH HOLTERMANN, Federal Emergency Management Agency, Washington, DC JAMES JAMES, American Medical Association, Chicago, IL JERRY JOHNSTON, National Association of Emergency Medical Technicians, Mt. Pleasant, IA ROBERT KADLEC, PRTM Management Consultants, Washington, DC BRIAN KAMOIE, The White House, Washington, DC LYNNE KIDDER, Business Executives for National Security, Washington, DC MICHAEL KURILLA, National Institute of Allergy and Infectious Diseases, Bethesda, MD ________________________ * IOM forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution. vii

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JAYNE LUX, National Business Group on Health, Washington, DC ANTHONY MACINTYRE, American College of Emergency Physicians, Washington, DC ANGELA MCGOWAN, Robert Wood Johnson Foundation, Princeton, NJ MARGARET MCMAHON, Emergency Nurses Association, Williamstown, NJ ERIN MULLEN, Pharmaceutical Research and Manufacturers of America, Washington, DC GERALD PARKER, Office of the Assistant Secretary for Preparedness and Response, Department of Health and Human Services, Washington, DC CHERYL PETERSON, American Nurses Association, Silver Spring, MD SALLY PHILLIPS, Agency for Healthcare Research and Quality, Rockville, MD STEVEN PHILLIPS, National Library of Medicine, Bethesda, MD EDITH ROSATO, National Association of Chain Drug Stores Foundation, Alexandria, VA (since July 2009) PHILLIP SCHNEIDER, National Association of Chain Drug Stores Foundation, Alexandria, VA (until July 2009) ROSLYNE SCHULMAN, American Hospital Association, Washington, DC DANIEL SOSIN, Centers for Disease Control and Prevention, Atlanta, GA SHARON STANLEY, American Red Cross, Washington, DC ERIC TONER, University of Pittsburgh Medical Center, Pittsburgh, PA REED TUCKSON, UnitedHealth Group, Minneapolis, MN MARGARET VANAMRINGE, The Joint Commission, Washington, DC IOM Staff BRUCE ALTEVOGT, Project Director CLARE STROUD, Program Officer ANDREW POPE, Director, Board on Health Sciences Policy ALEX REPACE, Senior Program Assistant viii

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Forum on Drug Discovery, Development, and Translation* GAIL CASSELL (Co-Chair), Eli Lilly and Company, Indianapolis, IN JEFFREY DRAZEN (Co-Chair), New England Journal of Medicine, Boston, MA BARBARA ALVING, National Center for Research Resources, Bethesda, MD LESLIE BENET, University of California–San Francisco, CA ANN BONHAM, Association of American Medical Colleges, Washington, DC LINDA BRADY, National Institute of Mental Health, Bethesda, MD ROBERT CALIFF, Duke University Medical Center, Durham, NC SCOTT CAMPBELL, American Diabetes Association, Alexandria, VA THOMAS CASKEY, University of Texas HSC at Houston, Texas PETER CORR, Celtics Therapeutics LLLP, New York JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD PAUL EISENBERG, Amgen, Inc., Thousand Oaks, CA GARY FILERMAN, ATLAS Research, Washington, DC GARRET FITZGERALD, University of Pennsylvania School of Medicine, Philadelphia, PA ELAINE GALLIN, The Doris Duke Charitable Foundation, New York STEVEN GALSON, Science Operation International Corporation, Rockville, MD HARRY GREENBERG, Stanford University School of Medicine, Stanford, CA STEPHEN GROFT, Office of Rare Disease Research, National Institutes of Health, Bethesda, MD PETER HONIG, Merck & Co., Inc. (Retired), Collegeville, PA ANNALISA JENKINS, Bristol Myers Squibb, Plainsboro, NJ MICHAEL KATZ, March of Dimes Foundation, New York JACK KEENE, Duke University Medical Center, Durham, NC RONALD KRALL, GlaxoSmithKline (Retired), Steamboat Springs, CO ________________________ * IOM forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution. ix

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FREDA LEWIS-HALL, Pfizer Inc., New York WILLIAM MATTHEW, National Institute of Neurological Disorders and Stroke, Bethesda, MD MARK MCCLELLAN, Brookings Institution, Washington, DC CAROL MIMURA, University of California–Berkeley, CA JOHN ORLOFF, Novartis Pharmaceuticals Corporation, East Hanover, NJ AMY PATTERSON, National Institutes of Health, Bethesda, MD JANET SHOEMAKER, American Society for Microbiology, Washington, DC LANA SKIRBOLL, National Institutes of Health, Bethesda, MD NANCY SUNG, Burroughs Wellcome Fund, Research Triangle Park, NC JORGE TAVEL, National Institute of Allergy and Infectious Diseases, Bethesda, MD JANET TOBIAS, Ikana Media, New York JOANNE WALDSTREICHER, Johnson & Johnson Pharmaceutical Research and Development, LLC, Raritan, NJ JANET WOODCOCK, U.S. Food and Drug Administration, Rockville, MD RAYMOND WOOSLEY, The Critical Path Institute, Tucson, AZ IOM Staff ANNE CLAIBORNE, Director (since April 2010) ROBERT B. GIFFIN, Director (until March 2010) REBECCA A. ENGLISH, Research Associate YEONWOO LEBOVITZ, Program Associate GENEA S. VINCENT, Senior Program Assistant x

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Reviewers This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the National Research Council’s Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its pub- lished report as sound as possible and to ensure that the report meets in- stitutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confi- dential to protect the integrity of the process. We wish to thank the fol- lowing individuals for their review of this report: Susan R. Cooper, Tennessee Department of Health Joseph A. DiMasi, Tufts Center for the Study of Drug Development Philip K. Russell, Department of Defense (retired) Patrick J. Scannon, XOMA, Ltd. P. Roy Vagelos, Merck & Co., Inc. (retired) Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the report before its release. The review of this report was overseen by Leslie Z. Benet. Appointed by the Institute of Medicine, he was responsible for making certain that an independent examination of this report was car- ried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authoring committee and the institution. xi

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Contents INTRODUCTION 1 About This Summary, 3 Charge to Workshop Participants, 4 BACKGROUND 5 The Public Health Perspective on Medical Countermeasure Development, Acquisition, and Use, 8 The FDA Perspective on the Countermeasures Enterprise: Moving Forward, 9 Government Procurement of Science, 11 PARTNERS IN A SINGLE MISSION, DIVERSE CONCERNS AND CHALLENGES 11 The Growing Threat of Bioweapons, 12 Gaps and Barriers to International Collaboration, 12 Issues for Federal Agencies Engaged in Countermeasures Development, 14 Challenges Facing the Innovative Biopharmaceutical Industry, 18 Research Infrastructure and Resources, 23 Liability, 24 End Users: Challenges for Public Health and Providers, 24 EXAMPLES OF SUCCESSFUL COUNTERMEASURES DEVELOPMENT AND DEPLOYMENT 25 Features of Successful Government Countermeasures Efforts, 25 Countermeasures Development in Industry, 29 xiii

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xiv CONTENTS PARTNERSHIPS AND ALTERNATIVE BUSINESS MODELS 30 Venture Philanthropy and Orphan Product Development Models, 30 Pharmaceutical Shared-Risk Approaches, 31 Planning for Failure, 32 Open Innovation Business Strategies, 33 Public–Private Partnerships, 34 Independent Third-Party Facilitation of Collaboration, 37 Strategic Investor Model, 37 ENGAGING INDUSTRY 38 Incentives: Push vs. Pull, 38 Incentives Not Needed?: Making a Strong Business Case, 41 NEW PARADIGMS, STRATEGIES, AND TACTICS FOR ENHANCING THE COUNTERMEASURES DEVELOPMENT ENTERPRISE 43 Outsourcing Program Management, 43 Government as a Strategic Partner, 45 Platform Technologies, 45 Revised PHEMCE Implementation Plan, 46 EXISTING REGULATORY TOOLS AND APPROACHES THAT CAN BE APPLIED TO ADVANCE COUNTERMEASURES DEVELOPMENT 47 Opportunities for Accelerating Approval of Medical Countermeasures: Evolving the Regulatory Framework, 47 The Way Forward: Themes from the Workshop, 49 CONCLUSION 53 APPENDIXES A References 57 B Workshop Agenda 59 C Registered Workshop Attendees 79 D Case Studies of HHS Chemical, Biological, Radiological, and Nuclear Medical Countermeasure Development Programs, Executive Summary 91 E Synthesis of Business Models and Economic and Market Incentives for Vaccines and Therapeutics 113