GLOBAL HEALTH RISK FRAMEWORK
Research and Development of
Medical Products
WORKSHOP SUMMARY
Theresa Wizemann, Michelle A. Mancher, and Anne B. Claiborne,
Rapporteurs
Board on Health Sciences Policy
Institute of Medicine
THE NATIONAL ACADEMIES PRESS
Washington, DC
THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001
This activity was supported by Sponsor Award No. 11849 from the Paul G. Allen Family Foundation, Grant No. 0150-0653 from the Ford Foundation, Investment ID OPP1130763 from The Bill & Melinda Gates Foundation, Dr. Ming Wai Lau, Grant No. 4829 from the Gordon and Betty Moore Foundation, Grant No. 2015 EBO 308 from The Rockefeller Foundation, Award No. GHN-G-00-07-00001-00 from the U.S. Agency for International Development, and the Wellcome Trust. Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project.
International Standard Book Number-13: 978-0-309-38099-7
International Standard Book Number-10: 0-309-38099-5
Digit Object Identifier: 10.17226/21853
Additional copies of this report are available for sale from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.
Copyright 2016 by the National Academy of Sciences. All rights reserved.
Printed in the United States of America
Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2016. Global health risk framework: Research and development of medical products: Workshop summary. Washington, DC: The National Academies Press. doi: 10.17226/21853.
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PLANNING COMMITTEE ON GLOBAL HEALTH RISK
FRAMEWORK: A WORKSHOP ON RESEARCH AND
DEVELOPMENT OF MEDICAL PRODUCTS1
MARIA FREIRE (Co-Chair), Foundation for the National Institutes of Health, Bethesda, MD
TADATAKA “TACHI” YAMADA (Co-Chair), Frazier Life Sciences, Seattle, WA
LUCIANA BORIO, U.S. Food and Drug Administration, Bethesda, MD
RAYMOND CHUA, Health Products Regulation Group, Health Sciences Authority, Singapore
NEIL FERGUSON, Department of Mathematical Biology, Imperial College London, London, United Kingdom
GLENDA GRAY, South African Medical Research Council, Cape Town, South Africa
MARGARET “PEGGY” HAMBURG, National Academy of Medicine, Washington, DC
DAVID KASLOW, PATH, Seattle, WA
PATRICK LUKULAY, U.S. Pharmacopeia, Rockville, MD
ADEL MAHMOUD, Princeton University, Princeton, NJ
MARGARETH NDOMONDO-SIGONDA, African Union—New Partnership for Africa’s Development Planning and Coordinating Agency, Johannesburg, South Africa
JOHN REX, AstraZeneca, Waltham, MA
JUDIT RIUS, Médecins Sans Frontiéres Access Campaign, New York, NY
PETER SMITH, London School of Hygiene & Tropical Medicine, London, United Kingdom
PATRICK VALLANCE, GlaxoSmithKline, London, United Kingdom
IOM Staff
ANNE B. CLAIBORNE, Project Director
MICHELLE A. MANCHER, Program Officer
KATHRYN HOWELL, Research Assistant (until November 2015)
BENJAMIN KAHN, Senior Program Assistant
ANDREW M. POPE, Director, Board on Health Sciences Policy
__________________
1 The National Academies of Sciences, Engineering, and Medicine’s 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.
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BOARD ON HEALTH SCIENCES POLICY1
JEFFREY KAHN (Chair), Johns Hopkins University, Baltimore, MD
ELI Y. ADASHI, Brown University, Providence, RI
WYLIE BURKE, University of Washington, Seattle
R. ALTA CHARO, University of Wisconsin–Madison
LINDA H. CLEVER, California Pacific Medical Center, RENEW, Mill Valley, CA
BARRY S. COLLER, The Rockefeller University, New York, NY
LEWIS R. GOLDFRANK, New York University School of Medicine, New York
BERNARD A. HARRIS, JR., Vesalius Ventures, Houston, TX
MARTHA N. HILL, Johns Hopkins University School of Nursing, Baltimore, MD
STEVEN E. HYMAN, Stanley Center, The Broad Institute, Cambridge, MA
ALAN M. JETTE, Boston University School of Public Health, Boston, MA
PATRICIA A. KING, Georgetown University Law Center, Washington, DC
STORY C. LANDIS, National Institute of Neurological Disorders and Stroke, Bethesda, MD
HARRY T. ORR, University of Minnesota, Minneapolis
UMAIR SHAH, Harris County Public Health & Environmental Services, Houston, TX
ROBYN STONE, Leading Age, Washington, DC
SHARON TERRY, Genetic Alliance, Washington, DC
REED V. TUCKSON, Tuckson Health Connections, LLC, Edina, MN
IOM Staff
HILARY BRAGG, Program Coordinator
ANDREW M. POPE, Director, Board on Health Sciences Policy
__________________
1 The National Academies of Sciences, Engineering, and Medicine’s boards do not review or approve individual products. The responsibility for the content of the workshop summary rests with the workshop rapporteurs and the institution.
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Reviewers
This workshop summary has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published workshop summary as sound as possible and to ensure that the workshop summary meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the process. We wish to thank the following individuals for their review of this workshop summary:
GARY NABEL, Sanofi
BERNARD PECOUL, Drugs for Neglected Diseases Initiative
BT SLINGSBY, Global Health Innovative Technology Fund
SAMBA SOW, Center for Vaccine Development–Mali
RAJEEV VENKAYYA, Takeda Pharmaceuticals
Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this workshop summary was overseen by ROBERT LAWRENCE, Johns Hopkins University. He was responsible for making certain that an independent examination of this workshop summary was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this workshop summary rests entirely with the rapporteurs and the institution.
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Acknowledgments
The National Academies of Sciences, Engineering, and Medicine staff and planning committee thank Dr. Gabriel Leung and his staff, including particularly Mr. Keith Tin, for graciously hosting and supporting Global Health Risk Framework: A Workshop on Research and Development of Medical Products at The University of Hong Kong in August 2015. The meeting support provided by the firm Weber Shandwick is also gratefully acknowledged.
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Contents
The Global Health Risk Framework Initiative
The Workshop on Research and Development of Medical Products
WHO Perspective on the Global Health Risk Framework
2 MODELS AND INCENTIVES FOR ENGAGEMENT
Public–Private Partnerships as the Essential Operating Model
Creating a Blend of Incentives for Engagement of Partners
Advancing the Science of Discovery
Intellectual Property and Sharing of Data and Reagents
Ethical Principles and Methodological Framework for Clinical Trial Designs
Practical Considerations and Community Engagement
5 CONVERGENCE OF REGULATORY EXPECTATIONS, REVIEW, AND APPROVAL
Coordination, Harmonization, and Convergence
6 MANUFACTURING, STOCKPILING, AND DISTRIBUTION
7 CRITICAL CONSIDERATIONS FOR FACILITATING MEDICAL PRODUCT RESEARCH AND DEVELOPMENT
Defining the Priorities for Research and Development
Acronyms and Abbreviations
AAV |
adeno-associated viruses |
AVAREF |
African Vaccine Regulatory Forum |
BARDA |
Biomedical Advanced Research and Development Authority |
BMGF |
The Bill & Melinda Gates Foundation |
BPO |
Biopreparedness Organization |
CDC |
Centers for Disease Control and Prevention (U.S.) |
CHAI |
Clinton Health Access Initiative |
DNDi |
Drugs for Neglected Diseases initiative |
DoD |
U.S. Department of Defense |
EMA |
European Medicines Agency |
EU |
European Union |
EUA |
emergency use authorization |
FDA |
U.S. Food and Drug Administration |
GAAS |
Ghana Academy of Arts and Sciences |
GDP |
good distribution practice |
GHIT |
Global Health Innovative Technology Fund |
GHRF |
Global Health Risk Framework |
GSK |
GlaxoSmithKline |
HHS |
U.S. Department of Health and Human Services |
HIV |
human immunodeficiency virus |
ICMRA |
International Coalition of Medicines Regulatory Authorities |
IHR |
International Health Regulations |
IOM |
Institute of Medicine |
IP |
intellectual property |
IRB |
institutional review board |
MCM |
medical countermeasure |
MERS |
Middle East respiratory syndrome |
MMV |
Medicines for Malaria Venture |
MSF |
Médecins Sans Frontières (Doctors Without Borders) |
MTA |
Material Transfer Agreement |
NAM |
National Academy of Medicine (U.S.) |
NGO |
nongovernmental organization |
NIH |
National Institutes of Health (U.S.) |
PCR |
polymerase chain reaction |
PHEMCE |
Public Health Emergency Medical Countermeasures Enterprise |
PPE |
personal protective equipment |
PREP |
Public Readiness and Emergency Preparedness |
RCT |
randomized controlled trial |
SAMRC |
South African Medical Research Council |
SARS |
severe acute respiratory syndrome |
SNS |
Strategic National Stockpile (U.S.) |
TB |
tuberculosis |
UBMTA |
Uniform Biological Materials Transfer Agreement |
UN |
United Nations |
UNICEF |
United Nations Children’s Fund |
WHO |
World Health Organization |