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

www.nap.edu

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.

The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Ralph J. Cicerone is president.

The National Academy of Engineering was established in 1964 under the charter of the National Academy of Sciences to bring the practices of engineering to advising the nation. Members are elected by their peers for extraordinary contributions to engineering. Dr. C. D. Mote, Jr., is president.

The National Academy of Medicine (formerly the Institute of Medicine) was established in 1970 under the charter of the National Academy of Sciences to advise the nation on medical and health issues. Members are elected by their peers for distinguished contributions to medicine and health. Dr. Victor J. Dzau is president.

The three Academies work together as the National Academies of Sciences, Engineering, and Medicine to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine.

Learn more about the National Academies of Sciences, Engineering, and Medicine at www.national-academies.org.

This page intentionally left blank.

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.

This page intentionally left blank.

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.

This page intentionally left blank.

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:

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.

This page intentionally left blank.

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.

This page intentionally left blank.

Contents

ACRONYMS AND ABBREVIATIONS

1 INTRODUCTION

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

Elements of Success

Challenges

3 DISCOVERY RESEARCH

Advancing the Science of Discovery

Intellectual Property and Sharing of Data and Reagents

Liability

4 DEVELOPMENT

Ethical Principles and Methodological Framework for Clinical Trial Designs

Methodological Design Considerations

Ethical Review

Regulatory Perspective

Practical Considerations and Community Engagement

5 CONVERGENCE OF REGULATORY EXPECTATIONS, REVIEW, AND APPROVAL

Coordination, Harmonization, and Convergence

6 MANUFACTURING, STOCKPILING, AND DISTRIBUTION

Policy Perspective

Manufacturing Capacity

Stockpiling Logistics

Supply Chain and Distribution

Forecasting Demand

7 CRITICAL CONSIDERATIONS FOR FACILITATING MEDICAL PRODUCT RESEARCH AND DEVELOPMENT

Defining the Priorities for Research and Development

Incentives

Discovery

Development

Regulations

Manufacturing

Distribution

8 CROSSCUTTING THEMES AND CLOSING REMARKS

Closing Remarks

APPENDIXES

A REFERENCES

B WORKSHOP STATEMENT OF TASK

C WORKSHOP AGENDA

D WORKSHOP SPEAKER BIOGRAPHIES

Acronyms and Abbreviations