International Regulatory Harmonization Amid
Globalization of Drug Development

Workshop Summary

Victoria Weisfeld and Tracy A. Lustig, Rapporteurs

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.

www.nap.edu

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NOTICE: The workshop that is the subject of this workshop summary 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.

This activity was supported by contracts between the National Academy of Sciences and the Department of Health and Human Services (HHSN26300023 [Under Base #HHSN263201200074I] and Contract No. N01-OD-4-2139 TO #276; HHSF22301026T [Under Base #HHSF223200810020I]), AbbVie Inc., American Diabetes Association, American Society for Microbiology, Amgen Inc., Association of American Medical Colleges, AstraZeneca, Bristol-Myers Squibb, Burroughs Wellcome Fund, Celtic Therapeutics, LLLP, Critical Path Institute, Doris Duke Charitable Foundation, Eli Lilly and Company, FasterCures, Friends of Cancer Research, GlaxoSmithKline, Johnson & Johnson, March of Dimes Foundation, Merck & Co., Inc., Novartis Pharmaceuticals Corporation, Pfizer Inc., and Sanofi. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the activity.

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Suggested citation: IOM (Institute of Medicine). 2013. International Regulatory Harmonization Amid Globalization of Drug Development: Workshop Summary. Washington, DC: The National Academies Press.

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OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

THE NATIONAL ACADEMIES

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PLANNING COMMITTEE FOR THE WORKSHOP ON INTERNATIONAL REGULATORY HARMONIZATION AMID GLOBALIZATION OF BIOMEDICAL RESEARCH AND MEDICAL PRODUCT DEVELOPMENT¹

THOMAS J. BOLLYKY (Co-Chair), Council on Foreign Relations, Washington, DC

STEVEN K. GALSON (Co-Chair), Amgen Inc., Thousand Oaks, CA

MICHAEL J. BRENAN, Aeras, Rockville, MD

RAYMOND CHUA, Singapore Health Sciences Authority

HANS-GEORG EICHLER, European Medicines Agency, London, UK

JAMES FITZGERALD, Pan American Health Organization, World Health Organization, Washington, DC

LYNN D. HUDSON, Critical Path Institute, Tucson, AZ

ANDREAS SEITER, World Bank, Washington, DC

ALICE E. TILL, PhRMA, Washington, DC

MARY LOU VALDEZ, Food and Drug Administration, Silver Spring, MD

JANET WOODCOCK, Food and Drug Administration, Rockville, MD

IOM Staff

ANNE B. CLAIBORNE, Forum Director

TRACY A. LUSTIG, Senior Program Officer

REBECCA A. ENGLISH, Associate Program Officer

ELIZABETH F. C. TYSON, Research Associate

ROBIN GUYSE, Senior Program Assistant

ANDREW M. POPE, Director, Board on Health Sciences Policy

___________________

¹ Institute of Medicine 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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FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION¹

JEFREY M. DRAZEN (Co-Chair), New England Journal of Medicine, Boston, MA

STEVEN K. GALSON (Co-Chair), Amgen Inc., Thousand Oaks, CA

RUSS BIAGIO ALTMAN, Stanford University, CA

MARGARET ANDERSON, FasterCures, Washington, DC

HUGH AUCHINCLOS, National Institute of Allergy and Infectious Diseases, Bethesda, MD

CHRISTOPHER P. AUSTIN, National Center for Advancing Translational Sciences, Bethesda, MD

ANN C. BONHAM, Association of American Medical Colleges, Washington, DC

LINDA BRADY, National Institute of Mental Health, Bethesda, MD

GAIL H. CASSELL, Harvard Medical School (Visiting), Carmel, IN

PETER B. CORR, Celtic Therapeutics, LLLP, New York, NY

ANDREW M. DAHLEM, Eli Lilly and Company, Indianapolis, IN

JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD

GARY L. FILERMAN, Atlas Health Foundation, McLean, VA

MARK J. GOLDBERGER, Abbott Pharmaceuticals, Rockville, MD

HARY B. GREENBERG, Stanford University School of Medicine, CA

PETER K. HONIG, AstraZeneca, Wilmington, PA

KATHY L. HUDSON, National Institutes of Health, Bethesda, MD

LYNN D. HUDSON, Critical Path Institute, Tucson, AZ

S. CLAIBORNE JOHNSTON, University of California, San Francisco

MICHAEL KATZ, March of Dimes Foundation, White Plains, NY

PETRA KAUFMANN, National Institute of Neurological Disorders and Stroke, Bethesda, MD

JACK D. KEENE, Duke University Medical Center, Durham, NC

RUSTY KELLEY, Burroughs Wellcome Fund, Research Triangle Park, NC

RONALD L. KRALL, University of Pennsylvania Center for Bioethics, Steamboat Springs, CO

FREDA C. LEWIS-HALL, Pfizer Inc., New York, NY

CAROL MIMURA, University of California, Berkeley

BERNARD H. MUNOS, InnoThink Center for Research in Biomedical Innovation, Indianapolis, IN

ELIZABETH (BETSY) MYERS, Doris Duke Charitable Foundation, New York, NY

___________________

¹ Institute of Medicine 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.

JOHN J. ORLOF, Novartis Pharmaceuticals Corporation, East Hanover, NJ

ROBERT E. RATNER, American Diabetes Association, Alexandria, VA

MICHAEL ROSENBLATT, Merck & Co., Inc., Whitehouse Station, NJ

JAMES S. SHANNON, GlaxoSmithKline, Brentford, Middlesex, UK

JANET SHOEMAKER, American Society for Microbiology, Washington, DC

ELLEN V. SIGAL, Friends of Cancer Research, Washington, DC

LANA R. SKIRBOLL, Sanofi, Washington, DC

BRIAN L. STROM, University of Pennsylvania Perelman School of Medicine, Philadelphia

JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine, New York, NY

JOANE WALDSTREICHER, Johnson & Johnson, New Brunswick, NJ

JANET WOODCOCK, Food and Drug Administration, Rockville, MD

IOM Staff

ANNE B. CLAIBORNE, Forum Director

REBECCA A. ENGLISH, Associate Program Officer

ELIZABETH F. C. TYSON, Research Associate

ROBIN GUYSE, Senior Program Assistant

ANDREW M. POPE, Director, Board on Health Sciences Policy

Reviewers

This workshop summary 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 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 Clyde J. Behney, Institute of Medicine. Appointed by the Institute of Medicine, 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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Contents

ACRONYMS

1 INTRODUCTION

Opening Remarks

International Harmonization: An Industry Perspective

International Harmonization: A Regulator’s Perspective

2 PRINCIPLES AND DEFINITIONAL CONSIDERATIONS

The Terminology Landscape and Options for Regulators

Standards Settings in the Context of Regulatory Harmonization

3 OVERVIEW OF THE CURENT GLOBAL REGULATORY LANDSCAPE

ICH

PANDRH

APEC Member Economies

Harmonization Initiatives in Africa

4 AREAS OF NEED FOR HARMONIZED STANDARDS AND BARRIERS TO PROGRES IN ADDRESING THE GAPS

Gaps from the Regulator’s Perspective

Gaps from the Perspective of Nongovernmental Organizations, Foundations, and Product Development Partnerships

Breakout Session Reports

5 CHARACTERISTICS OF HARMONIZED REGULATIONS AND REGULATORY STRUCTURES

Radiation Safety Standards

Reflections on the Experiences of WHO

6 FINDING SOLUTIONS: OPTIONS AND SYSTEMIC APPROACHES

Legal Framework

Cooperation and Collaboration

Mutual Trust and Recognition

Capacity Building

Business Case

Top-Level Support

Meta-Harmonization

Suggestions for Moving Forward

7 TACTICS AND STRATEGIES FOR A WAY FORWARD

Key Themes

Suggested Paths Forward

REFERENCES

APPENDIXES

A   WORKSHOP AGENDA

B   PARTICIPANT BIOGRAPHIES

Boxes

1-1 Statement of Task for the Workshop

1-2 Selected International Harmonization Efforts

5-1 Differing Units of Measurement

6-1 Examples from Other Industries

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Acronyms

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