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.
THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001
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.
International Standard Book Number-13: 978-0-309-28479-0
International Standard Book Number-10: 0-309-28479-1
Additional copies of this workshop summary 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.
For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu.
Copyright 2013 by the National Academy of Sciences. All rights reserved.
Printed in the United States of America
The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and religions since the beginning of recorded history. The serpent adopted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin.
Suggested citation: IOM (Institute of Medicine). 2013. International Regulatory Harmonization Amid Globalization of Drug Development: Workshop Summary. Washington, DC: The National Academies Press.
THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
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. C. D. Mote, Jr., 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. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council.
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.
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:
Tobias Massa, Bristol-Myers Squibb Company
John Purves, EPCA Ltd
Linda Rosenstock, University of California, Los Angeles
Nathalie Strub Wourgaft, Drugs for Neglected Diseases initiative
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.
Contents
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
Harmonization Initiatives in Africa
4 AREAS OF NEED FOR HARMONIZED STANDARDS AND BARRIERS TO PROGRES IN ADDRESING THE GAPS
| AMRH | African Medicines Regulatory Harmonization |
| APEC | Asia-Pacific Economic Cooperation |
| AU | African Union |
| AVAREF | African Vaccine Regulatory Forum |
| BMGF | Bill & Melinda Gates Foundation |
| CARICOM | Caribbean Community |
| CDER | Center for Drug Evaluation and Research |
| CHMP | Committee for Medicinal Products for Human Use (EMA) |
| CIRS | Centre for Innovation in Regulatory Science |
| CMC | Chemistry, Manufacturing and Controls |
| C-Path | Critical Path Institute |
| DNDi | Drugs for Neglected Diseases initiative |
| EAC | East African Community |
| EFPIA | European Federation of Pharmaceutical Industries and Associations |
| EMA | European Medicines Agency |
| EU | European Union |
| FDA | U.S. Food and Drug Administration |
| FDASIA | U.S. Food and Drug Administration Safety and Innovation Act |
| GATT | General Agreement on Tariffs and Trade |
| GCG | Global Cooperation Group (ICH) |
| IAEA | International Atomic Energy Agency |
| ICAO | International Civil Aviation Organization |
| ICH | International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use |
| IOM | Institute of Medicine |
| ISO | International Organization for Standardization |
| JPMA | Japanese Pharmaceutical Manufacturers Association |
| MERCOSUR | Mercado Común del Sur (the Southern Common Market, South America) |
| MHLW | Ministry of Health, Labor, and Welfare (Japan) |
| MS | multiple sclerosis |
| NAFTA | North American Free Trade Agreement |
| NDA | new drug application |
| NEPAD | New Partnership for Africa’s Development |
| NRC | U.S. Nuclear Regulatory Commission |
| PAHO | Pan American Health Organization |
| PANDRH | Pan American Network for Drug Regulatory Harmonization |
| PhRMA | Pharmaceutical Research and Manufacturers of America |
| PIC/S | Pharmaceutical Inspection Convention and Pharmaceutical Inspection Co-operation Scheme |
| PMDA | Pharmaceuticals and Medical Devices Agency (Japan) |
| QBD | Quality By Design |
| RHSC | Regulatory Harmonization Steering Committee (APEC) |
| SICA | Sistema de la Integración Centroamericana (the Central American Integration System) |
