National Academies Press: OpenBook
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

Building a National Framework for the Establishment of REGULATORY SCIENCE FOR DRUG DEVELOPMENT

Workshop Summary

Yeonwoo Lebovitz, Rebecca A. English, and Anne B. Claiborne, 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

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

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.

This project was supported by the Department of Health and Human Services (Contract Nos. N01-OD-4-2139 and 223001003T), the American Diabetes Association, the American Society for Microbiology, Amgen Inc., the Association of American Medical Colleges, Bristol-Myers Squibb, the Burroughs Wellcome Fund, Celtic Therapeutics, LLLP, the Critical Path Institute, the Doris Duke Charitable Foundation, Eli Lilly & Co., GlaxoSmithKline, Johnson & Johnson, Novartis Pharmaceuticals Corporation, and Pfizer, Inc. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the author(s) and do not necessarily reflect the view of the organizations or agencies that provided support for this project.

International Standard Book Number-13: 978-0-309-15889-3

International Standard Book Number-10: 0-309-15889-3

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 2011 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). 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press.

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

“Knowing is not enough; we must apply.

Willing is not enough; we must do.”

—Goethe

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES


Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

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. 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

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1

Gail H. Cassell (Co-Chair),

Eli Lilly and Company, Indiana

Jeffrey M. Drazen (Co-Chair),

New England Journal of Medicine, Massachusetts

Barbara Alving,

National Center for Research Resources, Maryland

Leslie Z. Benet,

University of California-San Francisco, California

Ann Bonham,

Association of American Medical Colleges, Washington, DC

Linda Brady,

National Institute of Mental Health, Maryland

Robert M. Califf,

Duke University Medical Center, North Carolina

Scott Campbell,

Foundation for the National Institutes of Health, Maryland

C. Thomas Caskey,

University of Texas-Houston Health Science Center, Texas

Peter B. Corr,

Celtic Therapeutics, LLLP, New York

James H. Doroshow,

National Cancer Institute, Maryland

Paul R. Eisenberg,

Amgen Inc., California

Gary L. Filerman,

Atlas Research, Washington, DC

Garret A. FitzGerald,

University of Pennsylvania School of Medicine, Pennsylvania

Elaine K. Gallin,

The Doris Duke Charitable Foundation, New York

Steven K. Galson,2

Amgen Inc., California

Harry B. Greenberg,

Stanford University School of Medicine, California

Stephen Groft,

National Institutes of Health, Maryland

Peter K. Honig,

AstraZeneca, Delaware

Annalisa Jenkins,

Bristol-Myers Squibb, New Jersey

Michael Katz,

March of Dimes Foundation, New York

Jack D. Keene,

Duke University Medical Center, North Carolina

Ronald L. Krall,

GlaxoSmithKline (retired), Colorado

Freda Lewis-Hall,

Pfizer, Inc., New York

William D. Matthew,

National Institute of Neurological Disorders and Stroke, Maryland

Mark B. McClellan,

Brookings Institution, Washington, DC

Carol Mimura,

University of California-Berkeley, California

John Orloff,

Novartis Pharmaceuticals Corporation, New Jersey

Amy P. Patterson,

National Institutes of Health, Maryland

1

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.

2

Steven K. Galson was with Science Applications International Corporation until September 30, 2010.

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

Janet Shoemaker,

American Society for Microbiology, Washington, DC

Ellen V. Sigal,

Friends of Cancer Research, Virginia

Nancy S. Sung,

Burroughs Wellcome Fund, North Carolina

Jorge A. Tavel,

National Institute of Allergy and Infectious Diseases, Maryland

Janet Tobias,

Ikana Media, New York

Joanne Waldstreicher,

Johnson & Johnson, New Jersey

Janet Woodcock,

U.S. Food and Drug Administration, Maryland

Raymond L. Woosley,

The Critical Path Institute, Arizona

IOM Staff

Anne B. Claiborne, Director (from April 5, 2010)

Robert B. Giffin, Director (until February 26, 2010)

Rebecca A. English, Research Associate

Yeonwoo Lebovitz, Program Associate

Genea S. Vincent, Senior Program Assistant

Rona Briere, Consulting Editor

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

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 published report as sound as possible and to ensure that the report 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 report:

STEVEN K. GALSON,1 Amgen Inc., California

PETER K. HONIG, AstraZeneca

CARL C. PECK, University of California, San Francisco

ELLEN V. SIGAL, Friends of Cancer Research

Although the reviewers listed above have provided many constructive comments and suggestions, they did not endorse the final draft of the report before its release. The review of this report was overseen by LESLIE Z. BENET, University of California, San Francisco. Appointed by the National Research Council and the Institute of Medicine, he was

1

Steven K. Galson was with Science Applications International Corporation until September 30, 2010.

Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

responsible for making certain that an independent examination of this report was carried 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 authors and the institution.

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

Figures and Boxes

FIGURES

3-1

 

Graphic presentation of the effect of acetaminophen on various mouse gene strains,

 

16

4-1

 

Breadth of FDA responsibilities by number of establishments as of 2007,

 

24

4-2

 

Flow of data in an Incubator for Innovation in Regulatory and Information Science (IIRIS)/Centers-of-Excellence (COE) model,

 

27

5-1

 

Visualization of how the current drug development model is growing disaggregated with involvement of various sectors to reduce risk in innovation,

 

32

5-2

 

Centers of Excellence in Regulatory Science Network,

 

35

6-1

 

Shifting opinions on America’s most important health issue, December 2003–February 2010,

 

41

6-2

 

Americans’ level of confidence in systems for monitoring the effectiveness and safety of new medicines and medical devices,

 

42

6-3

 

Americans’ views on FDA’s most important role,

 

42

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

BOXES

2-1

 

Some definitions of regulatory science,

 

6

2-2

 

Excerpts from the Commissioner’s speech,

 

8

3-1

 

Potential contributions of regulatory science to cancer therapy,

 

14

3-2

 

Hamner Institute’s study of inbred mice panels,

 

16

4-1

 

The IIRIS model,

 

28

5-1

 

Collaborative models in oncology,

 

34

6-1

 

The impact of patient advocacy for cancer therapeutics at FDA,

 

44

Page xiii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

Acronyms

AHRQ Agency for Healthcare Research and Quality

AIDS acquired immune deficiency syndrome

ARRA American Recovery and Reinvestment Act of 2009

BC Biomarkers Consortium

CBER Center for Biologics Evaluation and Research

CDC Centers for Disease Control and Prevention

CDER Center for Drug Evaluation and Research

CERTS Centers for Education and Research on Therapeutics

COE Centers of Excellence

COERS Centers of Excellence in Regulatory Science Network

CPI Critical Path Initiative

CTTI Clinical Trials Transformative Initiative

DARPA Defense Advanced Research Products Agency

FDA Food and Drug Administration

FDAAA FDA Amendments Act of 2007

FDAMA FDA Modernization Act of 1997

FNIH Foundation for the National Institutes of Health

GAO Government Accountability Office

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

HHS Department of Health and Human Services

HIV human immunodeficiency virus

ICT-21 Information Communication Technology for the 21st Century

IIRIS Incubator for Innovation in Regulatory and Information Science

IND Investigative New Drug Application

IOM Institute of Medicine

IOTF NCI–FDA Interagency Oncology Task Force

IT information technology

NCI National Cancer Institute

NDA New Drug Application

NIH National Institutes of Health

NIST National Institute of Standards and Technology

OMOP Observational Medical Outcomes Partnership

OODP Office of Oncology Drug Products

PDUFA Prescription Drug User Fee Act

PhRMA Pharmaceutical Research and Manufacturers of America

SAE serious adverse event

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

Preface

Regulatory agencies worldwide are tasked with a difficult mandate to provide large populations with efficient access to drugs that are both safe and effective. They must also balance this task while adapting to new technologies in medicine and information technology. These demands are further complicated by the increasing globalization of drug development and regulatory relationships. These challenges, along with problems with contaminated supplies, serious adverse events, and other compounding issues that accompany changes in administration, contribute to the over-burdening of a regulatory system whose resources have not increased along with its demands. We urgently need to ensure that our regulatory bodies have the autonomy, resources, and scientific support needed to function effectively to promote public health and safety.

The U.S. Food and Drug Administration (FDA) is a science-based agency responsible for regulating 80 percent of Americans’ consumable goods and a quarter of the U.S. economy. It is imperative that every one of FDA’s regulatory decisions be based on the best scientific evidence. Unfortunately, this has not always been possible due to several factors, including—but not limited to—inadequate human capital, leadership support, and funding. Above all, a weakening science base at the agency has threatened its ability to support its core regulatory functions and decisions. FDA needs to resolve this gap between scientific and regulatory decision making to ensure continuity of its duties in an environment of heightened public scrutiny on drug safety and rapid scientific advancements.

The public increasingly recognizes the importance of regulatory science. The notion of basing regulatory decisions on the best scientific knowledge

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×

available is not new, but never before have funds been deliberately appropriated for the accomplishment of this task. FDA’s 2011 budget proposal includes $25 million specifically allocated to the building of a regulatory science infrastructure at the agency. In anticipation of this event and as an acknowledgment of the “reform-ready” political atmosphere, the Institute of Medicine’s Forum on Drug Discovery, Development, and Translation held a public workshop with the following goals in mind:

  • Establish a clear definition of regulatory science, and engage the public and the policy community in a discussion of its challenges and opportunities.

  • Increase awareness of inadequate funding for regulatory science and the impact on the development of new therapies on patients.

  • Articulate priorities and strategies for building or rebuilding the infrastructure for regulatory science.

The one-day workshop featured leaders from government, such as Representative Rosa DeLauro (D-CT); Department of Health and Human Services Deputy Secretary, William Corr; and FDA Commissioner, Margaret Hamburg. Speakers from academia, industry, and patient advocacy groups provided a variety of perspectives and illuminated examples of the urgent need of a regulatory science infrastructure. Leaders in emerging technologies, such as genomics, biostatistics, and information technology—whose fields of study are influenced daily by regulatory decisions—deliberated on potential consequences arising from the failure to establish a robust scientific base at the agency level. In addition, moderated panels considered mechanisms for building a regulatory science infrastructure at FDA, analyzed existing implementation models, and posed strategies for engaging the public and policy makers.

This was a timely workshop that provided a valuable opportunity to bring together a diverse group for thoughtful discussion about improving drug development and strengthening regulatory science. I would like to thank all of the individuals—speakers, moderators, and panelists—who contributed to and participated in the workshop. In particular, I would like to thank a small team of participants in the workshop who spent many hours in advance of the workshop discussing the merits of Centers of Excellence in Regulatory Science and the critical characteristics of these centers including: K. Ahlport, M. Anderson, L. Benet, R. Califf, G. FitzGerald, S. Kim, J. Kramer, R. Nerem, D. Nordenberg, M. Osterholm, K. Schneeman, J. Shoemaker, E. Sigal, N. Sung, and J. Tobias. I would also like to thank the members of the Forum and Forum staff for their dedication and commitment to developing and executing this workshop.


Gail H. Cassell, Co-Chair

Forum on Drug Discovery, Development, and Translation

Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R1
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R2
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R3
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R4
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R5
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R6
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R7
Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R8
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R9
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R10
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R11
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R12
Page xiii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R13
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R14
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R15
Suggested Citation:"Front Matter." Institute of Medicine. 2011. Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/12968.
×
Page R16
Next: 1 Introduction »
Building a National Framework for the Establishment of Regulatory Science for Drug Development: Workshop Summary Get This Book
×
Buy Paperback | $29.00 Buy Ebook | $23.99
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

The Food and Drug Administration (FDA) is tasked with ensuring the safety and effectiveness of medicine. FDA's science base must be strong enough to make certain that regulatory decisions are based on the best scientific evidence. The IOM held a public workshop on February 26, 2010, to examine the state of regulatory science and to consider approaches for enhancing it.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    Switch between the Original Pages, where you can read the report as it appeared in print, and Text Pages for the web version, where you can highlight and search the text.

    « Back Next »
  6. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  7. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  8. ×

    View our suggested citation for this chapter.

    « Back Next »
  9. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!