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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
ADDRESSING THE BARRIERS TO PEDIATRIC DRUG DEVELOPMENT
Workshop Summary
Cori Vanchieri, Adrienne Stith Butler, and Andrea Knutsen, 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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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
THE NATIONAL ACADEMIES PRESS
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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.
Support for this project was provided by the Department of Health and Human Services (Contract Nos. 223-01-2460, HHSP233200600500P, HHSPP23320042509X1, and N01-OD-4-2139); the Department of Veterans Affairs (Contract No. V101(93)P-2136); Abbott Laboratories; American Diabetes Association; American Society for Microbiology; Amgen, Inc.; Association of American Medical Colleges; AstraZeneca Pharmaceuticals; Blue Cross Blue Shield Association; Burroughs Wellcome Fund; Doris Duke Charitable Foundation; Eli Lilly and Company; Entelos, Inc.; Genentech; GlaxoSmithKline; Johnson & Johnson; March of Dimes Foundation; Merck and Company; Pfizer, Inc.; Schering-Plough Research Institute; and UnitedHealth Foundation. 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: 978-0-309-10742-6
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Additional copies of this report are available from the
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Copyright 2008 by the National Academy of Sciences. All rights reserved.
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Suggested citation: IOM (Institute of Medicine). 2008. Addressing the barriers to pediatric drug development: Workshop summary. Washington, DC: The National Academies Press.
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.
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
“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.
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
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
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION
Gail H. Cassell (Co-Chair, 1/1/2005–12/31/2008),
Eli Lilly and Company
Jeffrey M. Drazen (Co-Chair, 7/1/2008–12/31/2008; Member, 1/1/2005–6/30/2008),
New England Journal of Medicine
Edward W. Holmes (Co-Chair, 1/1/2005–6/30/2008; Member, 7/1/2008–12/31/2008),
National University of Singapore
Barbara M. Alving (4/10/2008–12/31/2009),
National Center for Research Resources
Naomi Aronson (1/9/2007–12/31/2008),
Blue Cross and Blue Shield Association
Hal Barron (8/1/2008–12/31/2008),
Genentech, Inc.
Leslie Z. Benet (1/1/2005–12/31/2008),
University of California, San Francisco
Nina Bhardwaj (1/1/2005–9/7/2006),
New York University School of Medicine
Catherine Bonuccelli (1/9/2007–12/31/2008),
AstraZeneca Pharmaceuticals
Linda Brady (1/9/2007–12/31/2008),
National Institute of Mental Health
Robert M. Califf (1/1/2005–12/31/2008),
Duke University Medical Center
Scott Campbell (1/1/2005–12/31/2008),
American Diabetes Association
C. Thomas Caskey (1/1/2005–12/31/2008),
The University of Texas-Houston Health Science Center
Francis D. Chesley, Jr. (1/9/2007–10/25/2007),
Agency for Healthcare Research and Quality
Mark Clanton (1/1/2005–12/31/2006),
National Cancer Institute
Peter Corr (1/9/2007–12/31/2008),
Celtic Therapeutics
James H. Doroshow (7/19/2007–12/31/2008),
National Cancer Institute
William E. Evans (1/1/2005–12/31/2006),
St. Jude Children’s Research Hospital
Joseph M. Feczko (1/9/2007–12/31/2008),
Pfizer, Inc.
Wayne Fenton [Deceased] (1/1/2005–9/3/2006),
National Institute of Mental Health
Garret A. FitzGerald (1/1/2005–12/31/2008),
University of Pennsylvania School of Medicine
Elaine K. Gallin (1/1/2005–12/31/2008),
The Doris Duke Charitable Foundation
Steven K. Galson (1/9/2007–12/31/2008),
Office of the Surgeon General
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
Alan M. Garber (1/1/2005–12/31/2007),
Stanford University
Robert L. Garnick (11/16/2007–2/28/2008),
Genentech, Inc.
Mikhail Gishizky (1/1/2005–12/31/2008),
Entelos, Inc.
Stephen Groft (1/1/2005–12/31/2008),
National Institutes of Health
Carole A. Heilman (1/1/2005–3/13/2006),
National Institute of Allergy and Infectious Diseases
Peter K. Honig (7/19/2007–12/31/2008),
Merck & Co., Inc.
Dale Hu (1/1/2005–12/31/2005),
Centers for Disease Control and Prevention
Richard A. Justman (7/19/2007–12/31/2008),
United HealthGroup
Michael Katz (1/1/2005–12/31/2008),
March of Dimes Foundation
William F. Keane (1/1/2005–7/3/2007),
Merck & Co., Inc.
Chaitan Khosla (1/1/2005–12/31/2006),
Stanford University
Antonia Kolokathis (1/1/2005–12/31/2005),
Pfizer, Inc.
Allan M. Korn (1/1/2005–12/31/2006),
Blue Cross and Blue Shield Association
David Korn (1/1/2005–12/31/2008),
Association of American Medical Colleges
Ronald L. Krall (1/9/2007–12/31/2008),
GlaxoSmithKline
Jeffrey M. Leiden (1/1/2005–12/31/2007),
Clarus Ventures
John M. Leonard (1/9/2007–7/5/2007),
Abbott Laboratories
Nancy Loving (1/1/2005–6/7/2006),
National Coalition for Women with Heart Disease
John R. Marler (1/1/2005–12/31/2008),
National Institute of Neurological Disorders and Stroke
Musa Mayer (1/1/2005–12/31/2008),
AdvancedBC.org
Mark B. McClellan (4/2/2007–12/31/2008),
The Brookings Institution
Garry A. Neil (1/1/2005–3/19/2007),
Johnson & Johnson
Joshua J. Ofman (1/1/2005–12/31/2008),
Amgen, Inc.
Suzanne R. Pattee (1/1/2005–12/31/2008),
Cystic Fibrosis Foundation
Cecil B. Pickett (1/1/2005–12/31/2006),
Schering-Plough Research Institute
Joanne L. Rhoads (1/9/2007–12/31/2008),
National Institute of Allergy and Infectious Diseases [Retired]
Todd Rich (3/2/2008–12/31/2008),
Genentech, Inc.
Brian Schuster (1/1/2005–12/31/2006),
U.S. Department of Veterans Affairs
B. A. Schwetz (1/1/2005–9/30/2007),
U.S. Department of Health and Human Services
Janet Shoemaker (1/1/2005–12/31/2008),
American Society for Microbiology
Lana Skirboll (1/9/2007–12/31/2008),
National Institutes of Health
Nancy S. Sung (1/1/2005–12/31/2008),
Burroughs Wellcome Fund
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
James R. Swartz (1/1/2005–12/31/2005),
Stanford University
Jorge A. Tavel (7/19/2007–12/31/2008),
National Institute of Allergy and Infectious Diseases
Reed V. Tuckson (1/1/2005–7/3/2007),
UnitedHealth Group
Sean Tunis (1/1/2005–12/31/2006),
Centers for Medicare & Medicaid Services
Janet Woodcock (1/1/2005–12/31/2008),
U.S. Food and Drug Administration
IOM Stafff
Robert B. Giffin (Director, 7/13/2006–present)
Alexander K. Ommaya (Director, 1/1/2005–7/7/2006)
Adrienne Stith Butler (Senior Program Officer, 12/1/2006–5/31/2007)
Sally Robinson (Program Officer, 12/18/2006–present)
Heather Begg (Program Associate, 1/1/2005–7/7/2006)
Andrea Knutsen (Senior Program Assistant, 10/3/2005–present)
Jennifer Rainey (Research Assistant, 1/1/2005–7/7/2006)
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
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 deliberative process.
We wish to thank the following individuals for their review of this report:
Marion E. Broome, Indiana University School of Nursing and Nursing Outlook Journal
P. Joan Chesney, St. Jude Children’s Research Hospital
Thomas G. Dewitt, Cincinnati Children’s Hospital Medical Center
William E. Evans, St. Jude Children’s Research Hospital
Michael Katz, March of Dimes Birth Defects Foundation
John S. March, Department of Psychiatry and Behavioral Science, Duke University
Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the report before its release. The review of this report was overseen
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
by Dr. Ralph E. Kauffman, Professor Emeritus, University of Missouri-Kansas City. Appointed by the Institute of Medicine, he was 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 authoring committee and the institution.
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
Contents
Summary
1
1
Introduction
7
Response to Drugs in Various Age Groups,
7
Product Labeling,
8
Organization of This Summary,
9
2
Regulatory Framework
10
Best Pharmaceuticals for Children Act,
12
Pediatric Research Equity Act,
14
Impact of Pediatric Drug Legislation,
16
Discussion,
19
3
Current Challenges in Developing and Prescribing Drugs for Children
20
Barriers to Pediatric Drug Development,
20
Formulations,
26
Dosing, Bioavailability, and Drug Response,
28
4
Models for Enhancing Pediatric Drug Development
29
Vaccine Development in the United States,
29
The European Union’s New Regulatory Approach,
31
The St. Jude’s Model for Pediatric Oncology Drugs,
34
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Addressing the Barriers to Pediatric Drug Development: Workshop Summary
5
Challenges and Opportunities for the Future
36
Systemic Solutions,
36
Elimination of Economic Barriers,
38
Concluding Thoughts,
39
References
41
Appendixes
A
Workshop Agenda
43
B
Speaker Biographies
46
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