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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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SAFE AND
EFFECTIVE
MEDICINES
FOR CHILDREN

Pediatric Studies Conducted Under the Best Pharmaceuticals
for Children Act and the Pediatric Research Equity Act

Committee on Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA)

Board on Health Sciences Policy

Marilyn J. Field and Thomas F. Boat, Editors

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS
Washington, D.C.
www.nap.edu

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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THE NATIONAL ACADEMIES PRESS     500 Fifth Street, NW     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. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance.

This study was supported by Contract No. DHHS-8598, TO #16, between the National Academy of Sciences and the Food and Drug Administration, U.S. Department of Health and Human Services. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the authors and do not necessarily reflect the views of the organizations or agencies that provided support for the project.

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Suggested citation: IOM (Institute of Medicine). 2012. Safe and effective medicines for children: Pediatric studies conducted under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

“Knowing is not enough; we must apply.
Willing is not enough; we must do.”

Goethe

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INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

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. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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COMMITTEE ON PEDIATRIC STUDIES
CONDUCTED UNDER BPCA AND PREA

THOMAS F. BOAT (Chair), Vice President for Health Affairs, Christian R. Holmes Professor and Dean of the College of Medicine, University of Cincinnati, Ohio

PETER C. ADAMSON, Professor of Pediatrics and Pharmacology at the University of Pennsylvania School of Medicine, Chief of the Division of Clinical Pharmacology and Therapeutics at The Children’s Hospital of Philadelphia, and Director of Clinical and Translational Research at The Children’s Hospital of Philadelphia Research Institute

RICHARD E. BEHRMAN, Consultant, Santa Barbara, California

F. SESSIONS COLE III, Park J. White, M.D., Professor of Pediatrics, Professor of Cell Biology and Physiology, Washington University School of Medicine, and Chief Medical Officer, St. Louis Children’s Hospital, Missouri

BRIAN FELDMAN, Professor of Pediatrics, Medicine, and Health Policy, Management and Evaluation and Professor of the Dalla Lana School of Public Health, University of Toronto, Canada

PAT FURLONG, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy, Middletown, Ohio

ERIC KODISH, Director of the Center for Ethics, Humanities and Spiritual Care at Cleveland Clinic, and F. J. O’Neill Professor and Chair of Bioethics and Professor of Pediatrics, Lerner College of Medicine of Case Western Reserve University, Ohio

JENNIFER LI, Professor of Pediatrics (Cardiology), Professor of Medicine (Cardiology), Director of Pediatric Clinical Research, Duke Clinical Research Institute; Core Director of Pediatrics, Duke Translational Medicine Institute; and Division Chief, Pediatric Cardiology, Duke University Health System, Durham, North Carolina

CHRISTINA M. MARKUS, Partner and Deputy Practice Leader, FDA & Life Sciences Group, King and Spalding LLP, Washington, DC

MILAP C. NAHATA, Division Chairman and Professor, College of Pharmacy, and Professor of Pediatrics and Internal Medicine, College of Medicine of the Ohio State University, Columbus

MARK A. RIDDLE, Professor of Psychiatry and Pediatrics and Director of the Children’s Interventions Research Program in Psychiatry, Johns Hopkins University School of Medicine, Baltimore, Maryland

JOSEPH W. ST. GEME III, James B. Duke Professor and Chair of Pediatrics and Professor of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, North Carolina

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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ROBERT WARD, Professor of Pediatrics and Medical Director of Pediatric Pharmacology, University of Utah, Salt Lake City

Committee Consultants and Background Paper Authors

CHARLES J. COTÉ, Professor of Anaesthesia, Harvard Medical School, Cambridge, Massachusetts

LARA ELLINGER, Department of Pharmacy Practice, College of Pharmacy, University of Illinois at Chicago

MICHAEL GABAY, Department of Pharmacy Practice, College of Pharmacy, University of Illinois at Chicago

ANDREW HERSHEY, Department of Neurology, Children’s Hospital Medical Center, Cincinnati, Ohio

MATTHEW M. LAUGHON, Department of Pediatrics, University of North Carolina at Chapel Hill

THE LEWIN GROUP, Falls Church, Virginia

P. BRIAN SMITH, Duke University Medical Center and Duke Clinical Research Institute, Durham, North Carolina

JOAN M. STACHNIK, Department of Pharmacy Practice, College of Pharmacy, University of Illinois at Chicago

IOM Staff

MARILYN J. FIELD, Senior Program Officer

CLAIRE F. GIAMMARIA, Research Associate

ROBIN E. PARSELL, Senior Program Assistant

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

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Acknowledgments

In preparing this report, the committee and project staff benefited greatly from the assistance and expertise of many individuals and groups. Important information and insights came from three public meetings that the committee organized to collect information and perspectives from officials from the Food and Drug Administration (FDA) and the National Institutes of Health and individuals from organizations representing pharmaceutical and biotechnology companies, pediatricians, researchers, and patient and family advocates. A number of speakers at these meetings, including Anne Zajicek, Natella Y. Rakhmanina, Samuel Maldonado, and Ronald Portman, also shared their knowledge at other times during the course of the study. Appendix A includes the agendas of the public meetings.

The committee appreciates the contributions of the authors of the background papers and analyses that appear as Appendixes B, C, and D. We likewise appreciate the analyses conducted by staff of The Lewin Group (including Nancy Walczak, Ian Glen, and Cynthia Schuster) and their patience in discussing the details of these analyses, which changed extensively over the course of the project. Consultants Andrew Hershey and Charles Coté assisted with the analysis of studies and labeling changes involving migraine and anesthetic products. At the College of Pharmacy, University of Illinois at Chicago, Amy LoDolce was very helpful in initiating the work on what became Appendix D of this report.

Robert Nelson, our project officer at FDA, and his colleague, Catherine Lee, provided information and clarification on a seemingly endless number of questions about FDA policies, procedures, and documents. They consulted with many others at FDA in the process, and we appreciate those

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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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who helped them answer our questions. Lisa Mathis and Julia Dunne at FDA also assisted staff in understanding additional aspects of FDA activities related to pediatric drug studies. At the American Academy of Pediatrics, Tamar Haro and Mark Del Monte provided helpful background information.

We also appreciate the work of copyeditor Michael Hayes and Debra Gilliam, Chanda Chay, and John Bowers at Caset Associates. Within the National Academies, we acknowledge the assistance of Adam Berger, Laura Harbold, Donna Randall, Vilija Teel, and Sarah Ziegenhorn, among many others.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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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 reports 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:

Jon S. Abramson, Wake Forest University School of Medicine

Marilee C. Allen, Johns Hopkins University School of Medicine

Daniel Benjamin, Duke Clinical Research Center

Susan S. Ellenberg, University of Pennsylvania School of Medicine

Chris Feudtner, Children’s Hospital of Philadelphia

Henry G. Grabowski, Duke University

Sean Hennessy, University of Pennsylvania School of Medicine

Raphael Hirsch, University of Pittsburgh School of Medicine

Steven Joffe, Dana-Farber Cancer Institute

Michael Katz, March of Dimes Foundation

Michael Labson, Covington & Burling LLP

Fernando D. Martinez, Arizona Health Sciences Center

Josef Neu, University of Florida

Arthur W. Nienhuis, St. Jude Children’s Research Hospital

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Alastair J. Wood, Symphony Capital LLC

Kathryn C. Zoon, National Institute of Allergy and Infectious Diseases

Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations, nor did they see the final draft of the report before its release. The review of this report was overseen by Ellen Wright Clayton, Vanderbilt University, and Charles E. Phelps, University of Rochester. Appointed by the National Research Council and the Institute of Medicine, these individuals were responsible for making certain that an independent examination of this report was carried out in accordance with the 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.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Preface

Children, in general, are healthier than their adult counterparts, particularly as adults reach the fifth decade of life and beyond. However, children do have multiple acute illnesses each year, and a substantial number of children, often estimated to be 20 percent or more, are burdened with chronic health disorders, some of them disabling or life threatening. Medical attention, including evidence-based prescription of drugs or biologics, is vital for their well-being.

In addition, children constitute a smaller percentage of the U.S. population than adults, so drugs are often designed for adults and initially tested and approved for use in adult populations. Clinicians, however, often begin to use these drugs—as is legal—with children without guidance from well-controlled clinical studies. Over time it has become apparent that pharmacologically, as well as in many other ways, children are not “small adults.” In the 1980s and 1990s, policy makers, pediatricians, and others increasingly recognized the need to study the efficacy and safety of drugs in children. Key responses to that recognition—different policies that incentivize or require studies of drugs in children—are the focus of this report. The Best Pharmaceuticals for Children Act (BPCA) provides incentives for drug studies in children, and the Pediatric Research Equity Act (PREA) requires such studies in certain situations. Since the late 1990s, these policies (and their predecessors) have improved the availability of reliable information, which should, in turn, improve the appropriate use of therapeutic agents for children in clinical practice.

This Institute of Medicine (IOM) report, which was called for by Congress, documents improvements in the availability of evidence about

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

the safety and efficacy of drugs in children following the adoption of these policies and their implementation by the Food and Drug Administration (FDA). It reflects the work of an IOM committee, representing a wide range of relevant expertise, that worked diligently for more than a year to collect data on pediatric studies conducted under BPCA and PREA and to assess those data. The members of the committee engaged in lively debates and, in the end, came to conclusions that we believe will contribute to understanding and improving these policies and the pediatric studies prompted by them. For much of its work, the committee primarily relied on documents that were either posted on the FDA website (mostly documents issued after September 27, 2007) or supplied over a period of months by FDA after redaction (mostly documents issued earlier, before Congress required that they be made public).

Committee members pored through hundreds of pages of written requests and FDA clinical and other reviews to extract pertinent information. Thus, unlike many IOM committees, members both created and analyzed the data necessary to reach important conclusions. Also, unlike many other IOM committees, our committee was not asked to make recommendations, with one exception related to recently enacted policies to provide incentives for pediatric studies of biologics. The report was therefore constructed to transmit the conclusions of the committee’s assessments of studies under BPCA and PREA, as well as conclusions from these assessments that might form the basis for future steps by FDA and Congress to build on the strengths and correct some of the shortcomings of these policies or their application.

The committee assessed the data from a spectrum of perspectives: pediatric, psychiatric, pharmacologic, ethical, legal, health policy, and consumer. The committee was assisted in this effort by a number of consultants and contributors to the task of assembling data for this review and sharing fresh insights. Importantly, the committee would like to recognize and express appreciation for the tireless leadership of our committee study director, Marilyn Field, and for the contributions of her staff colleagues, Claire Giammaria and Robin Parsell. It was their efforts that allowed the committee to evaluate and come to conclusions based on an enormous array of data. Above all, the committee hopes that its efforts will encourage ongoing scientifically and ethically sound study of drugs and biologics, particularly for children who are not yet advantaged by therapies demonstrated to be safe and effective for their medical conditions.

Thomas F. Boat, Chair
Committee on Pediatric Studies Conducted Under BPCA and PREA

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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6-3   Criteria for Selecting Drugs for Priority Investigation in Newborns

7-1   Major Amendments to Written Requests for Pediatric Studies of Drugs for Treatment of Migraine

7-2   Elements in Written Requests That Could Limit the Potential of Studies to Yield Useful Information

7-3   Aspects of Studies as Planned or Executed That May Have Limited the Usefulness of Information Submitted

7-4   Examples of Informative Labeling Changes

7-5   Concerns About Clarity of Labeling Changes

8-1   Examples of Products with PREA Waivers or Orphan Designation Exemptions for Which Pediatric Studies Are Listed at ClinicalTrials.gov

8-2   Products with No Indication of Pediatric Studies in Labeling, FDA Approval Letters, or Clinical Trials Registry (ClinicalTrials.gov)

FIGURES

5-1   Use of extrapolation to support pediatric efficacy claims

7-1   Changes in drug labeling associated with BPCA, PREA (including the Pediatric Rule), or both, July 1998 through October 2011

7-2   Number of written requests (WR) issued and number of grants of exclusivity, by year, July 1998 through September 2011

B-1   Preferred sources of new dosing information

C-1   Structures of nitroglycerin (C3H5N3O9), a conventional drug, and alteplase, a recombinant form of human tissue plasminogen activator

E-1   Trial design options for pediatric hypertension trials provided for by FDA written requests

TABLES

1-1   Historical Data on Drugs Without Adequate Labeling for Pediatric Use

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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3-1   Underlying Patent or Exclusivity Incentives That Can Be Extended with Pediatric Exclusivity

3-2   Reasons for Waiver of Pediatric Assessment Requirements Authorized Under PREA with Examples from Recent NDA or BLA Approvals

3-3   Selected Public Information Requirements of BPCA and PREA Through 2007 Reauthorization

5-1   Summary of PAC Recommendations or Actions from the Safety Review 1 Year After a Labeling Change Resulting from a Study Conducted Under BPCA or PREA, June 1, 2003, to June 30, 2011

5-2   FDA Analysis of Use of Extrapolation of Efficacy from Adult to Pediatric Population, Studies Conducted Under BPCA, 1998 to 2009

5-3   Use of Extrapolation in IOM Sample of BPCA and PREA Labeling Changes

6-1   Therapeutics Commonly Used in Neonatal Intensive Care

6-2   Labeling Changes for Drugs for Treatment of HIV Infection from Studies That Included Neonates

6-3   Written Requests for Neonatal Drug Studies Referred by FDA to NIH, by Patent Status and Study Status

ADDENDUM Labeling Changes Based on Neonate Studies Requested Under BPCA or Required Under PREA, July 1998 Through December 2010

7-1   Progress of Pediatric Studies Deferred Under PREA, 2007 to 2010

7-2   Types of Pediatric Studies for Labeling Changes Conducted Under BPCA and PREA Between September 27, 2007, and June 30, 2011

8-1   Summary Information on Biologics Studied in Children

B-1   Infant Dosing Compared with Adult Dosing of Commonly Used Antimicrobials for Bloodstream Infections

B-2   Sources for Prescribing Information for Clinicians

B-3   Recent Pediatric Labeling Changes Identified by FDA and Comparison to Commonly Used Resources

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Abbreviations and Acronyms

AAP      

American Academy of Pediatrics

ACR      

American College of Rheumatology

ADHD      

attention deficit hyperactivity disorder

AERS      

Adverse Event Reporting System

AHA      

American Heart Association

AHRQ      

Agency for Healthcare Research and Quality

                 
BLA      

Biologics License Application

BPCA      

Best Pharmaceuticals for Children Act

BPCIA      

Biologics Price Competition and Innovation Act

                 
CBER      

Center for Biologics Evaluation and Research

CDC      

Centers for Disease Control and Prevention

CDER      

Center for Drug Evaluation and Research

CDRH      

Center for Devices and Radiological Health

CNS      

central nervous system

COG      

Children’s Oncology Group

CYP      

cytochrome P450

                 
DESI      

Drug Efficacy Study Implementation

DMC      

data monitoring committee

DSI      

Division of Scientific Investigations of the Center for Drug Evaluation and Research

                 
EMA      

European Medicines Agency

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EU      

European Union

                 
FDA      

Food and Drug Administration

FDAAA      

Food and Drug Administration Amendments Act of 2007

FDAMA      

Food and Drug Administration Modernization and Accountability Act of 1997

FDC Act      

Federal Food, Drug, and Cosmetic Act of 1938

FEV1      

forced expiratory volume in 1 second

FOIA      

Freedom of Information Act

                 
GCP      

good clinical practice

GERD      

gastroesophageal reflux disease

                 
HHS      

U.S. Department of Health and Human Services

                 
IBD      

inflammatory bowel disease

IGIV      

immune globulin intravenous

IM      

intramuscular

IND      

Investigational New Drug

IOM      

Institute of Medicine

IRB      

institutional review board

IV      

intravenous

                 
JIA      

juvenile idiopathic arthritis

                 
NDA      

New Drug Application

NICHD      

National Institute of Child Health and Human Development

NIH      

National Institutes of Health

NME      

new molecular entity

NRC      

National Research Council

                 
PAC      

Pediatric Advisory Committee

PASI 75      

75 percent or greater improvement from baseline in the psoriasis area and severity index

PD      

pharmacodynamics

PDCO      

European Union Pediatric Committee

PDR      

Physicians’ Desk Reference

PeRC      

Pediatric Review Committee

PHS      

Public Health Service

PIP      

pediatric investigation plan

PK      

pharmacokinetics

PPI      

proton pump inhibitor

PREA      

Pediatric Research Equity Act

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SC      

subcutaneous

                 
TNF      

tumor necrosis factor

                 
UKHCDO      

United Kingdom Hemophilia Center Doctors’ Organization

UNC      

University of North Carolina

                 
WHO      

World Health Organization

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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Page xviii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Page xxii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Page xxiii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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Page xxiv Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
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The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.

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