PEDIATRIC DRUG RESEARCH AND THE FDA

PEDIATRIC DRUG RESEARCH AND THE FDA

Library of Congress Cataloging-in-Publication Data

CONTENTS

Chapter 1: FDA’S AUTHORITY TO ENSURE THAT DRUGS PRESCRIBED TO CHILDREN ARE SAFE AND EFFECTIVE

SUMMARY

INTRODUCTION

UNDERSTANDING DRUG EFFECTS IN CHILDREN

WHY MANUFACTURERS HAVE NOT TESTED MOST DRUGS IN CHILDREN

BPCA AND PREA: LAWS TO ENCOURAGE PEDIATRIC DRUG RESEARCH

Best Pharmaceuticals for Children Act

Pediatric Marketing Exclusivity

FDA-NIH Collaboration

Off-Patent Products

Manufacturer-Declined Studies

Other Provisions

Pediatric Research Equity Act

New Applications

Products on the Market

Other Provisions

ISSUES FOR REAUTHORIZATION OF BPCA AND PREA

Relationship between BPCA and PREA

Examining the Need for Pediatric Market Exclusivity

Examining the Need for Permanent PREA Authorization

Costs and Benefits of Pediatric Marketing Exclusivity

Labeling

Current Pediatric Labeling Requirements

Questions for Congressional Consideration

Measuring the Impact on Pediatric Drug Research

Enforcement

CONCLUDING COMMENTS

APPENDIX A. ACRONYMS

APPENDIX B. CURRENT LAW EVOLVED FROM EARLIER ATTEMPTS

Rule on Drug Labeling: 1979

Revised Rule: 1994

Food and Drug Administration Modernization Act of 1997

Pediatric Rule: Proposed 1997, Finalized 1998, Effective 1999-2002

End Notes

Chapter 2: PEDIATRIC RESEARCH: PRODUCTS STUDIED UNDER TWO RELATED LAWS, BUT IMPROVED TRACKING NEEDED BY FDA

WHY GAO DID THIS STUDY

WHAT GAO RECOMMENDS

WHAT GAO FOUND

ABBREVIATIONS

BACKGROUND

PREA

BPCA

The Pediatric Advisory Committee

Internal Control

AT LEAST 130 PRODUCTS HAVE BEEN STUDIED IN NUMEROUS THERAPEUTIC AREAS UNDER PREA AND BPCA, BUT FDA DOES NOT KNOW IF ADDITIONAL PRODUCTS HAVE BEEN STUDIED

FDA Cannot be Certain How Many Additional Products Have Been Studied under PREA; Most Requests for Waivers and Deferrals Have Been Granted

50 Products Have Been Studied under BPCA since Its 2007 Reauthorization

Drug and Biological Products Were Studied in a Wide Range of Therapeutic Areas

Few Drugs Have Been Studied When Sponsors Have Declined Written Requests

ALL PRODUCTS WITH COMPLETED PEDIATRIC STUDIES HAD LABELING CHANGES, AND FDA’S GOALS OFTEN DIFFER FROM THE PREA REQUIREMENT FOR REACHING AGREEMENT ON LABELING CHANGES

ALL PRODUCTS WITH COMPLETED PEDIATRIC STUDIES HAD LABELING CHANGES, AND FDA’S GOALS OFTEN DIFFER FROM THE PREA REQUIREMENT FOR REACHING AGREEMENT ON LABELING CHANGES

All Products with Completed Pediatric Studies since the 2007 Reauthorization Had Labeling Changes That Included Important Pediatric Information

FDA’s Goals Often Differ from the PREA Requirement for Reaching Agreement on Labeling Changes

STAKEHOLDERS IDENTIFIED AGENCY GUIDANCE, UNCERTAINTY ASSOCIATED WITH REAUTHORIZATION, AND LACK OF ECONOMIC INCENTIVES AS POTENTIAL CHALLENGES TO CONDUCTING PEDIATRIC STUDIES

CONCLUSION

RECOMMENDATION FOR EXECUTIVE ACTION

AGENCY COMMENTS AND OUR EVALUATION

APPENDIX I. INCLUSION OF NEONATES IN DRUG AND BIOLOGICAL PRODUCT STUDIES

APPENDIX II. INCLUSION OF ETHNIC AND RACIAL MINORITY PARTICIPANTS IN PEDIATRIC DRUG STUDIES

APPENDIX III. PEDIATRIC DRUG AND BIOLOGICAL PRODUCT STUDIES IN THE EUROPEAN UNION AND THE UNITED STATES

The European Union’s Paediatric Regulation

European Union and United States Collaboration

End Notes

INDEX

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