Excerpt

The purpose of the workshop was to identify barriers to the development and testing of drugs for pediatric populations, as well as ways in which the system can be improved to facilitate better treatments for children. The Forum invited representatives from the U.S. Food and Drug Administration (FDA), the National Institutes of Health, the American Academy of Pediatrics, the pharmaceutical industry, academia, and several patient advocacy groups to discuss: the current regulatory framework; current challenges in prescribing and developing drugs for children; models for enhancing pediatric drug development; and challenges and opportunities for the future.

Contents

• The National Academies
• Forum on Drug Discovery, Development, and Translation
• Reviewers
• Summary
  • REGULATORY FRAMEWORK
  • CURRENT CHALLENGES IN DEVELOPING AND PRESCRIBING DRUGS FOR CHILDREN
  • MODELS FOR ENHANCING PEDIATRIC DRUG DEVELOPMENT
  • CHALLENGES AND OPPORTUNITIES FOR THE FUTURE
• 1. Introduction
  • RESPONSE TO DRUGS IN VARIOUS AGE GROUPS
  • PRODUCT LABELING
  • ORGANIZATION OF THIS SUMMARY
• 2. Regulatory Framework
  • BEST PHARMACEUTICALS FOR CHILDREN ACT
  • PEDIATRIC RESEARCH EQUITY ACT
  • IMPACT OF PEDIATRIC DRUG LEGISLATION
  • DISCUSSION
• 3. Current Challenges in Developing and Prescribing Drugs for Children
  • BARRIERS TO PEDIATRIC DRUG DEVELOPMENT
  • FORMULATIONS
  • DOSING, BIOAVAILABILITY, AND DRUG RESPONSE
• 4. Models for Enhancing Pediatric Drug Development
  • VACCINE DEVELOPMENT IN THE UNITED STATES
  • THE EUROPEAN UNION’S NEW REGULATORY APPROACH
  • THE ST. JUDE’S MODEL FOR PEDIATRIC ONCOLOGY DRUGS
• 5. Challenges and Opportunities for the Future
  • SYSTEMIC SOLUTIONS
  • ELIMINATION OF ECONOMIC BARRIERS
  • CONCLUDING THOUGHTS
• References
• Appendixes
  • A Workshop Agenda
  • B Speaker Biographies

Cori Vanchieri and Adrienne Stith Butler, Rapporteurs.

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; Astra- Zeneca 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.

Suggested citation:

IOM (Institute of Medicine). 2008. Addressing the barriers to pediatric drug development: Workshop summary. Washington, DC: The National Academies Press.

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.