pediatric information provided on drug labels. The result has been a substantial increase in pediatric drug trials, with corresponding information being added to the labels for 115 drugs. Nonetheless, a pressing need for more study remains. Although incentives exist for the study of new, on-patent drugs, some argue that additional incentives are needed, especially to encourage testing of older, off-patent drugs. The two existing laws that address the need to study drugs in pediatric populations—the Best Pharmaceuticals for Children Act (BPCA)2 and the Pediatric Research Equity Act (PREA)3—will sunset in October 2007 without congressional action.4

In this context, and given the urgency of addressing the current gap in pediatric drug safety, the Institute of Medicine’s Forum on Drug Discovery, Development, and Translation held a 1-day workshop, Addressing the Barriers to Pediatric Drug Development, on June 13, 2006. 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.


Regulatory efforts to protect children from harmful medications began in the early part of the twentieth century. Many of the initial laws were established in response to specific incidents involving products that caused harm, especially to children; according to Dr. Lisa Mathis of the FDA, however, the resulting laws benefited adults disproportionately. Information on the use of drugs in children was limited and remained insufficient for decades.


Best Pharmaceuticals for Children Act. Public Law 107–109. (2002).


Pediatric Research Equity Act. Public Law 108–155. (2003).


Subsequent to this workshop, both BPCA and PREA were reauthorized by Congress as part of the Food and Drug Administration Amendments Act of 2007, Public Law 110-85, which was signed by the President in September 2007.

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