To best prioritize needs in pediatric therapeutics, the 2007 BPCA legislation directed NIH, in consultation with the U.S. Food and Drug Administration (FDA) and experts in pediatric research, to:
- Develop and publish a priority list of needs in pediatric therapeutics, including drugs or indications that require study, and update this list every 3 years.
- Consider the following information in developing and prioritizing the list:
- Therapeutic gaps in pediatrics, including developmental pharmacology, pharmacogenetic determinants of drug response, metabolism of drugs and biologics in children, and pediatric clinical trials
- Pediatric diseases, disorders, or conditions where more complex knowledge and testing of therapeutics, including drugs and biologics, may be beneficial in pediatric populations
The 2007 legislation also noted that infrastructure for conducting pediatric pharmacological research, including research networks and trained pediatric investigators, was adequate.
Legislation further refined the prioritization process in 2010. The revised process emphasizes the following four principles:
- Prioritization must be a well-defined process that includes a systematic approach and clear objectives and outcomes.
- The process must have well-defined objectives and criteria for measuring priority, and these objectives have to be mutually exclusive (not overlap) and of a manageable number.
- Prioritization must be a dynamic but legitimate and fair process that incorporates transparency, stakeholder input, and strong leadership.
- Prioritization must involve experts to inform and contribute to the process and add credibility to the outcomes.
As part of this process, the BPCA convenes Working Group Meetings on specific therapeutic areas. The working groups include national expert scientists, researchers, and clinicians, along with representatives from NICHD and the FDA. These groups hold regular teleconferences and virtual meetings to fulfill their two primary charges:
- Discuss scientific questions that need to be addressed for drugs and indications in that Working Group’s area of expertise. Considerations must be within the context of the current FDA-approved label (if it exists), noting specifically where pediatric information is underdeveloped or lacking.
- Consider the practicalities (given the scope and funding of the BPCA) of conducting additional studies of the drug and/or indication of interest.
Meeting minutes from all BPCA Working Groups are available in the Archives.
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