A primary function of the BPCA is to prioritize needs in pediatric therapeutics.
Under the 2002 BPCA, the prioritization process was based on three major factors:
- Availability of information concerning the safe and effective use of a drug in the pediatric population and the need for additional information
- Potential health benefits in the pediatric population resulting from new studies
- Possible need for reformulation of existing products
The law that reauthorized the BPCA in 2007 also revised the prioritization process to emphasize knowledge gaps in therapeutic areas, as opposed to knowledge gaps about specific drug products.
The 2007 legislation directed the 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
- Particular 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 both 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 to add credibility to the outcomes.