The 2002 BPCA established a prioritization process for off-patent medications 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 reauthorization of 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 prioritization process was further refined in 2010 to emphasize 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 must 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.
NIH, in consultation with the U.S. Food and Drug Administration (FDA) and experts in pediatric research, is mandated 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
As part of the prioritization process, NICHD solicits nominations from BPCA stakeholders to be considered for priority areas for the following year. These nominations are reviewed, scored, and tiered by expert volunteers. A sample nomination form is available for review (PDF 69 KB). The BPCA Priority List of Needs in Pediatric Therapeutics is then created and informs trials conducted by the Pediatric Trials Network.
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 lael (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.
The BPCA priority list consists of key therapeutic needs in the medical treatment of children and adolescents. The list is organized by therapeutic area, which can be a group of conditions, a subgroup of the population, or a setting of care.
Each calendar year, new therapeutic areas are selected for discussion and further prioritization. Priority lists for recent years appear below.
- Priority List of Pediatric Therapeutic Needs as of March 1, 2019 (PDF 431 KB)
- Priority List of Pediatric Therapeutic Needs as of June 30, 2017 (PDF 157 KB)
- Priority List of Pediatric Therapeutic Needs as of July 31, 2015 (PDF 138 KB)
- Priority List of Pediatric Therapeutic Needs as of July 8, 2014 (PDF 264 KB)
- Priority Lists from 2003-2012
BPCA's first priority list included off-patent drugs that needed further study. It was published in 2003 in the Federal Register. The list was updated annually through 2006. BPCA continues to publish a Federal Register notice for each priority list, when necessary.
BPCA hosts and participates in a variety of meetings related to its efforts, initiatives, and collaborations.
BPCA Stakeholder Meetings include presentations about the status of the program, progress from BPCA-funded research, review and discussion of proposed future therapeutic areas, and updates on currently funded studies. In addition, working groups often present about their activities related to gaps in pediatric therapeutic research.
Information on other conferences and symposia is available in the Archives.
2019 BPCA Stakeholders Meeting
March 22, 2019
Meeting Minutes for the 2019 BPCA Stakeholders Meeting (PDF 458 KB)
2016 BPCA Stakeholders Meeting
December 8, 2016
Meeting Minutes for the 2016 BPCA Stakeholders Meeting (PDF 363 KB)
2014 BPCA Stakeholders Meeting
December 10, 2014
Meeting Minutes for the 2014 BPCA Stakeholders Meeting (PDF 202 KB)
Meeting minutes from 2003-2012 are available in the Archives.
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