One of the primary mandates of BPCA is to prioritize and sponsor clinical trials of therapeutics in pediatric populations. Doing so requires collaboration among NICHD, other NIH institutes, non-government organizations, and the U.S. Food and Drug Administration (FDA). Specific goals of the clinical research program include the following:
- Novel clinical trial design
- Regulatory rigorous protocols conducted in collaboration with the respective FDA divisions
- Expertise in pharmacology modeling and simulations
- Submission of trial results to the FDA for label change considerations
The requirements and expectations of the BPCA clinical program include the following:
- Carefully monitoring all BPCA-sponsored studies in children for compliance with all federal regulations
- Submitting quality data from the clinical studies to the FDA for evaluation and additional analyses in consideration of label modification
- Making de-identified study data available to investigators and the public
The ultimate outcome of the clinical trials and research efforts of the BPCA program is a change in a medication label to improve the knowledge and safe use of that drug in the pediatric population.
In September 2010, NICHD awarded a contract to launch PTN at Duke Clinical Research Institute (DCRI), to conduct current pediatric clinical drug trials for a variety of therapeutic areas and medical devices. PTN's main objective is to provide an environment and appropriate infrastructure for conducting safe and effective pediatric clinical trials for BPCA.
ClinicalTrials.gov also lists all clinical trials sponsored or funded by the federal government, including BPCA-related trials, in addition to trials managed by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations.
For more information and resources about clinical studies in children, visit NIH's Clinical Research Trials and You.
NICHD and DCRI are interested in candidates who have clinical trial concepts and who would like to collaborate with PTN. Pediatric physicians can submit ideas using a concept sheet (DOCX 1.72 MB).