Pediatric Clinical Trials

Among the mandates of BPCA is to design and sponsor clinical trials on 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 include the following:

  • Identify therapeutic gaps in pediatric diseases, disorders or conditions in which expanded knowledge of treatment strategies, including drugs and biologics, may be required
  • Identify drugs that still need to be studied in children due to lack of dosing, safety or efficacy data
  • Sponsor clinical trials for those drugs

View a listing of current BPCA clinical trials (PDF 268 KB).

These clinical trials are conducted under regulatory rigor so that the data can be submitted to the FDA for label change consideration. Requirements 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 addition to a label change, there are other avenues for sharing BPCA-supported resources with researchers, healthcare providers, and the public, including publications, training opportunities, lectures, collaborations, and working group recommendations and presentations.​

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