Federal legislation and regulations require that prescription drugs be tested for safety and efficacy in a specific population, at a specific dosage, and for a specific time period before they are approved by the U.S. Food and Drug Administration (FDA) for clinical use.
Use of drugs outside of the prescribed label indications is considered "off-label" use. Far more drugs have been approved for clinical use in adults than for pediatric populations, which leads to a high level of "off-label" use of drugs in children who need them for various medical conditions. As a result, most drugs used to treat children are used off-label, without an adequate understanding of appropriate dose, safety, or efficacy. BPCA's role is to address these knowledge gaps.
NIH's BPCA efforts aim to:
- Identify drugs (primarily off-patent drugs) that need to be studied in children due to lack of dosing, safety, or efficacy data
- Sponsor clinical trials for those drugs, which includes:
- Identifying appropriate investigators and clinical sites to conduct these studies
- Carefully monitoring drug labeling studies in children
- Submit study data to the FDA for consideration of label modification
- Make the study data available to researchers and the public
Congress appropriates approximately $25 million from the NIH budget each fiscal year for the operation of the BPCA program. NICHD contributes approximately 25 percent of BPCA funding from its annual budget. The remaining funds are drawn from more than 20 other NIH institutes with significant pediatric research portfolios.