Peer-reviewed article
95347 Examining the Impact of the BPCA: Promoting Pediatric Inclusion in Clinical Trials and Pediatric-Specific Drug Information
Ly A, Church T ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This study examined how the Best Pharmaceuticals for Children Act (BPCA) in the United States has promoted the inclusion of children in clinical drug trials and whether this leads to pediatric-specific drug information. Between 2016 and 2018, 22 drugs received marketing exclusivity under BPCA, but analysis of their clinical trials showed that children represented less than 1% of participants in 65 of 118 eligible trials. Although 20 of the 22 drugs eventually received pediatric drug label information, most trials still included both adults and children rather than focusing on pediatric-only populations. The findings suggest that while BPCA has increased pediatric drug labeling, actual pediatric participation in clinical trials remains inadequate.
Key points
- Of 118 clinical trials eligible for assessment, 65 had less than 1% pediatric representation
- 20 of 22 BPCA-compliant drugs received pediatric indications in drug labels
- 135 of 196 trials were open to both adults and pediatrics, while only 10 were open to the entire pediatric population
- 65% of drugs with pediatric indications (13 of 20) had more mixed adult-pediatric trials than pediatric-only trials
- Adequate pediatric clinical trial representation is necessary for developing meaningful pediatric drug labeling
Limitations
This analysis is limited to drugs receiving BPCA marketing exclusivity between 2016-2018 in the United States and does not assess the clinical quality or safety outcomes of pediatric drug use. The study does not examine barriers to pediatric recruitment or compare outcomes between high and low pediatric participation trials.
Abstract
ABSTRACT IMPACT: It provides insight in the relationship between pediatrics and clinical research and how pediatric participation in CT translates to clinical significance in form of drug labels, which inform clinicians on how to prescribe pediatric medications. OBJECTIVES/GOALS: Assessing the extent that the Best Pharmaceuticals for Children Act (BPCA) advances pediatric inclusion in clinical trials (CTs) and the availability of pediatric-specific drug information METHODS/STUDY POPULATION: The BPCA provides the U.S. Food and Drug Administration (FDA) authority to solicit sponsors whose drugs may benefit pediatric populations. Participation is voluntary and provides additional market exclusivity and pediatric information. CTs that received marketing exclusivity from 2016-2018 under BPCA were reviewed using Clinicaltrials.gov to access the legislation’s impact. CTs were categorized according to eligibility: (1) pediatric and adult groups, (2) pediatrics, and (3) pediatric sub-groups. Studies were excluded for ambiguous age data. Studies open to both groups were evaluated for pediatric participation. Each drug was searched in DailyMed.com for published pediatric indications. RESULTS/ANTICIPATED RESULTS: Between 2016 - 2018, 22 drugs received marketing exclusivity under BPCA. Of the 196 CTs conducted for these drugs, 135 were available to adults and pediatrics, 10 were available to the entire pediatric population, and 51 were available to specific pediatric sub-populations. Exclusion criteria permitted only 118 of the CTs for assessment where eligibility included both pediatric and adult populations, of which 65 of these had less than 1% pediatric representation. Of the 22 drugs, 20 have pediatric indications. Over this three-year period, the number of CTs where adults and pediatrics were eligible were greater than CTs for pediatric only or pediatric subpopulations. DISCUSSION/SIGNIFICANCE OF FINDINGS: It is prevalent for BPCA compliant CTs to include both; 65% of drugs (13/20) with pediatric indications had more studies involving both groups than only pediatrics. Adequate pediatric CT representation is necessary for developing pediatric drug labeling with meaningful data for clinical indications.
Findings the Hub has extracted
Each claim below is labelled by its type of design and links back to this report.
- Intervention
- Best Pharmaceuticals for Children Act (BPCA) - legislation providing FDA authority to solicit drug sponsors to conduct pediatric studies, with incentives of additional market exclusivity and pediatric information
- Population
- Clinical trials for 22 drugs that received BPCA marketing exclusivity from 2016-2018 in the United States
Key finding
20 of 22 drugs (91%) with BPCA marketing exclusivity received pediatric indications in drug labels