Pediatric Exclusivity-Associated Revenues and Labeling Changes, 2013 to 2023.

Objective: To determine the benefits and costs associated with the Best Pharmaceuticals for Children Act's (BPCA) pediatric exclusivity provision from 2013-2023 by analyzing the trials and label changes derived from the BPCA and the revenues associated with the 6-month extension.

Study design: In this retrospective cohort study, public FDA and ClinicalTrials.gov data were used to identify drugs granted BPCA pediatric exclusivity from 2013-2023, evaluate clinical trials, and categorize labeling changes attributable to pediatric exclusivity. For drugs with new generic competition, the commercial SSR Health database was used to evaluate revenues during the extension period.

Results: From 2013-2023, 229 clinical trials were conducted under the BPCA and 110 drugs received pediatric exclusivity, resulting in new efficacy labeling for 104 (97%) and new safety labeling for 21 (20%) drugs. Pediatric exclusivity was granted a median of 2.34 (IQR, 1.27-4.19) years prior to generic entry. Forty (36%) drugs granted pediatric exclusivity had generic entry during our study period. Median excess revenue attributable to the incentive was $133.8 million (IQR, $58.8-275.0 million) per exclusivity grant for a total of $9.03 billion.

Conclusions: The BPCA generates clinical information for treating children that augments drug labeling, while accounting for substantial revenues to pharmaceutical manufacturers due to delaying generic competition. Changes to implementation of the BPCA could allow more timely and cost-effective acquisition of this information.