Presentation (CTI)

OBJECTIVES: Food and Drug Administration (FDA) guidance recommends administering patient-reported outcome (PRO) measures to pediatric populations. This study compared PRO measure implementation in pediatric versus adult populations for biological and novel drug therapies approved by the FDA from 2021-2025.
METHODS: Searches were conducted in the FDA Novel Drug Approvals and Biological Approvals listings. For each product identified, the corresponding product label was retrieved. ClinicalTrials.gov and MEDLINE® were searched for registrational trial records and publications. PRO-related information from all sources was extracted and summarized.
RESULTS: Among 267 products reviewed, similar proportions included PRO labeling statements for products indicated solely for adults (17.6%) and for those indicated for both adult and pediatric populations (14.3%), with most statements on pediatric PRO endpoints limited to adolescents. Inclusion of PRO measures in registrational trials was slightly more frequent in products indicated for both adult and pediatric populations (65.7%) than for products indicated solely for adults (58.2%); however, nearly one-quarter (23.9%) of these trials did not include PRO measures for older children and/or adolescents. No products indicated solely for pediatric use received a PRO labeling statement, and only 38.5% included PRO measures in their registrational trials. Among products with PRO measures in their registrational trials, publications of PRO-related results were more frequent for products indicated solely for adults (44.5%) or for both adult and pediatric populations (52.9%) than for those indicated solely for a pediatric population (30.8%).
CONCLUSIONS: Despite FDA guidance, PRO measures remain underutilized for pediatric populations in registrational trials of biological and novel drug therapies, particularly for children under age 12 and for indications limited to pediatric populations. Expanding the incorporation of pediatric PRO measures into clinical development programs could help improve patient-centered treatment outcomes in this population.