OBJECTIVES: Tremendous advances are being made in treatments that improve survival rates and health-related quality of life (HRQoL) in rare childhood diseases. ClinicalTrials.gov database can provide insight into trends in the use of patient-reported outcome measures (PROMs) in a contemporaneous manner, unlike published results, which are usually available several years after studies start. This study aims to examine trends in the use of generic PROMs in registered pediatric clinical trials.

METHODS: ClinicalTrials.gov database was searched to identify pediatric clinical trials between January 2008 and December 2021 that used generic pediatric PROMs. A list of generic pediatric PROMs was obtained from a recently published systematic review (Kwon et al. 2021). The search strategy involved using combinations of PROMs' full names, abbreviations, and all relevant versions strung together using Boolean operators. Proportions of pediatric clinical trials using PROMs were obtained, stratified by start year and phase, and their characteristics were assessed.

RESULTS: 41,310 pediatric clinical trials were identified. There has been an increase in the proportion of generic PROMs used in pediatric clinical trials, from 1.7% in 2008(n=33) to 5.7% in 2021(n=215). Among trials that used generic PROMs, 95% used non-preference-based PROMs, and 6% used preference-based PROMs. The Pediatric Quality of Life Inventory (PedsQL) was the most commonly used non-preference PROM (n=791), while EQ-5D-Y was the most commonly used preference-based PROM (n=22). The Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric exhibited the most rapid growth of all measures, from 2 registered studies in 2012 to 37 in 2021. Similar trends were observed in a subgroup analysis of registered phase 3 and 4 studies.

CONCLUSIONS: Despite recommendations by regulatory agencies, the inclusion of PROMs remains uncommon in pediatric clinical trials. Efforts should be undertaken to include PROMs in pediatric clinical trials as they represent an underused opportunity to capture HRQoL treatment outcomes in pediatric populations.