Abstract

Objectives

This study examined trends in patient-reported outcome measure (PROM) and clinician-reported outcome measure (ClinROM) use in US Food and Drug Administration (FDA) orphan drug labels from 2018 to 2024, comparing PROM utilization with findings from 2002 to 2017.

Methods

We reviewed FDA-approved orphan drug labels for new molecular entities and biologic license applications with orphan designation from January 1, 2018, to December 31, 2024. Eligible labels referenced a PROM and/or ClinROM. Data were abstracted on approval year, FDA-expedited review pathway, study design, endpoint ranking, instrument category, outcomes measured, and published validation references. Descriptive and trend analyses (P .05) were conducted and PROM findings compared with 2002 to 2017 rates.

Results

Among 207 eligible labels from 2018 to 2024, 13.5% included PROMs, 10.1% included ClinROMs, and 5.3% referenced both. PROM use increased 5.2% (13.5% vs 8.3%) since 2002 to 2017. PROMs were primary endpoints in >60% of 2018 to 2024 labels—a modest increase since 2002 to 2017—and ClinROMs were primary in more than three-fourths. “Rare Disease Specific” instruments were included in .05) were observed between endpoint ranking and instrument type for labels including PROMs, as well as both PROMs and ClinROMs across review periods.

Conclusions

PROM and ClinROM inclusion in FDA orphan drug labels is uncommon. Greater integration could improve care and better convey clinically meaningful treatment value.