Abstract

Objectives

To examine trends in patient-reported outcome (PRO) measure use in clinical trials (2008-2023) and analyze adoption patterns of common PRO measures across geographic regions, disease categories, and age groups.

Methods

We identified PRO measures from PROQOLID and health technology assessment recommendations, including generic instruments (EuroQol 5-Dimension [EQ-5D], SF-36/12/8/6D, Patient-Reported Outcomes Measurement Information System [PROMIS]), condition-specific measures (EORTC QLQ-C30/QLU-C10D, Functional Assessment of Chronic Illness Therapy), and pediatric instruments (Pediatric Quality of Life Inventory [PedsQL], EQ-5D-Y, PROMIS Pediatric, Child Health Questionnaire, Child Health Utility 9D). Using a custom Python algorithm (96% sensitivity, 97% specificity), we searched for interventional studies incorporating PRO measures.

Results

Among 475 844 registered trials, PRO measure usage increased substantially. Adult trials incorporating PROs grew from 12.7% (n = 1783) in 2008 to 23.5% (n = 7808) in 2023; pediatric trials increased from 8.0% (n = 231) to 17.7% (n = 1118). EQ-5D surpassed SF measures as the most widely used generic instrument in 2017 (2023: EQ-5D n = 1023, SF n = 781), growing at a compound annual growth rate of 16.2%, whereas PROMIS demonstrated the most rapid expansion with a compound annual growth rate of 51.9%. PRO measures were most frequently implemented in trials studying pathological conditions, nervous system diseases, and neoplasms, with distinct regional preferences: EQ-5D predominated in Europe, whereas SF and PROMIS measures were more common in North America.

Conclusions

By 2023, nearly one-quarter of adult trials included PRO measures, with growing pediatric implementation. PRO integration continues expanding, driven by regulatory and health technology assessment requirements. EQ-5D’s emergence as the leading measure may reflect widespread health technology assessment endorsement. These findings inform PRO selection strategies and identify opportunities for greater pediatric implementation and regional adoption.