A Growing Trend: The Expanding Use of Patient-reported Outcome Measures (PROMs) in Clinical Trials (2008-2023)

Author(s)

Justin Yu, PharmD;
University of Illinois-Chicago, Post-doctoral Fellow, Chicago, IL, USA

Presentation Documents

OBJECTIVES: Patient-reported outcome measures (PROMs) have gained prominence in clinical trials following the 2009 FDA guidance on PROMs and establishment of PCORI. This study analyzed trends in the use of PROMs across clinical trials from 2008 to 2023, focusing on widely used generic and disease-specific PROMs across adult and pediatric populations, disease states, and regions.
METHODS: Generic (EQ-5D, SF-36/12/8/6D, PROMIS), condition-specific (EORTC QLQ-C30/QLU-C10D, FACIT), and pediatric (PedsQL, EQ-5D-Y, PROMIS Pediatric, CHQ, CHU9D) PROMs were identified through the PROQOLID database and health technology assessment (HTA) resources. A Python-based algorithm was used to search ClinicalTrials.gov for interventional studies that included at least one of the specified PROMs. Annual trends, regional patterns, and disease-specific usage were summarized.
RESULTS: Analysis of 475,844 registered trials between 2008 and 2023 revealed PROMs adoption increased from 12.7% (n=1,783) to 23.5% (n=7,808) in adult trials, and from 8.0% (n=231) to 17.7% (n=1,118) in pediatric trials. EQ-5D surpassed SF measures as the most frequently used generic PROM in 2017 (2023: EQ-5D=1,023; SF=781). PROMIS exhibited the largest growth (33% annually), reaching 524 trials in 2023. Among pediatric PROMs, PedsQL remained the most used (2023: n=128). PROMs were most commonly used in trials addressing pathological conditions (n=5,316), nervous system diseases (n=4,923), and neoplasms (n=3,794) between 2008 and 2023. Regional trends over this period indicated a preference for EQ-5D in Europe (n=3,334) and SF/PROMIS in North America (n=3,599; n=464).
CONCLUSIONS: By 2023, nearly one-quarter of adult trials and a growing proportion of pediatric trials included PROMs, marking a significant rise alongside a 220% increase in clinical trials from 2008 to 2023. This growth reflects heightened recognition of PROMs' value in capturing patient experiences and increased regulatory and HTA requirements. EQ-5D’s emergence as the leading measure aligns with widespread HTA endorsement. These findings may guide PRO selection strategies and highlight gaps in pediatric adoption and regional policy.

Conference/Value in Health Info

2025-05, ISPOR 2025, Montréal, Quebec, CA

Value in Health, Volume 28, Issue S1

Code

PCR23

Topic

Patient-Centered Research

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes

Disease

No Additional Disease & Conditions/Specialized Treatment Areas

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