OBJECTIVES: PROs can be used to measure symptom severity, patient function, and patient satisfaction. Typically, clinical trials for RA include composite endpoints, such as the American College of Rheumatology Score (i.e. ACR20) which includes a PRO component, as primary endpoints. Other PRO measures in RA are “stand-alone” (i.e. evaluated by themselves) and could provide more information on the condition and its impacts. The objective of this study was to identify stand-alone PRO measures used in adult RA clinical trials.

METHODS: Interventional (pharmaceutical), phase III, ongoing and completed clinical trials in adults with RA in the United States, Canada, France, Germany, Italy, Spain, United Kingdom, and Japan were identified using ClinicalTrials.gov. Trials were limited between December 2013 and December 2018, and were evaluated to identify stand-alone PRO measures. PRO measures were stratified into generic, condition-specific, or RA-specific through a PRO database or PubMed search.

RESULTS: Of 2,267 trials, 11 trials met inclusion criteria and 17 unique stand-alone PRO measures were identified. Most of the commonly used PRO measures were not RA-specific, such as the Health Assessment Questionnaire Disability Index (HAQ-DI) (11 trials), Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) (6 trials), Short Form-36 (SF-36) (3 trials), and EuroQol-5D (EQ-5D) (3 trials). Additionally, the 8 identified RA-specific measures were only used once each among included trials.

CONCLUSIONS: While 8 of the 17 measures identified were RA-specific, they were not widely used. Most clinical trials conducted within the past 5 years used generic or condition-specific PRO measures focused on physical function and overall health-related quality of life, without being specific to RA. Other measures used infrequently include ones that focused on sleep, mood, and work productivity. Future research should focus on conducting a gap analysis of RA-specific PROs measures to determine which measures are fit-for-purpose for inclusion in future clinical trials.