The State of Patient-Reported Outcome Measures in Rheumatology

Area of Science:

• Rheumatology Clinical Trials
• Patient-Reported Outcomes
• Regulatory Science

Background:

• Patient-reported outcomes (PROs) are crucial for evaluating treatment efficacy in rheumatology.
• The quality and timeliness of PRO reporting in clinical trials supporting US Food and Drug Administration (FDA) approvals have not been previously assessed.

Purpose of the Study:

• To evaluate the quality and timeliness of PRO reporting in clinical trials for new rheumatological indications approved by the FDA.
• To assess adherence to Consolidated Standards of Reporting Trials (CONSORT)-PRO guidelines.

Main Methods:

• Identified clinical trials supporting FDA rheumatology approvals (1995-2021).
• Assessed PRO publication status, achievement of minimum clinically important difference (MCID) or statistical significance (P < 0.05), and CONSORT-PRO compliance.
• Analyzed time from FDA approval to PRO publication using hazard ratios and Kaplan-Meier estimates.

Main Results:

• Of 262 reported PROs across 110 trials, only 1.1% met all 5 CONSORT-PRO items; 16.7% met none.
• While most PROs showed statistical significance (85.1%), only 56.9% met MCID thresholds.
• One-third of PROs were not published upfront, and 10.4% remained unpublished for ≥ 4 years post-trial reporting.

Conclusions:

• Compliance with CONSORT-PRO reporting standards is inadequate.
• Significant delays in PRO publication exist, with a substantial proportion remaining unpublished long after trial completion.
• There is a critical need to enhance PRO reporting quality and ensure prompt publication in rheumatology research.