J Rheumatol. 2024 Jun 1:jrheum.2023-1073. doi: 10.3899/jrheum.2023-1073. Online ahead of print.
ABSTRACT
OBJECTIVE: No prior studies in rheumatology have evaluated the quality or timeliness of patient reported outcome (PRO) measure reporting.
METHODS: Clinical trials that informed new Food and Drug Administration (FDA) approvals for the first rheumatological indication between 1995-2021 were identified. Whether collected PROs were published, met minimal clinically important (MCID) difference or statistical significance (p < 0.05) thresholds, or were consistent with CONSORT-PRO reporting standards was recorded. Hazard ratios and the Kaplan-Meier estimate were used to assess the time from FDA approval to PRO publication.
RESULTS: Thirty one FDA approvals corresponded with 110 pivotal trials and 262 reported patient reported outcome measures (PROs). One of the included studies (1.1%) met all five recommended items, 10/90 (11.1%) met 4 items, 17/90 (18.9%) met 3 items, 21/90 (23.3%) met 2 items, 26/90 (28.9%) met 1 item, and 15/90 (16.7%) met none of the reporting standards. Most PROs met MCID thresholds (149/262, 56.9%) and were statistically significant (223/262, 85.1%). Of our subset analysis, one third of PROs were not published upfront (70/212, 33.0%) and one of nine (22/212, 10.4%) remained unpublished four or more years after initial trial reporting. Publication rates were highest for HAQ-DI (97.4%) and lowest for SF-36 (81.8%). Less than half of these published PROs met MCID and statistical significance thresholds (94/212, 44.3%).
CONCLUSION: One in nine PROs remained unpublished for more than 4 years after initial trial reporting, and compliance with CONSORT PRO reporting guideline was poor. Efforts should be made to ensure PROs are adequately reported and expeditiously published.
PMID:38825357 | DOI:10.3899/jrheum.2023-1073
