Use of patient-reported outcomes in ophthalmology clinical trials between 2014 and 2023.

Clinical Snapshot

Key Findings

• P Value: p=0.013 for intervention type association, p<0.001 for CONSORT adherence

• Effect Size: Pharmaceutical interventions less likely to use PROs (aOR 0.15)

• Primary Outcome: 26.1% of ophthalmic RCTs included PROs, with only 8.4% using them as primary outcomes

• Nnt Or Sensitivity: 95.4% of PRO studies used standardised PROMs, 73.5% used ophthalmology-specific tools

• Confidence Interval: 95% CI 0.03 to 0.67 for pharmaceutical vs health service interventions

Clinical Application

Highly feasible - provides clear guidance for improving PRO integration in existing trial frameworks Relevant to Australian ophthalmology research through RANZCO, university medical centres, and TGA-regulated clinical trials requiring patient-centred outcomes Ophthalmologists conducting clinical trials, researchers in vision science, clinical trial methodologists

Abstract

BACKGROUND/AIMS: Patient-reported outcomes (PROs) evaluate health and functional status, and PRO measures (PROMs) are standardised tools for measuring PROs. Together, they provide valuable insights into treatment efficacy, safety and practicality not captured by traditional clinical endpoints. This cross-sectional analysis with a systematic search component aims to investigate the use, interpretation and reporting of PROs and PROMs in ophthalmic randomised controlled trials (RCTs). METHODS: Ophthalmic RCTs published in the top 10 highest impact factor ophthalmic and medical journals between 2014 and 2023 were systematically reviewed. The frequency of PRO inclusion and adherence to Consolidated Standards of Reporting Trials (CONSORT) PRO guidelines was assessed. The relationship between PRO utilisation and study-level and journal-level characteristics was explored with multivariable regression. RESULTS: Among 9436 records screened, 333 RCTs met eligibility criteria. Of these, 87 (26.1%) included PROs, and 28 (8.4%) used them as primary outcomes. Most studies (83/87, 95.4%) leveraged PROMs, with ophthalmology-specific tools predominating (73.5%). Minimal important differences (MIDs) were rarely used (2.3%) for PRO interpretation. At least 8/13 CONSORT PRO Extension items were reported in 33.3% studies, and trials with primary PRO endpoints had better adherence (p<0.001). PRO utilisation was less likely in trials with lower 5-year journal impact factor (adjusted OR (aOR) 0.99, 95% CI 0.98 to 1.00, p=0.037) and pharmaceutical compared with health service interventions (aOR 0.15, 95% CI 0.03 to 0.67, p=0.013). CONCLUSIONS: PRO integration and interpretation remain limited in high-impact ophthalmic RCTs, despite offering a meaningful adjunct to objective endpoints. Future trials should adopt validated and condition-specific PROMs and establish MIDs to enhance interpretation.

References

1. 1.Li, X., Chen, N., Hsiao, H., Hanna, A., Lakhani, M., Kwan, A. T. H., Tao, B. K., Xie, J. S., & Margolin, E. (2024). Use of patient-reported outcomes in ophthalmology clinical trials between 2014 and 2023. British Journal of Ophthalmology. https://doi.org/10.1136/bjo-2024-326961