The use of patient-reported outcome research in modern ophthalmology: impact on clinical trials and routine clinical practice

Abstract

This review article considers the rising demand for patient-reported outcome measures (PROMs) in modern ophthalmic research and clinical practice. We review what PROMs are, how they are developed and chosen for use, and how their quality can be critically appraised. We outline the progress made to develop PROMs in each clinical subspecialty. We highlight recent examples of the use of PROMs as secondary outcome measures in randomized controlled clinical trials and consider the impact they have had. With increasing interest in using PROMs as primary outcome measures, particularly where interventions have been found to be of equivalent efficacy by traditional outcome metrics, we highlight the importance of instrument precision in permitting smaller sample sizes to be recruited. Our review finds that while there has been considerable progress in PROM development, particularly in cataract, glaucoma, medical retina, and low vision, there is a paucity of useful tools for less common ophthalmic conditions. Development and validation of item banks, administered using computer adaptive testing, has been proposed as a solution to overcome many of the traditional limitations of PROMs, but further work will be needed to examine their acceptability to patients, clinicians, and investigators.

Keywords:

• patient-reported outcome measures
• Rasch analysis
• eye disease
• randomized controlled trials

Supplementary material

Table S1 PubMed search

Author contributions

All authors contributed to data analysis. TB drafted the manuscript in consultation with all co-authors, who provided critical revision. All authors gave final approval of the version to be published, and agree to be accountable for all aspects of the work.

Disclosure

Tasanee Braithwaite has received funding support from the charity Olivia’s Vision and is a Cochrane Eyes and Vision Contact Editor. Part of this work contributed to her thesis (University of Oxford, Doctor of Medicine), submitted to the University of Oxford for the Doctor of Medicine degree. Melanie Calvert is funded by the NIHR Birmingham Biomedical Research Centre and the NIHR Surgical Reconstruction and Microbiology Research Centre at the University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham. Konrad Pesudovs has over 25 years of experience in vision and eye disease-related PROMs, has created numerous instruments including the Visual Disability Assessment, the Quality of Life Impact of Refractive Correction, the Contact Lens Impact on Quality of Life, the Quality of Vision Questionnaire, and the Eye-tem Bank. He has been involved in the revalidation using Rasch analysis of numerous instruments mentioned in this review including the Catquest 9SF, the IVI, and the GAL9. He is a proponent of quality assessment of outcomes using PROMs. Inevitably some of his works are mentioned herein. The views expressed are those of the authors and not necessarily those of the NHS, the NIHR, or the Department of Health. Melanie Calvert reports receipt of personal fees from Astellas, Takeda, and Merck and grants from Macmillan Cancer Support and the NIHR and is a coinvestigator within Health Data Research UK Midlands. The authors report no other conflicts of interest in this work.