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

Abstract

Tasanee Braithwaite,1,2 Melanie Calvert,1,3 Alastair Gray,4 Konrad Pesudovs,5 Alastair K Denniston1,6–8 1Centre for Patient Reported Outcomes Research and NIHR Birmingham Biomedical Research Centre, University of Birmingham, Edgbaston, Birmingham, UK; 2Moorfields Eye Hospital, London, UK; 3Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, UK; 4Health Economics Research Centre, Nuffield Department of Population Health, University of Oxford, Oxford, UK; 5Consultant, Adelaide, SA, Australia; 6Department of Ophthalmology, Queen Elizabeth Hospital Birmingham, University Hospitals Birmingham NHSFT, Birmingham, UK; 7Institute of Inflammation and Ageing, University of Birmingham, Birmingham, UK; 8NIHR Biomedical Research Centre (Moorfields Eye Hospital/UCL), London, UK 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

Keywords

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