[PERSPECTIVES] Beyond the NEI-VFQ: Recent Experience in the Development and Utilization of Patient-Reported Outcomes for Inherited Retinal Diseases

Todd Durham1, Judit Banhazi2, Francesco Patalano2 and Thiran Jayasundera3 1Foundation Fighting Blindness, Columbia, Maryland 21045, USA 2Novartis, Basel-Stadt 4056, Switzerland 3University of Michigan Kellogg Eye Center, Department of Ophthalmology and Visual Sciences, Ann Arbor, Michigan 48105, USA Correspondence: tdurhamfightingblindness.org

Patient-reported outcome measures (PROMs) are tools designed to capture how a patient feels or functions, without the input or interpretation of anyone else. The earliest PROMs used in studies of inherited retinal diseases (IRDs) lack the validity required for therapy development today. The NEI-VFQ was one of the earliest PROMs developed using concept elicitation and cognitive debriefing of patients, but it lacks items that are common to patients with IRDs and it has poor measurement properties. Recent advances in PROM development include the Michigan Retinal Degeneration Questionnaire (MRDQ) and the ViSIO-PRO for nonsyndromic retinitis pigmentosa (RP), both of which have been qualitatively and quantitatively validated. As these new tools are used in clinical studies, they will generate additional evidence about their measurement characteristics. With the latest advances in PROM development for IRDs, it is now possible to move beyond the NEI-VFQ to measure what is truly important to patients.

留言 (0)

沒有登入
gif