Patient-reported outcome measures in inherited retinal degeneration gene therapy trials. (2nd January 2020)

• Record Type: Journal Article
• Authors: Lacy, Gabrielle D.; Abalem, Maria Fernanda; Musch, David C.; Jayasundera, Kanishka T.
• Is Part Of: Ophthalmic genetics. Volume 41:Number 1(2020)
• Page Start: 1
• Page End: 6
• Publication Date: 2020-01-02
• Subjects: Patient-reported outcomes -- gene therapy -- inherited retinal degeneration -- clinical trial -- PRO -- retinal dystrophy; Eye -- Diseases -- Genetic aspects -- Periodicals; Eye Diseases -- genetics -- Periodicals; Eye Diseases -- in infancy & childhood -- Periodicals; 617.7
• DOI: 10.1080/13816810.2020.1731836
• Languages: English
• ISSNs: 1381-6810

Abstract

ABSTRACT: Patient-reported outcome (PRO) measures have the potential to uniquely capture patient experience and serve as an outcome measure in inherited retinal degeneration (IRD) gene therapy trials. An IRD-specific patient-reported outcome measure may yield valuable information that has not been obtained from inherited retinal dystrophy gene therapy trials published to-date. Existing PRO measures have inherent limitations for use in IRD gene therapy trials. Developing an applicable patient-reported outcome measure for such trials needs to incorporate patient input from the target population, demonstrate sound psychometric properties, and be made in accordance with U.S. Food and Drug Administration (FDA) guidelines. This review will discuss the currently available PRO instruments, their limitations for IRD therapeutic trials, and suggestions for future PRO development in IRD populations. The PRO instruments highlighted were identified in PubMed search of English-language journals and previously published review articles.