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

Abstract

and Research (CBER). 2020. Guidance for industry-patient-focused drug development: collecting comprehensive and representative input. FDA,

Postoperative vision-related quality of life and functional vision outcomes with the Argus II Retinal Prosthesis

“…Regardless, there are documented issues with the NEI-VFQ, namely its multidimensionality 27 , and that the subscales are not psychometrically sound. 28,29 Additionally, it lacks items that are speci c to patients with inherited retinal diseases and has weak measurement properties 30 . Generally, the use of the NEI-VFQ should be re-evaluated in patients with profound vision loss, such as the patient population included in this study.…”

Self-Reported Functional Vision in USH2A-Associated Retinal Degeneration as Measured by the Michigan Retinal Degeneration Questionnaire

“… 3 , 5 To help assess the efficacy of current and future therapies, as well as to investigate the natural course of disease in a manner relevant to patient functioning, patient-reported outcomes (PROs) from reliable and validated questionnaires can be utilized to measure changes in FV. 6 – 8 …”

“… 7 , 9 – 11 Documenting these domains requires PRO tools specifically designed for and tested in target populations to capture the unique difficulties and patients’ self-perceived feelings regarding their health and FV in these diseases. 1 , 8 The Michigan Retinal Degeneration Questionnaire (MRDQ) is a validated and psychometrically calibrated PRO instrument designed using the FDA guidelines to measure vision functioning in patients with rod-cone dystrophy, cone/cone-rod dystrophy, or macular dystrophy. 7 Patients being treated may perceive improvement in FV, making this PRO tool attractive for use in capturing self-reported patient functioning validly and reliably.…”

“…3,5 To help assess the efficacy of current and future therapies, as well as to investigate the natural course of disease in a manner relevant to patient functioning, patient-reported outcomes (PROs) from reliable and validated questionnaires can be utilized to measure changes in FV. [6][7][8] There have been several efforts to create vision-specific PRO instruments. One of the earliest PRO measures used in the study of eye diseases is the National Eye Institute Visual Function Questionnaire (NEI-VFQ); later, subsequent PRO measures became available including the Activity Inventory (AI), Daily Living Tasks Dependent on Vision (DLTV), and the Veterans Affairs Low-Vision Visual Functioning Questionnaire (VA LV VFQ).…”

“…One of the earliest PRO measures used in the study of eye diseases is the National Eye Institute Visual Function Questionnaire (NEI-VFQ); later, subsequent PRO measures became available including the Activity Inventory (AI), Daily Living Tasks Dependent on Vision (DLTV), and the Veterans Affairs Low-Vision Visual Functioning Questionnaire (VA LV VFQ). [8][9][10][11] However, in contrast to other more prevalent eye diseases, patients with IRDs have greater phenotypic variability as well as unique FV limitations. For example, PRO measures used in age-related macular degeneration or diabetic retinopathy do not have domains relevant to photosensitivity, peripheral vision loss, or color vision problems seen in those with retinal dystrophies.…”