Health state utilities associated with X-linked retinitis pigmentosa (XLRP).

Abstract

Background: X-linked retinitis pigmentosa (XLRP) is a rare, inherited retinal disease characterized by impairment in visual field and visual acuity with continuous progression leading to blindness. Gene therapies for XLRP are under investigation, and health state utilities are needed for use in cost-utility analyses examining the value of these treatments.

Objective: This study aimed to estimate utilities associated with XLRP severity.

Methods: Eleven health state vignettes depicting combinations of impairment in visual field and visual acuity associated with XLRP were developed based on literature review and input from clinicians, patients, and a caregiver. Vignettes included text describing visual acuity impairment, visual field impairment, night blindness, impact on quality of life, and two images representing the combination of visual field and visual acuity impairment for each health state. Health states were valued in time trade-off interviews with general population respondents in the UK.

Results: A total of 245 participants completed interviews (51.0% female; mean age = 41.4 years; Newcastle, n = 80; London, n = 85; Edinburgh, n = 80). In a ranking task, participants preferred health states with less severe visual impairment, and this preference was reflected in the utilities. Mean (standard deviation) utilities ranged from 0.900 (0.121) for the health state with no visual acuity impairment and mild visual field impairment to 0.271 (0.478) for the health state describing blindness.

Conclusion: Results highlight the substantial impact of visual impairment on health state preference and quality of life. The health state utilities estimated in this study may be appropriate for use in cost-effectiveness models evaluating treatments for XLRP.

Jel classification codes: I1; I12; I19.