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

Louis S. Matza (), Nan Li (), Katie D. Stewart (), Mahmoud Hashim (), Tom Denee (), Feng Pan (), Qiaoyi Zhang (), Jennifer Lee (), Michel Michaelides () and Hendrik P. N. Scholl ()
Additional contact information
Louis S. Matza: Evidera
Nan Li: Janssen Pharmaceutical K.K
Katie D. Stewart: Evidera
Mahmoud Hashim: Janssen Global Services, LLC
Tom Denee: Middle East and Africa
Feng Pan: Janssen Global Services, LLC
Qiaoyi Zhang: Janssen Global Services, LLC
Jennifer Lee: Middle East and Africa
Michel Michaelides: UCL Institute of Ophthalmology and Moorfields Eye Hospital
Hendrik P. N. Scholl: Medical University of Vienna

The European Journal of Health Economics, 2025, vol. 26, issue 7, No 11, 1263-1273

Abstract: 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

Keywords: Health state utility; X-linked retinitis pigmentosa; XLRP; Visual impairment; Rare disease; Time trade-off (search for similar items in EconPapers)
Date: 2025
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DOI: 10.1007/s10198-025-01761-y

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