Louis S Matza, Nan Li, Katie D Stewart, Mahmoud Hashim, Tom Denee, Feng Pan, Qiaoyi Zhang, Jennifer Lee, Michel Michaelides, Hendrik P N Scholl
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引用次数: 0
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.
期刊介绍：
The European Journal of Health Economics is a journal of Health Economics and associated disciplines. The growing demand for health economics and the introduction of new guidelines in various European countries were the motivation to generate a highly scientific and at the same time practice oriented journal considering the requirements of various health care systems in Europe. The international scientific board of opinion leaders guarantees high-quality, peer-reviewed publications as well as articles for pragmatic approaches in the field of health economics. We intend to cover all aspects of health economics:
• Basics of health economic approaches and methods
• Pharmacoeconomics
• Health Care Systems
• Pricing and Reimbursement Systems
• Quality-of-Life-Studies The editors reserve the right to reject manuscripts that do not comply with the above-mentioned requirements. The author will be held responsible for false statements or for failure to fulfill the above-mentioned requirements.
Officially cited as: Eur J Health Econ
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