Health State Utilities Associated With X-Linked Retinitis Pigmentosa in the United States

Speaker(s)

Matza L1, Li N2, Stewart K1, Hashim M3, Pan F2, Zhang Q2, Milentijevic D4, Lee CH4, Michaelides M5, Scholl H6
1Patient-Centered Research, Evidera, Bethesda, MD, USA, 2Janssen Global Services, LLC, Raritan, NJ, USA, 3Janssen Global Services, LLC, Beerse, Belgium, 4Janssen Scientific Affairs, LLC, Titusville, NJ, USA, 5UCL Institute of Ophthalmology and Moorfields Eye Hospital, London, UK, 6Institute of Molecular and Clinical Ophthalmology and Department of Ophthalmology, University of Basel, Basel, Switzerland

Presentation Documents

OBJECTIVES: X-linked retinitis pigmentosa (XLRP) is a rare genetic retinal disease characterized by impairment in visual field (VF) and visual acuity (VA) with gradual loss of vision leading to blindness. Gene therapies for XLRP are currently under investigation, and health state utilities will be needed for use in cost-utility analyses to examine the value of these treatments. This study aimed to estimate health state utilities associated with XLRP severity based on preferences of general population respondents in the US.

METHODS: Eleven health state vignettes describing combinations of impairment in visual field (VF) and visual acuity (VA) associated with XLRP were developed based on a literature review, clinicians’ input, and interviews with caregivers and patients with XLRP. Vignettes included textual descriptions of VF and VA decline, night blindness, and impact of XLRP, along with two images depicting each combination of VF and VA impairment. The images portrayed a street scene and a group of three people. Health state utilities were estimated in time-trade-off interviews with general population respondents from two locations in the US.

RESULTS: A total of 107 participants completed interviews (49.5% male; mean age=47.3 years; 54 from Seattle, Washington and 53 from Bethesda, Maryland). In a ranking task, health states representing less severe impairment were generally preferred over health states with more severe impairment. Mean (SD) health state utilities also followed this pattern, ranging from 0.904 (0.124) for the mildest health state (no VA impairment, mild VF impairment) to 0.323 (0.489) for the health state representing blindness.

CONCLUSIONS:

Mild VF/VA impairment was associated with higher mean utilities. Relatively low utilities for more severe health states highlight the substantial impact of vision impairment on quality of life caused by XLRP. The health state utilities estimated in this study may be useful in assessing cost-effectiveness for XLRP treatments in the US.

Code

PCR173

Topic

Patient-Centered Research

Topic Subcategory

Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes

Disease

Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases