UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation

Hughes, D; Lenny, A; Shah, K; Longworth, L; Devercelli, G; Ayodele, O; (2023) Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation. Journal of Health Economics and Outcomes Research , 10 (1) pp. 80-88. 10.36469/001c.71344. Green open access

[thumbnail of 71344-estimation-of-health-state-utility-values-in-fabry-disease-using-vignette-development-and-valuation.pdf]
Preview
PDF
71344-estimation-of-health-state-utility-values-in-fabry-disease-using-vignette-development-and-valuation.pdf - Published Version

Download (276kB) | Preview

Abstract

Background: Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients' health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. Objectives: The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. Methods: Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. Results: Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees' responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). Discussion: Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. Conclusions: Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.

Type: Article
Title: Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.36469/001c.71344
Publisher version: https://doi.org/10.36469/001c.71344
Language: English
Additional information: © The Authors 2023. Original content in this paper is licensed under the terms of the Creative Commons Attribution 4.0 International (CC BY 4.0) Licence (https://creativecommons.org/licenses/by/4.0/).
Keywords: Fabry disease, economic models, enzyme replacement therapy, health state utilities, health-related quality of life
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/10171425
Downloads since deposit
14Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item