Hughes Derralynn, Lenny Andrew, Shah Koonal, Longworth Louise, Devercelli Giovanna, Ayodele Olulade
Royal Free London NHS Foundation Trust, University College London, United Kingdom.
PHMR, London, United Kingdom.
J Health Econ Outcomes Res. 2023 Apr 10;10(1):80-88. doi: 10.36469/001c.71344. eCollection 2023.
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. 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. 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. 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). 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. 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.
健康状态效用是衡量与健康相关的生活质量的指标，反映了对患者健康状况改善的重视程度，是估计质量调整生命年所必需的。关于法布里病（FD）的健康状态效用数据有限。在本研究中，我们使用情景构建和赋值来制定健康状态效用。本研究的目的是使用情景构建和赋值来估计适合纳入FD治疗经济模型的健康状态效用值。健康状态情景是通过对FD患者进行半结构化定性电话访谈，并参考已发表的文献和专家意见而制定的。每个情景在在线调查中由英国普通人群成员使用复合时间权衡（TTO）方法进行赋值，该方法旨在确定受访者为了完全健康地生活而愿意与每种受损健康状态进行交换的时间。对来自英国的8名成年FD患者（50%为女性）进行了访谈。他们是通过多种途径招募的，包括患者组织和社交媒体。受访者的回答、已发表文献的证据以及临床专家的意见为6种健康状态情景（疼痛、中度临床显性FD [CEFD]、重度CEFD、终末期肾病 [ESRD]、中风和心血管疾病 [CVD]）和3种组合健康状态（重度CEFD + ESRD、重度CEFD + CVD和重度CEFD + 中风）的制定提供了依据。对来自英国普通人群的1222名参与者进行了情景赋值调查，这些参与者是外部调查组织的成员并同意参与本研究；1175份调查问卷成功完成并纳入分析。对TTO问题的回答被转换为每种健康状态的效用值。疼痛是效用值最高的健康状态（0.465），而重度CEFD + ESRD是最低的（0.033）。总体而言，随着情景严重程度的增加，平均效用值下降，这表明受访者更愿意用生命年进行交换以避免严重的健康状态。健康状态情景反映了FD对所有主要与健康相关的生活质量领域的影响，可能有助于支持FD治疗的经济建模。
