Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off.

IF 2 Q2 ECONOMICS

PharmacoEconomics Open Pub Date : 2024-11-01 Epub Date: 2024-08-05 DOI:10.1007/s41669-024-00515-5

Maria Farris, Stephen Goodall, Richard De Abreu Lourenco, Brendan Mulhern, Kathleen Manipis, Elena Meshcheriakova, Milena Lewandowska

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引用次数: 0

Abstract

Purpose: Inherited retinal disease (IRD) causes progressive loss of visual function, degenerating towards complete blindness. Economic evaluation of gene therapies for rare forms of genetic IRDs have had to rely on health-related quality of life (HR-QoL) estimates from other diseases because there is limited data available for such a rare condition. This study aimed to estimate Australian societal-based utility values for IRD health states that can be used in cost-utility analyses (CUA) using a time trade-off (TTO) protocol adapted from a UK study.

Methods: The EuroQol Valuation Technology (EQVT) protocol composite TTO (cTTO) framework was followed, which includes worse-than-death (WTD) states and quality control (QC) measures. Preferences were collected from a general population sample of 110 Australian adult participants. Five health state vignettes from the UK study which had been validated with patients and clinicians were presented randomly to participants during videoconferencing (VC) interviews with one of four interviewers. Technical and protocol feasibility were assessed in a pilot of 10 interviews. QC measures were used to monitor interviewers' performance during the study.

Results: One participant withdrew consent. The final analysis was conducted on 109 respondents (including 4 non-traders). The average time to complete the interview was 44.2 minutes (SD 8.7). Participants reported mean visual analogue scale (VAS) scores between 63.15 for 'moderate impairment' and 17.98 for 'hand motion' to 'no light perception'. Mean health state utilities (HSU) varied between 0.76 (SD 0.26) in 'moderate impairment', and 0.20 (SD 0.58) in 'hand motion' to 'no light perception'. Of all HSU evaluations, 14% were considered WTD which most commonly occurred in the most severe visually impaired health state.

Conclusion: This study provides valuable information on HSUs across a range of IRD health states from the Australian general population perspective. The utilities obtained in this study can be used as inputs into CUA of IRD therapies.

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利用时间权衡法估算澳大利亚人口对遗传性视网膜疾病的效用。

目的：遗传性视网膜疾病（IRD）会导致视功能逐渐丧失，最终完全失明。对罕见遗传性 IRD 的基因疗法进行经济评估时，不得不依赖于其他疾病的健康相关生活质量（HR-QoL）估算值，因为这种罕见疾病的可用数据非常有限。本研究旨在估算基于澳大利亚社会的 IRD 健康状况效用值，该效用值可用于成本效用分析 (CUA)，使用的是根据英国一项研究改编的时间权衡 (TTO) 方案：方法：采用欧洲质量评估技术（EQVT）协议复合 TTO（cTTO）框架，其中包括比死亡更差（WTD）状态和质量控制（QC）措施。偏好是从 110 名澳大利亚成年参与者的普通人群样本中收集的。在视频会议（VC）访谈中，四名访谈者中的一人随机向参与者展示了英国研究中的五个健康状态小故事，这些小故事已经过患者和临床医生的验证。在 10 次试点访谈中对技术和协议的可行性进行了评估。在研究过程中，采用质量控制措施对访谈者的表现进行监控：一名受访者放弃同意。对 109 名受访者（包括 4 名非交易员）进行了最终分析。完成访谈的平均时间为 44.2 分钟（标准差为 8.7 分钟）。参与者报告的视觉模拟量表（VAS）平均得分介于 63.15 分（"中度障碍"）和 17.98 分（"手部运动 "至 "无光感"）之间。健康状况效用（HSU）的平均值在 "中度障碍 "和 "手部运动 "至 "无光感 "之间，分别为 0.76（标准差 0.26）和 0.20（标准差 0.58）。在所有 HSU 评估中，14% 被认为是 WTD，这最常见于最严重的视力受损健康状况：本研究从澳大利亚普通人群的角度出发，提供了有关一系列 IRD 健康状况下 HSU 的宝贵信息。本研究获得的效用可作为 IRD 治疗的 CUA 输入。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

PharmacoEconomics Open Multiple-

CiteScore

3.50

自引率

0.00%

发文量

审稿时长

8 weeks

期刊介绍： PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.