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以斑秃为例,探索一种以患者为中心的方法,以确定合适的替代病症作为效用数据的来源。

Exploring a Patient-Centered Methodology to Identify Suitable Proxy Conditions as a Source for Utility Data, Using Alopecia Areata as a Case Example.

作者信息

Biggane Alice M, Schiavo Sofia, Randall Rowena, Elgares Masara, Carter Patrice

机构信息

Pfizer Ltd, Walton Oaks, Tadworth, England, UK.

Health Economics and Outcomes Research Ltd, Cardiff Gate Business Park, Cardiff, Wales, UK.

出版信息

Value Health. 2025 Apr;28(4):599-610. doi: 10.1016/j.jval.2025.01.017. Epub 2025 Feb 6.

DOI:10.1016/j.jval.2025.01.017
PMID:39922299
Abstract

OBJECTIVES

In the absence of EQ-5D utility data, the National Institute for Health and Care Excellence recommends sourcing utility values from a proxy health condition, provided that data indicate that the health-related quality of life (HRQoL) impact of the conditions is similar. Given that there is no standard practice for identifying proxy conditions, this study presents a patient-centered approach for selecting a proxy condition, using alopecia areata (AA) as an example.

METHODS

A structured electronic search was conducted to identify conceptual models for conditions similar to AA with overlapping HRQoL domains. Conceptual models were compared to determine the most suitable proxy condition, defined as the condition with the most overlapping domains and reported similarities between patient HRQoL. A search and comparison of utility data between AA and the selected proxy condition was conducted to validate their comparability in HRQoL impact.

RESULTS

Seven conditions with overlapping HRQoL domains with AA were identified using conceptual models. Atopic dermatitis (AD) had the most overlapping domains (n = 6) and the greatest evidence of similarities between patient HRQoL; thus, it was selected as the proxy condition. Conceptual models indicated that both conditions affected patients' physical, emotional, and functional well-being. Furthermore, AD and AA utilities were comparable, supporting the choice of AD as the proxy condition.

CONCLUSIONS

Given the correlation observed between AA and the selected proxy condition using this methodology, this study presents a potential approach to identify proxy conditions for diseases that may be underrepresented in terms of HRQoL data.

摘要

目的

在缺乏EQ - 5D效用数据的情况下,英国国家卫生与临床优化研究所建议从替代健康状况中获取效用值,前提是数据表明这些状况对健康相关生活质量(HRQoL)的影响相似。鉴于目前尚无识别替代状况的标准做法,本研究以斑秃(AA)为例,提出一种以患者为中心的选择替代状况的方法。

方法

进行结构化电子检索,以识别与AA类似且HRQoL领域重叠的状况的概念模型。比较概念模型以确定最合适的替代状况,定义为具有最多重叠领域且报告患者HRQoL之间存在相似性的状况。对AA与所选替代状况之间的效用数据进行检索和比较,以验证它们在HRQoL影响方面的可比性。

结果

使用概念模型识别出7种与AA的HRQoL领域重叠的状况。特应性皮炎(AD)具有最多的重叠领域(n = 6),并且在患者HRQoL之间存在相似性的证据最为充分;因此,它被选为替代状况。概念模型表明这两种状况均会影响患者的身体、情感和功能健康。此外,AD和AA的效用具有可比性,支持将AD选为替代状况。

结论

鉴于使用该方法观察到AA与所选替代状况之间的相关性,本研究提出了一种潜在方法,可为在HRQoL数据方面可能代表性不足的疾病识别替代状况。

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