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条件特异性偏好测量在卫生技术评估中的作用。

The Role of Condition-Specific Preference-Based Measures in Health Technology Assessment.

机构信息

School of Health and Related Research (ScHARR), University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.

Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152, Glattpark-Opfikon (Zurich), Switzerland.

出版信息

Pharmacoeconomics. 2017 Dec;35(Suppl 1):33-41. doi: 10.1007/s40273-017-0546-9.

DOI:10.1007/s40273-017-0546-9
PMID:29052164
Abstract

A condition-specific preference-based measure (CSPBM) is a measure of health-related quality of life (HRQOL) that is specific to a certain condition or disease and that can be used to obtain the quality adjustment weight of the quality-adjusted life-year (QALY) for use in economic models. This article provides an overview of the role and the development of CSPBMs, and presents a description of existing CSPBMs in the literature. The article also provides an overview of the psychometric properties of CSPBMs in comparison with generic preference-based measures (generic PBMs), and considers the advantages and disadvantages of CSPBMs in comparison with generic PBMs. CSPBMs typically include dimensions that are important for that condition but may not be important across all patient groups. There are a large number of CSPBMs across a wide range of conditions, and these vary from covering a wide range of dimensions to more symptomatic or uni-dimensional measures. Psychometric evidence is limited but suggests that CSPBMs offer an advantage in more accurate measurement of milder health states. The mean change and standard deviation can differ for CSPBMs and generic PBMs, and this may impact on incremental cost-effectiveness ratios. CSPBMs have a useful role in HTA where a generic PBM is not appropriate, sensitive or responsive. However, due to issues of comparability across different patient groups and interventions, their usage in health technology assessment is often limited to conditions where it is inappropriate to use a generic PBM or sensitivity analyses.

摘要

一种特定疾病偏好的衡量指标(CSPBM)是一种特定于特定疾病或病症的健康相关生活质量(HRQOL)衡量指标,可用于获得质量调整生命年(QALY)的质量调整权重,用于经济模型。本文概述了 CSPBM 的作用和发展,并介绍了文献中现有的 CSPBM。本文还比较了通用偏好衡量指标(generic PBM)概述了 CSPBM 的心理测量特性,并考虑了 CSPBM 相对于 generic PBM 的优缺点。CSPBM 通常包括对该病症重要但可能对所有患者群体不重要的维度。在广泛的病症中存在大量的 CSPBM,这些指标涵盖了广泛的维度,也有更具症状或单一维度的指标。心理测量证据有限,但表明 CSPBM 在更准确地衡量轻度健康状态方面具有优势。CSPBM 和 generic PBM 的平均变化和标准差可能不同,这可能会影响增量成本效益比。在不适合使用 generic PBM 或敏感性分析的情况下,CSPBM 在 HTA 中具有有用的作用,敏感或响应性。然而,由于在不同患者群体和干预措施之间的可比性问题,其在卫生技术评估中的使用通常限于不适合使用 generic PBM 的情况下或进行敏感性分析。

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Pharmacoeconomics. 2017 Dec;35(Suppl 1):21-31. doi: 10.1007/s40273-017-0545-x.
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Estimating Health-State Utility for Economic Models in Clinical Studies: An ISPOR Good Research Practices Task Force Report.
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Pharmacoeconomics. 2025 Apr 27. doi: 10.1007/s40273-025-01501-3.
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