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开发和测试从特定疾病测量指标(和其他基于患者的结果测量指标)中推导出基于偏好的健康测量指标的方法。

Developing and testing methods for deriving preference-based measures of health from condition-specific measures (and other patient-based measures of outcome).

机构信息

Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, UK.

出版信息

Health Technol Assess. 2012 Jul;16(32):1-114. doi: 10.3310/hta16320.

DOI:10.3310/hta16320
PMID:22832015
Abstract

OBJECTIVES

Generic preference-based measures such as EQ-5D are widely used to estimate quality-adjusted life-years but may not be available or, more importantly, appropriate in some medical conditions. Condition-specific preference-based measures (CSPBMs) provide an alternative to generic measures that may be more relevant in some conditions. This project conducted five studies to examine issues in the development and use of CSPBMS: (1) literature review of measures; (2) deriving health states values for classifications with highly correlated dimensions; (3) impact of condition labelling; (4) impact of add-on dimensions; and (5) comparative performance of measures.

DESIGN

(1) Systematic search and literature review; (2) and (5) psychometric analyses on existing data; (2), (3) and (4) valuation surveys and survey analyses.

SETTING

Valuation surveys conducted using face-to-face interviews in the respondents' homes.

PARTICIPANTS

Valuation surveys conducted using representative samples of the UK general population.

INTERVENTIONS

Not applicable.

MAIN OUTCOME MEASURES

The project developed a CSPBM CORE-6D and analyses AQL-5D, CORE-6D, EORTC-8D, EQ-5D, OAB-5D and SF-6D data.

RESULTS

(1) There was substantial variability in methods used to develop CSPBMs. (2) A new method for generating states using Rasch analysis was undertaken, which successfully dealt with the problem of highly correlated domains. (3) Condition labels affected utility values but this was dependent on the condition and severity of the health state. (4) Adding on an extra dimension affected health-state values and preference weights for other dimensions. (5) The performance of CSPBMs was comparable with that of their parent instrument and of generic preference-based measures with better performance for discrimination between severity groups.

CONCLUSIONS

CSPBMs have an important role for economic evaluation, for which generic measures are inappropriate. However, their use in economic evaluation may be compromised by naming the condition; the exclusion of side effects and comorbidities; and focusing effects. Whether a reduction in comparability should be accepted depends on the extent of any gain in validity and responsiveness. This will depend on the condition and measure in question. Research agenda: (1) The appropriateness of generic preference-based measures should be examined in more conditions (and compared with CSPBMs). (2) Further quantitative and qualitative work is requested into the impact of, and reasons for labelling effects. (3) Use of add-ons for condition-specific measures (for side effects and comorbidities) and as a solution to the limitation of generic measures should be explored.

FUNDING

The National Institute for Health Research Health Technology Assessment programme.

摘要

目的

通用偏好量表,如 EQ-5D,被广泛用于估计质量调整生命年,但在某些医疗情况下可能不可用或更重要的是不适用。特定于疾病的偏好量表(CSPBM)为通用量表提供了替代方案,在某些情况下可能更相关。本项目进行了五项研究,以探讨 CSPBMS 开发和使用中的问题:(1)对量表的文献综述;(2)对高度相关维度的分类进行健康状态值的推导;(3)疾病标签的影响;(4)附加维度的影响;以及(5)量表的比较性能。

设计

(1)系统搜索和文献综述;(2)和(5)对现有数据进行心理测量分析;(2)、(3)和(4)估值调查和调查分析。

设置

在受访者家中进行面对面访谈的估值调查。

参与者

使用英国一般人群的代表性样本进行估值调查。

干预措施

无。

主要观察指标

项目开发了 CSPBM CORE-6D,并分析了 AQL-5D、CORE-6D、EORTC-8D、EQ-5D、OAB-5D 和 SF-6D 数据。

结果

(1)开发 CSPBM 的方法存在很大差异。(2)采用 Rasch 分析进行了生成状态的新方法,成功解决了高度相关领域的问题。(3)疾病标签会影响效用值,但这取决于疾病和健康状况的严重程度。(4)增加额外维度会影响健康状态值和其他维度的偏好权重。(5)CSPBM 的性能与母体仪器和通用偏好量表相当,在区分严重程度组方面具有更好的性能。

结论

CSPBM 对于不适合使用通用偏好量表的经济评估具有重要作用。然而,疾病名称、排除副作用和合并症以及关注效果可能会影响其在经济评估中的应用。是否接受可比性的降低取决于有效性和响应性的任何提高程度。这将取决于具体情况和所涉及的措施。研究议程:(1)应在更多情况下检查通用偏好量表的适宜性(并与 CSPBM 进行比较)。(2)需要进一步进行定量和定性研究,以了解和解释标签效应的影响和原因。(3)应探讨针对特定疾病的量表(用于副作用和合并症)和通用量表的局限性的附加组件的使用。

资金来源

英国国家卫生研究院健康技术评估计划。

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