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基于模型的经济评估研究中的可推广性评估:骨质疏松症的结构化综述

Assessing generalisability in model-based economic evaluation studies: a structured review in osteoporosis.

作者信息

Urdahl Hege, Manca Andrea, Sculpher Mark J

机构信息

Merck Sharp and Dohme, Hertford, UK.

出版信息

Pharmacoeconomics. 2006;24(12):1181-97. doi: 10.2165/00019053-200624120-00004.

Abstract

To support decision making, many countries have now introduced some formal assessment process to evaluate whether health technologies represent good 'value for money'. These often take the form of decision models that can be used to explore elements of importance to generalisability of study results across clinical settings and jurisdictions. The objective of this review was to assess whether articles reporting decision-analytic models in the area of osteoporosis provided enough information to enable decision makers in different countries/jurisdictions to fully appreciate the variability of results according to location and be able to apply the evaluation to their own setting. Of the 18 articles included in the review, only three explicitly stated the decision-making audience. It was not possible to infer a decision-making audience in eight studies. The target population was well reported, as were resource and cost data, and clinical data used for estimates of relative risk reduction. However, baseline risk was rarely adapted to the relevant jurisdiction, and when no decision maker was explicit it was difficult to assess whether the reported cost and resource use data were in fact relevant. A few studies used sensitivity analysis to explore elements of generalisability, such as compliance rates and baseline fracture risk rates, although such analyses were generally restricted to evaluating parameter uncertainty. This review found that variability in cost effectiveness across locations is addressed to a varying extent in modelling studies in the field of osteoporosis, limiting their use for decision makers across different locations. Transparency of reporting is expected to increase as methodology develops and decision makers publish 'reference case' type guidance.

摘要

为支持决策制定,许多国家现已引入一些正式评估程序,以评估卫生技术是否具有良好的“性价比”。这些程序通常采用决策模型的形式,可用于探究对研究结果在不同临床环境和司法管辖区的可推广性具有重要意义的因素。本综述的目的是评估在骨质疏松症领域报告决策分析模型的文章是否提供了足够信息,使不同国家/司法管辖区的决策者能够充分认识到结果因地点而异的情况,并能够将该评估应用于自身环境。在该综述纳入的18篇文章中,只有3篇明确指出了决策受众。在8项研究中无法推断出决策受众。目标人群、资源和成本数据以及用于估计相对风险降低的临床数据均有良好报告。然而,基线风险很少根据相关司法管辖区进行调整,而且当没有明确的决策者时,很难评估所报告的成本和资源使用数据是否实际上相关。一些研究使用敏感性分析来探究可推广性因素,如依从率和基线骨折风险率,尽管此类分析通常仅限于评估参数不确定性。本综述发现,骨质疏松症领域建模研究在不同程度上考虑了不同地点成本效益的差异,这限制了它们对不同地点决策者的用途。随着方法的发展以及决策者发布“参考案例”类型的指南,报告的透明度有望提高。

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