Assessing generalisability by location in trial-based cost-effectiveness analysis: the use of multilevel models

Assessing generalisability by location in trial-based cost-effectiveness analysis: the use of multilevel models
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DOI:
10.1002/hec.914
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发表时间:
2005-05-01
期刊:
影响因子:
2.1
通讯作者:
Briggs, AH
Briggs, AH
中科院分区:
医学3区
文献类型:
--
作者:
Manca, A;Rice, N;Briggs, AH

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医疗保健中的成本效益分析(CEA)越来越多地与多中心和多国随机对照临床试验(RCT)一起进行。增加使用随机CEA的目的是考虑患者之间的抽样变异性的成本效益数据假设观察是独立分布的。然而,如果数据中存在分层结构,也就是说,如果在特定地点招募的患者之间的成本和结果存在相关性,则可能导致成本效益的地点间差异。考虑到中心和国家在临床实践、患者病例组合和提供医疗保健的单位成本等因素方面往往存在差异,在多地点试验中可能会出现这种情况。不承认这一特征可能会导致基于试验的经济研究得出误导性结论。多层次模型(MLM)是一个分析框架,可用于处理分层成本效益数据。使用最近进行的经济分析的数据,本文显示了如何多层次建模可以用来获得(a)更适当的估计人口平均增量成本效益和相关的标准误差相比,标准的随机CEA;和(B)特定地点的增量成本效益的估计,可用于适当地探索中心/国家之间的成本效益结果的变异性。版权所有(c)2004年约翰威利父子有限公司。
Cost-effectiveness analysis (CEA) in health care is increasingly conducted alongside multicentre and multinational randomised controlled clinical trials (RCTs). The increased use of stochastic CEA is designed to account for between-patient sampling variability in cost-effectiveness data assuming that observations are independently distributed. However, between-location variability in cost-effectiveness may result if there is a hierarchical structure in the data; that is, if there is correlation in costs and outcomes between patients recruited in particular locations. This may be expected in multi-location trials given that centres and countries often differ in factors such as clinical practice, patient case-mix and the unit costs of delivering health care. A failure to acknowledge this feature may lead to misleading conclusions in a trial-based economic study. Multilevel modelling (MLM) is an analytical framework that can be used to handle hierarchical cost-effectiveness data. Using data from a recently conducted economic analysis, this paper shows how multilevel modelling can be used to obtain (a) more appropriate estimates of the population average incremental cost-effectiveness and associated standard errors compared to standard stochastic CEA; and (b) location-specific estimates of incremental cost-effectiveness which can be used to explore appropriately the variability between centres/countries of the cost-effectiveness results. Copyright (c) 2004 John Wiley & Sons, Ltd.