Transportability of Comparative Effectiveness and Cost-Effectiveness between Countries
Transportability of Comparative Effectiveness and Cost-Effectiveness between Countries
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DOI：
10.1111/j.1524-4733.2010.00751.x
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发表时间：
2010-06-01
期刊：
影响因子：
4.5
通讯作者：
Briggs, Andrew
中科院分区：
文献类型：
--
作者：
Briggs, Andrew
今天进行的大多数临床试验都是多中心的，许多是跨国的。多个中心和国家的纳入通常允许在较短的时间内录取更大的样本量，这提供了更大的统计能力。其他优势包括更大的普适性，以及在药物试验的情况下，赞助商有机会使用结果在一个以上的国家进行注册。同时，这种设计对解释由此产生的成本效益比提出了一些挑战(S)。这些挑战之一是，是否可以假定成本效益分析所需的数据--以及成本效益建议--在不同国家都是相同的。本文对跨国经济分析的文献进行了简要的综述。第一节涉及经济数据可转移性的背景，第二节讨论多国审判经济分析协商一致会议的结果。第三部分讨论了处理跨国数据分析的两种主要统计方法--固定效应和随机效应建模方法--以及最近才在文献中采用的第三种方法，其基础是对成本效益计算的组成部分进行建模。然后使用最近发表的走向COPD健康革命(TORCH)试验的成本效益分析的结果来说明这种方法[1]。最后一节为在比较效益和成本效益研究中使用这种方法提供了一些结论。
Most clinical trials performed today are multicenter and many are multinational. The inclusion of multiple centers and countries usually allows the enrollment of a larger sample size in a shorter period of time, which affords greater statistical power. Other advantages include the perception of greater generalizability and the opportunity for the sponsor, in the case of a drug trial, to use the results for registration in more than one country. At the same time, this design poses a number of challenges for interpreting the resulting cost-effectiveness ratio (s). One of these challenges is whether the data required for cost-effectiveness analysis—and thus the cost-effectiveness recommendations—can be assumed to be equivalent across the different countries. In this article, a brief overview of the literature on economic analysis of multinational studies is presented. The first section relates to the background on the transferability of economic data and the second section discusses the results of a consensus conference on economic analysis of multinational trials. The third section considers the two main statistical approaches that have been advocated for handling the analysis of multinational data—fixed effect and random effect modeling methods—plus a third approach that has only recently been employed in the literature based on modeling the components of the costeffectiveness calculus. This approach is then illustrated using the results of the recently published cost-effectiveness analysis of the TOwards a Revolution in COPD Health (TORCH) trial [1]. A final section offers some conclusions for the use of such methods in comparative effectiveness and cost-effectiveness studies.