Cook John, Drummond Michael, Heyse Joseph F
Merck Research Laboratories, Blue Bell, PA 19422, USA.
Stat Methods Med Res. 2004 Apr;13(2):157-76. doi: 10.1191/0962280204sm359ra.
Healthcare decision makers are increasingly requesting information on the cost and cost-effectiveness of new medicines at the time of product launch. In order to provide this information, data on healthcare resource utilization and, in some cases, costs, may be collected in clinical trials. In this paper, we discuss some of the issues statisticians need to address when it is appropriate to include these economic endpoints in the trial. Several design issues are discussed, including the alternative types of and methods for collecting economic endpoint data, sample size and generalizability. Alternative approaches in the analysis of resource utilization, cost and cost-effectiveness are also presented. Finally, several of the analytic approaches are applied to actual data from a clinical trial.
医疗保健决策者越来越多地要求在新药上市时提供有关其成本和成本效益的信息。为了提供这些信息，可能会在临床试验中收集医疗保健资源利用情况的数据，在某些情况下还会收集成本数据。在本文中，我们讨论了统计学家在临床试验中适当纳入这些经济终点时需要解决的一些问题。讨论了几个设计问题，包括收集经济终点数据的替代类型和方法、样本量和普遍性。还介绍了资源利用、成本和成本效益分析中的替代方法。最后，将几种分析方法应用于一项临床试验的实际数据。
