Zethraeus Niklas, Johannesson Magnus, Jönsson Bengt, Löthgren Mickael, Tambour Magnus
Stockholm School of Economics, Centre for Health Economics, Stockholm, Sweden.
Pharmacoeconomics. 2003;21(1):39-48. doi: 10.2165/00019053-200321010-00003.
No consensus has yet been reached on how to analyse uncertainty in economic evaluation studies where individual patient data are available for costs and health effects. This paper summarises the available results regarding the analysis of uncertainty on the cost-effectiveness plane and argues for using the net-benefit approach when analysing uncertainty in cost-effectiveness studies. The net-benefit approach avoids the interpretation and statistical problems related to the incremental cost effectiveness ratio and implies several advantages. First, traditional statistical methods can be used for confidence-interval estimation and hypothesis testing. Second, calculation of the optimal sample size and the power of the study are facilitated allowing the correlation between costs and effects to vary within and between patient groups. Third, the use of a Bayesian approach to cost-effectiveness analysis is facilitated. Fourth, a formal relation between cost-effectiveness acceptability curves and statistical inference is provided. Finally, the net-benefit approach gives the Fieller's limits of the confidence interval for the incremental cost-effectiveness ratio in the cost-effectiveness plane. Based on these advantages the net-benefit approach should strongly be considered when analysing uncertainty in cost-effectiveness analyses.
对于如何分析可获取个体患者成本和健康效应数据的经济评估研究中的不确定性,目前尚未达成共识。本文总结了在成本效益平面上分析不确定性的现有结果,并主张在分析成本效益研究中的不确定性时采用净效益方法。净效益方法避免了与增量成本效益比相关的解释和统计问题,并具有若干优势。首先,传统统计方法可用于置信区间估计和假设检验。其次,便于计算最优样本量和研究效能,允许成本与效应之间的相关性在患者组内和组间有所不同。第三,便于使用贝叶斯方法进行成本效益分析。第四,提供了成本效益可接受性曲线与统计推断之间的形式关系。最后,净效益方法给出了成本效益平面上增量成本效益比置信区间的菲勒极限。基于这些优势,在分析成本效益分析中的不确定性时,应大力考虑净效益方法。