Cook J R, Heyse J F
Merck Research Laboratories, P.O. Box 4, West Point, PA 19486, USA.
Stat Med. 2000 Nov 15;19(21):2989-3003. doi: 10.1002/1097-0258(20001115)19:21<2989::aid-sim599>3.0.co;2-g.
Economic evaluations of medical technologies involve a consideration of both costs and clinical benefits, and an increasing number of clinical studies include a specific objective of assessing cost-effectiveness. These studies measure the trade-off between costs and benefits using the cost-effectiveness ratio (CE ratio), which is defined as the net incremental cost per unit of benefit provided by the candidate therapy. In this paper we review the statistical methods which have been proposed for estimating 95 per cent confidence intervals for cost-effectiveness ratios. We show that the use of an angular transformation of the standardized ratio stabilizes the variance of the estimated CE ratio, and provides a clearer interpretation of study results. An estimate of the 95 per cent confidence interval for the CE ratio in the transformed scale is easily made using the jack-knife or bootstrap. The available methods are compared using data from a long term study of mortality in patients with congestive heart failure.
医学技术的经济评估涉及对成本和临床效益的考量，并且越来越多的临床研究包含评估成本效益这一特定目标。这些研究使用成本效益比（CE比）来衡量成本与效益之间的权衡，CE比的定义是候选治疗每提供一单位效益的净增量成本。在本文中，我们回顾了已被提出的用于估计成本效益比95%置信区间的统计方法。我们表明，对标准化比值进行角变换的使用可稳定估计的CE比的方差，并能更清晰地解读研究结果。使用刀切法或自助法可轻松得出变换尺度下CE比的95%置信区间估计值。利用一项针对充血性心力衰竭患者死亡率的长期研究数据对现有方法进行了比较。
