A simulation study to compare methods for constructing confidence intervals for the incremental cost-effectiveness ratio

A simulation study to compare methods for constructing confidence intervals for the incremental cost-effectiveness ratio
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DOI:
10.1007/s10742-006-0017-9
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发表时间:
2007-06-01
影响因子:
1.5
通讯作者:
Zhou, Xiao-Hua
Zhou, Xiao-Hua
中科院分区:
其他
文献类型:
--
作者:
Fan, Ming-Yu;Zhou, Xiao-Hua

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增量成本效益比(ICER)是健康科学中公认的成本效益指标。由于随机变量比率的复杂性,为ICER构建适当的置信区间具有挑战性。已经提出了许多方法,但这些方法的系统比较是罕见的。此外,关于最佳方法的建议也相互矛盾。Polsky等人(Health Econ.6:243-252,1997)和Briggs等人(Stat. 18:3245-3262,1999)分别进行了比较四种和八种方法的模拟研究。库克和海斯(Cook and Heyse)Med.19:2989-3003,2000)提出了一种角度变换,用于解决使用自举响应的ICER估计的不正确排序的问题,并比较了有和没有变换的几种方法。在本文中,我们进行了广泛的模拟比较最常用的方法,以及尚未在上述模拟研究中进行评估的新方法。我们模拟了各种分布的样本,并考虑了真实标准化增量效应接近于零时不太理想的情况。结果表明,基于Fieller方法,bootstrap百分位数方法和bootstrap标准方法的置信区间在不同的样本分布中始终产生合理的覆盖率。其他方法在很大程度上取决于样本大小,以及标准化增量效应离零有多远。当标准化增量效应很小时,基于转换方法的置信区间是非常有问题的。我们还用一个真实的例子说明了我们的结果。
The incremental cost-effectiveness ratio (ICER) is a well accepted measure of cost-effectiveness in health sciences. Due to the complexity of a ratio of random variables, constructing an appropriate confidence interval for the ICER is challenging. Many methods have been proposed, yet systematic comparisons of these methods are rare. Also, there have been conflicting recommendations for the optimum methodology. Polsky et al. (Health Econ. 6: 243-252, 1997) and Briggs et al. (Stat. Med. 18: 3245-3262, 1999) have performed simulation studies comparing four and eight methods, respectively. Cook and Heyse (Stat. Med. 19: 2989-3003, 2000) have proposed an angular transformation for addressing the problem of improper ordering of ICER estimates using bootstrap resampling, and compared several methods with and without the transformation. In this paper we have conducted an extensive simulation to compare the most commonly used methods as well as new methods that have not yet been evaluated in the aforementioned simulation studies. We simulated samples from a wide range of distributions, and considered the less desired scenarios when the true standardized incremental effect was near zero. The results suggest that confidence intervals based on Fieller's method, bootstrap-percentile method, and bootstrap-standard method consistently yield reasonable coverage percentages across different sample distributions. Other methods depend heavily on sample sizes, as well as how far the standardized incremental effect is away from zero. Confidence intervals based on the transformed methods are exceptionally problematic when the standardized incremental effect is small. We have also illustrated our results with a real example.