Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.
Net benefit separation and the determination curve: A probabilistic framework for cost-effectiveness estimation.
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DOI：
10.1177/0962280221995972
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发表时间：
2021-05
影响因子：
2.3
通讯作者：
Mitra N
中科院分区：
文献类型：
--
作者：
Spieker AJ;Illenberger N;Roy JA;Mitra N
在比较两种治疗方法的总体价值时，必须考虑临床有效性和成本。人们越来越关心如何将成本和效益措施结合起来，以便为政策提供信息，促进适当的资源分配。净货币效益汇总了平均成本和临床结局差异的信息;然后开发了成本-效果可接受性曲线，以表征净货币效益证据强度随支付意愿阈值波动而变化的程度。从平均差异之外的成本/临床结局特征中获得见解的方法尚未开发，但也可能是信息性的。我们提出了一种新的概率措施的成本效益的基础上的随机排序的个人净效益分布在每个治疗。我们的方法能够适应观察数据中经常遇到的特征，包括混杂和删失，并补充了它提供的见解中的净货币收益。我们进行了一系列的模拟，以评估有限样本的性能，并说明我们提出的方法使用模拟数据的基础上，子宫内膜癌患者的研究。
Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy and promote adequate resource allocation. The net monetary benefit aggregates information on differences in mean cost and clinical outcomes; the cost-effectiveness acceptability curve was then developed to characterize the extent to which the strength of evidence regarding net monetary benefit changes with fluctuations in the willingness-to-pay threshold. Methods to derive insights from characteristics of the cost/clinical outcomes besides mean differences remain undeveloped but may also be informative. We propose a novel probabilistic measure of cost-effectiveness based on the stochastic ordering of the individual net benefit distribution under each treatment. Our approach is able to accommodate features frequently encountered in observational data including confounding and censoring, and complements the net monetary benefit in the insights it provides. We conduct a range of simulations to evaluate finite-sample performance and illustrate our proposed approach using simulated data based on a study of endometrial cancer patients.
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