To make informed health policy decisions regarding a treatment, we must consider both its cost and its clinical effectiveness. In past work, we introduced the net benefit separation (NBS) as a novel measure of cost-effectiveness. The NBS is a probabilistic measure that characterizes the extent to which a treated patient will be more likely to experience benefit as compared to an untreated patient. Due to variation in treatment response across patients, uncovering factors that influence cost-effectiveness can assist policy makers in population-level decisions regarding resource allocation. In this paper, we introduce a regression framework for NBS in order to estimate covariate-specific NBS and find determinants of variation in NBS. Our approach is able to accommodate informative cost censoring through inverse probability weighting techniques, and addresses confounding through a semiparametric standardization procedure. Through simulations, we show that NBS regression performs well in a variety of common scenarios. We apply our proposed regression procedure to a realistic simulated data set as an illustration of how our approach could be used to investigate the association between cancer stage, comorbidities and cost-effectiveness when comparing adjuvant radiation therapy and chemotherapy in post-hysterectomy endometrial cancer patients.
翻译:为了就治疗作出知情的保健政策决定,我们必须考虑其成本和临床效果。在过去的工作中,我们引入净福利分离(NBS)作为成本效益的新衡量标准。NBS是一种概率性衡量,它说明接受治疗的病人比未经治疗的病人更有可能受益的程度。由于病人的治疗反应不同,发现影响成本效益的因素可以帮助决策者就资源分配作出人口层面的决定。在本文件中,我们为NBS引入了一个回归框架,以便估计特定变异的NBS, 并发现NBS的变异决定因素。我们的方法能够通过反常概率加权技术进行信息成本审查,并通过半参数标准化程序解决混杂问题。我们通过模拟,表明NBS回归在各种常见情况下表现良好。我们提出的回归程序适用于现实的模拟数据集,以说明我们的方法如何用来调查癌症阶段、诱变性和成本效益之间的关联,以比较后物理癌症的辐射疗法和化学疗法。