论文标题
用于患者级分区生存成本效用分析的贝叶斯框架
A Bayesian framework for patient-level partitioned survival cost-utility analysis
论文作者
论文摘要
与临床试验一起收集的患者级健康经济数据是技术评估过程的重要组成部分,以便为资源分配决策提供信息。对于诸如癌症治疗之类的生命治疗,成本效益/实用程序数据的建模可能涉及某种形式的分区生存分析,其中与健康相关的生活质量和后期期间的生存时间的测量都结合在一起,以产生一些临床量的临床量度(例如,质量调整质量调节的生存)。此外,资源使用数据通常是从不同服务的健康记录中收集的,从中获得不同的成本组成部分(例如,治疗,医院或不良事件成本)。这些分析中的一个关键问题是,有效性和成本数据都呈现出一些复杂性,包括非正常性,尖峰和缺失,应使用适当的方法来解决。贝叶斯建模提供了一种强大的工具,该工具在最近的健康经济学和统计文献中越来越流行,以相对简单的方式共同处理这些问题。本文提出了一个通用的贝叶斯框架,该框架考虑了基于试验的分区生存成本效用数据的复杂关系,有可能为决策者提供更充分的证据,以告知决策过程。我们的方法是由基于试验的数据来激励并应用于一个工作示例,该数据评估了针对晚期非小细胞肺癌患者的新治疗方法的成本效益。
Patient-level health economic data collected alongside clinical trials are an important component of the process of technology appraisal, with a view to informing resource allocation decisions. For end of life treatments, such as cancer treatments, modelling of cost-effectiveness/utility data may involve some form of partitioned survival analysis, where measures of health-related quality of life and survival time for both pre- and post-progression periods are combined to generate some aggregate measure of clinical benefits (e.g. quality-adjusted survival). In addition, resource use data are often collected from health records on different services from which different cost components are obtained (e.g. treatment, hospital or adverse events costs). A critical problem in these analyses is that both effectiveness and cost data present some complexities, including non-normality, spikes, and missingness, that should be addressed using appropriate methods. Bayesian modelling provides a powerful tool which has become more and more popular in the recent health economics and statistical literature to jointly handle these issues in a relatively easy way. This paper presents a general Bayesian framework that takes into account the complex relationships of trial-based partitioned survival cost-utility data, potentially providing a more adequate evidence for policymakers to inform the decision-making process. Our approach is motivated by, and applied to, a working example based on data from a trial assessing the cost-effectiveness of a new treatment for patients with advanced non-small-cell lung cancer.