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.
翻译:与临床试验同时收集的病人一级健康经济数据是技术评估过程的一个重要组成部分,目的是为资源分配决定提供信息。在癌症治疗等生命期结束治疗方面,成本效益/效用数据的建模可能涉及某种形式的分离生存分析,在这种分析中,应当采用适当的方法来处理与健康有关的前进期和后进期生活质量和生存时间的衡量方法,以便产生某种综合的临床效益(例如经质量调整的存活率)。此外,资源使用数据往往从不同服务的健康记录中收集,从中获取不同的费用组成部分(例如治疗、医院或不利事件费用)。这些分析中的一个关键问题是,有效性和成本数据都存在一些复杂性,包括不常态、激增和缺失等,应当以适当的方法加以解决。巴伊斯模型提供了一种强有力的工具,在最近的健康经济学和统计文献中越来越普遍,可以比较容易地联合处理这些问题。本文介绍了一个总体的巴伊斯框架,其中考虑到基于试验的分离生存成本或不良事件的成本。这些分析中的一个关键问题是,从我们有动机的临床评估过程到基于一种具有新动性的数据,从我们有活力的临床的诊断方法提供一种更充分的证据。