Green Nathan, Kurt Murat, Moshyk Andriy, Larkin James, Baio Gianluca
Department of Statistical Science, UCL, London, UK.
Worldwide Health Economics and Outcomes Research, Bristol Myers Squibb, Lawrence, New Jersey, USA.
Stat Med. 2025 Jun;44(13-14):e70132. doi: 10.1002/sim.70132.
Time to an event of interest over a lifetime is a central measure of the clinical benefit of an intervention used in a health technology assessment (HTA). Within the same trial, multiple end-points may also be considered. For example, overall and progression-free survival time for different drugs in oncology studies. A common challenge is when an intervention is only effective for some proportion of the population who are not clinically identifiable. Therefore, latent group membership as well as separate survival models for identified groups need to be estimated. However, follow-up in trials may be relatively short leading to substantial censoring. We present a general Bayesian hierarchical framework that can handle this complexity by exploiting the similarity of cure fractions between end-points; accounting for the correlation between them and improving the extrapolation beyond the observed data. Assuming exchangeability between cure fractions facilitates the borrowing of information between end-points. We undertake a comprehensive simulation study to evaluate the model performance under different scenarios. We also show the benefits of using our approach with a motivating example, the CheckMate 067 phase 3 trial consisting of patients with metastatic melanoma treated with first line therapy.
一生中发生感兴趣事件的时间是卫生技术评估(HTA)中使用的干预措施临床益处的核心衡量指标。在同一试验中,也可能会考虑多个终点。例如,肿瘤学研究中不同药物的总生存期和无进展生存期。一个常见的挑战是,当一种干预措施仅对部分临床上无法识别的人群有效时。因此,需要估计潜在的组群归属以及已识别组群的单独生存模型。然而,试验中的随访可能相对较短,导致大量删失。我们提出了一个通用的贝叶斯分层框架,该框架可以通过利用终点之间治愈比例的相似性来处理这种复杂性;考虑它们之间的相关性,并改善对观测数据之外的推断。假设治愈比例之间具有可交换性有助于在终点之间借用信息。我们进行了一项全面的模拟研究,以评估不同场景下模型的性能。我们还通过一个具有启发性的例子展示了使用我们方法的益处,即由接受一线治疗的转移性黑色素瘤患者组成的CheckMate 067 3期试验。