Basket trials allow simultaneous evaluation of a single therapy across multiple cancer types or subtypes of the same cancer. Since the same treatment is tested across all baskets, it may be desirable to borrow information across them to improve the statistical precision and power in estimating and detecting the treatment effects in different baskets. We review recent developments in Bayesian methods for the design and analysis of basket trials, focusing on the mechanism of information borrowing. We explain the common components of these methods, such as a prior model for the treatment effects that embodies an assumption of exchangeability. We also discuss the distinct features of these methods that lead to different degrees of borrowing. Through simulation studies, we demonstrate the impact of information borrowing on the operating characteristics of these methods and discuss its broader implications for drug development. Examples of basket trials are presented in both phase I and phase II settings.
篮式试验允许同时评估单一疗法在多种癌症类型或同一癌症的不同亚型中的效果。由于所有篮子均接受相同治疗测试,因此可能需要在不同篮子间借用信息,以提高估计和检测不同篮子中治疗效果时的统计精确度和效力。本文回顾了篮式试验设计与分析的贝叶斯方法最新进展,重点关注信息借用机制。我们解释了这些方法的共同组成部分,例如体现可交换性假设的治疗效果先验模型。同时讨论了这些方法导致不同程度信息借用的独特特征。通过模拟研究,我们展示了信息借用对这些方法操作特性的影响,并探讨其对药物开发的更广泛意义。文中还提供了篮式试验在Ⅰ期和Ⅱ期研究中的实例。
Bayesian Methods for Information Borrowing in Basket Trials: An Overview