Williamson S Faye, Jacko Peter, Jaki Thomas
Department of Mathematics and Statistics, Lancaster University, UK.
Biostatistics Research Group, Population Health Sciences Institute, Newcastle University, UK.
Comput Stat Data Anal. 2022 Oct;174:107407. doi: 10.1016/j.csda.2021.107407.
The design of sequential experiments and, in particular, randomised controlled trials involves a trade-off between operational characteristics such as statistical power, estimation bias and patient benefit. The family of randomisation procedures referred to as Constrained Randomised Dynamic Programming (CRDP), which is set in the Bayesian decision-theoretic framework, can be used to balance these competing objectives. A generalisation and novel interpretation of CRDP is proposed to highlight its inherent flexibility to adapt to a variety of practicalities and align with individual trial objectives. CRDP, as with most response-adaptive randomisation procedures, hinges on the limiting assumption of patient responses being available before allocation of the next patient. This forms one of the greatest barriers to their implementation in practice which, despite being an important research question, has not received a thorough treatment. Therefore, motivated by the existing gap between the theory of response-adaptive randomisation (which is abundant with proposed methods in the immediate response setting) and clinical practice (in which responses are typically delayed), the performance of CRDP in the presence of fixed and random delays is evaluated. Simulation results show that CRDP continues to offer patient benefit gains over alternative procedures and is relatively robust to delayed responses. To compensate for a fixed delay, a method which adjusts the time horizon used in the optimisation objective is proposed and its performance illustrated.
序贯试验的设计,尤其是随机对照试验的设计,涉及到统计效能、估计偏差和患者获益等操作特征之间的权衡。在贝叶斯决策理论框架下的一类随机化程序,即约束随机动态规划(CRDP),可用于平衡这些相互竞争的目标。本文提出了CRDP的一种推广和新颖解释,以突出其适应各种实际情况并与个体试验目标相一致的内在灵活性。与大多数反应自适应随机化程序一样,CRDP取决于在下一个患者分配之前能够获得患者反应的极限假设。这构成了它们在实际应用中的最大障碍之一,尽管这是一个重要的研究问题,但尚未得到彻底解决。因此,鉴于反应自适应随机化理论(在即时反应环境中有大量提出的方法)与临床实践(其中反应通常会延迟)之间存在的现有差距,本文评估了在存在固定延迟和随机延迟的情况下CRDP的性能。模拟结果表明,与其他程序相比,CRDP继续为患者带来获益,并且对延迟反应具有相对较强的稳健性。为了补偿固定延迟,本文提出了一种调整优化目标中使用的时间范围的方法,并说明了其性能。