贝叶斯决策理论随机化程序的一般化及其对延迟响应的应对

Generalisations of a Bayesian decision-theoretic randomisation procedure and the impact of delayed responses

Computational Statistics and Data Analysis · 2021
被引 11
ABS 3

中文导读

本文推广了约束随机动态规划(CRDP)方法,使其能灵活适应不同试验目标,并评估了在响应延迟情况下的表现,发现CRDP仍优于其他方法且对延迟稳健。

Abstract

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.

临床试验设计贝叶斯统计序贯分析响应自适应随机化