An automated platform trial framework for A/B testing

IF 1.4 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Contemporary Clinical Trials Communications Pub Date : 2024-11-04 DOI:10.1016/j.conctc.2024.101388
Wenru Zhou , Miranda Kroehl , Maxene Meier , Alexander Kaizer
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Abstract

This paper proposes a platform trial for conducting A/B tests with multiple arms and interim monitoring to investigate the impact of several factors on the expected sample size and probability of early stopping. We examined the performance of three stopping boundaries: O’Brien Fleming (OBF) stopping for either futility or difference (both), Pocock stopping for futility only, and fixed sample size design. We simulated twelve scenarios of different orders of arms based on various effect sizes, as well as considered 1 or 3 interim looks. The overall findings are summarizing in a flowchart to provide intuitive guidance for the design of the platform based on the simulation. We found that it is better to use OBF stopping for both if there is any effective variant and the trial is sufficiently powered to detect the expected effect size. If the study is underpowered to detect a difference, we recommend fixed sample size design to gather as much information as possible, however if the expected sample size is important to minimize, we recommend using Pocock boundaries with futility monitoring. Our results aimed at helping high-tech companies conduct their own studies without requiring extensive knowledge of clinical trial design and statistical methodology.
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用于 A/B 测试的自动化平台试验框架
本文提出了一种进行多臂 A/B 试验和中期监测的平台试验,以研究多个因素对预期样本量和提前停止概率的影响。我们考察了三种停止界限的性能:奥布莱恩-弗莱明(O'Brien Fleming,OBF)停止无效或差异(两者),波科克(Pocock)停止无效,以及固定样本量设计。我们根据不同的效应大小模拟了 12 种不同数量级的手臂,并考虑了 1 或 3 次中期观察。我们以流程图的形式总结了总体研究结果,以便为基于模拟的平台设计提供直观指导。我们发现,如果存在任何有效的变体,且试验有足够的动力来检测预期效应大小,那么最好都使用 OBF 停止。如果研究动力不足,无法检测到差异,我们建议采用固定样本量设计,以收集尽可能多的信息;但如果预期样本量对最小化很重要,我们建议采用带有无效性监测的 Pocock 边界。我们的研究成果旨在帮助高科技公司开展自己的研究,而无需掌握丰富的临床试验设计和统计方法知识。
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来源期刊
Contemporary Clinical Trials Communications
Contemporary Clinical Trials Communications Pharmacology, Toxicology and Pharmaceutics-Pharmacology
CiteScore
2.70
自引率
6.70%
发文量
146
审稿时长
20 weeks
期刊介绍: Contemporary Clinical Trials Communications is an international peer reviewed open access journal that publishes articles pertaining to all aspects of clinical trials, including, but not limited to, design, conduct, analysis, regulation and ethics. Manuscripts submitted should appeal to a readership drawn from a wide range of disciplines including medicine, life science, pharmaceutical science, biostatistics, epidemiology, computer science, management science, behavioral science, and bioethics. Contemporary Clinical Trials Communications is unique in that it is outside the confines of disease specifications, and it strives to increase the transparency of medical research and reduce publication bias by publishing scientifically valid original research findings irrespective of their perceived importance, significance or impact. Both randomized and non-randomized trials are within the scope of the Journal. Some common topics include trial design rationale and methods, operational methodologies and challenges, and positive and negative trial results. In addition to original research, the Journal also welcomes other types of communications including, but are not limited to, methodology reviews, perspectives and discussions. Through timely dissemination of advances in clinical trials, the goal of Contemporary Clinical Trials Communications is to serve as a platform to enhance the communication and collaboration within the global clinical trials community that ultimately advances this field of research for the benefit of patients.
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