Group Response‐Adaptive Randomization With Delayed and Missing Responses

IF 1.8 4区 医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY Statistics in Medicine Pub Date : 2024-09-17 DOI:10.1002/sim.10220
Guannan Zhai, Yang Li, Lixin Zhang, Feifang Hu
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Abstract

Response‐adaptive randomization (RAR) procedures have been extensively studied in the literature, but most of the procedures rely on updating the randomization after each response, which is impractical in many clinical trials. In this article, we propose a new family of RAR procedures that dynamically update based on the responses of a group of individuals, either when available or at fixed time intervals (weekly or biweekly). We show that the proposed design retains the essential theoretical properties of Hu and Zhang's doubly adaptive biased coin designs (DBCD), and performs well in scenarios involving delayed and randomly missing responses. Numerical studies have been conducted to demonstrate that the new proposed group doubly adaptive biased coin design has similar properties to the Hu and Zhang's DBCDs in different situations. We also apply the new design to a real clinical trial, highlighting its advantages and practicality. Our findings open the door to studying the properties of other group response adaptive designs, such as urn models, and facilitate the application of response‐adaptive randomized clinical trials in practice.
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具有延迟和缺失响应的分组响应自适应随机化
文献中对反应自适应随机化(RAR)程序进行了广泛的研究,但大多数程序都依赖于在每次反应后更新随机化,这在许多临床试验中都是不切实际的。在本文中,我们提出了一系列新的 RAR 程序,这些程序可根据一组个体的反应进行动态更新,更新时间可以是有反应时,也可以是固定的时间间隔(每周或每两周)。我们的研究表明,所提出的设计保留了胡和张的双重自适应偏置硬币设计(DBCD)的基本理论特性,并在涉及延迟和随机缺失响应的情况下表现良好。我们进行了数值研究,证明新提出的分组双适应偏置硬币设计在不同情况下具有与胡和张的 DBCD 相似的特性。我们还将新设计应用于实际临床试验,突出了它的优势和实用性。我们的发现为研究其他群体反应自适应设计(如瓮模型)的特性打开了大门,并促进了反应自适应随机临床试验在实践中的应用。
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来源期刊
Statistics in Medicine
Statistics in Medicine 医学-公共卫生、环境卫生与职业卫生
CiteScore
3.40
自引率
10.00%
发文量
334
审稿时长
2-4 weeks
期刊介绍: The journal aims to influence practice in medicine and its associated sciences through the publication of papers on statistical and other quantitative methods. Papers will explain new methods and demonstrate their application, preferably through a substantive, real, motivating example or a comprehensive evaluation based on an illustrative example. Alternatively, papers will report on case-studies where creative use or technical generalizations of established methodology is directed towards a substantive application. Reviews of, and tutorials on, general topics relevant to the application of statistics to medicine will also be published. The main criteria for publication are appropriateness of the statistical methods to a particular medical problem and clarity of exposition. Papers with primarily mathematical content will be excluded. The journal aims to enhance communication between statisticians, clinicians and medical researchers.
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