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In most clinical trials, patients are randomized with equal probability among treatments to obtain an unbiased estimate of the treatment effect. Response-adaptive randomization (RAR) has been proposed for ethical reasons, where the…

Applications · Statistics 2019-09-16 Thevaa Chandereng , Rick Chappell

Response adaptive randomization (RAR) is appealing from methodological, ethical, and pragmatic perspectives in the sense that subjects are more likely to be randomized to better performing treatment groups based on accumulating data.…

Methodology · Statistics 2022-08-03 Tianyu Zhan , Lu Cui , Ziqian Geng , Lanju Zhang , Yihua Gu , Ivan S. F. Chan

Response-Adaptive Randomization (RAR) is part of a wider class of data-dependent sampling algorithms, for which clinical trials are typically used as a motivating application. In that context, patient allocation to treatments is determined…

Methodology · Statistics 2022-06-09 David S. Robertson , Kim May Lee , Boryana C. Lopez-Kolkovska , Sofia S. Villar

Maximizing statistical power in experimental design often involves imbalanced treatment allocation, but several challenges hinder its practical adoption: (1) the misconception that equal allocation always maximizes power, (2) when only…

Methodology · Statistics 2025-09-17 Stef Baas , Lukas Pin , Sofía S. Villar , William F. Rosenberger

Response-adaptive randomization (RAR) has been studied extensively in conventional, single-stage clinical trials, where it has been shown to yield ethical and statistical benefits, especially in trials with many treatment arms. However, RAR…

Methodology · Statistics 2024-01-09 Peter Norwood , Marie Davidian , Eric Laber

Response-adaptive randomisation (RAR) can considerably improve the chances of a successful treatment outcome for patients in a clinical trial by skewing the allocation probability towards better performing treatments as data accumulates.…

Applications · Statistics 2017-03-14 Sofia S. Villar , Jack Bowden , James Wason

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Although response-adaptive randomisation (RAR) has gained substantial attention in the literature, it still has limited use in clinical trials. Amongst other reasons, the implementation of RAR in real world trials raises important practical…

Applications · Statistics 2025-09-16 Rajenki Das , Nina Deliu , Mark Toshner , Sofía S Villar

A constrained Markov decision process (CMDP) approach is developed for response-adaptive procedures in clinical trials with binary outcomes. The resulting CMDP class of Bayesian response -- adaptive procedures can be used to target a…

Methodology · Statistics 2024-01-31 Stef Baas , Aleida Braaksma , Richard J. Boucherie

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

Methodology · Statistics 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

In this paper, we propose an approximate dynamic programming (ADP) algorithm to solve a Markov decision process (MDP) formulation for the admission control of elective patients. To manage the elective patients from multiple specialties…

Optimization and Control · Mathematics 2021-03-10 Jian Zhang , Mahjoub Dridi , Abdellah El Moudni

In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

Methodology · Statistics 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

The Alternating Direction Method of Multipliers (ADMM) has gained a lot of attention for solving large-scale and objective-separable constrained optimization. However, the two-block variable structure of the ADMM still limits the practical…

Optimization and Control · Mathematics 2020-03-24 Kresimir Mihic , Mingxi Zhu , Yinyu Ye

The majority of response-adaptive randomisation (RAR) designs in the literature rely on efficacy data to guide dynamic patient allocation. However, their applicability becomes limited in settings where efficacy outcomes, such as survival,…

Methodology · Statistics 2026-02-23 Maria Vittoria Chiaruttini , Lukas Pin , Sofia S. Villar

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

Methodology · Statistics 2022-11-14 Martin Law

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

Rheumatoid arthritis (RA) is an autoimmune condition caused when patients' immune system mistakenly targets their own tissue. Machine learning (ML) has the potential to identify patterns in patient electronic health records (EHR) to…

Quantitative Methods · Quantitative Biology 2022-10-25 Shengjia Chen , Nikunj Gupta , Woodward B. Galbraith , Valay Shah , Jacopo Cirrone

We study the design of multi-armed parallel group clinical trials to estimate personalized treatment rules that identify the best treatment for a given patient with given covariates. Assuming that the outcomes in each treatment arm are…

Statistics Theory · Mathematics 2022-07-13 David Azriel , Yosef Rinott , Martin Posch

The conventional more-is-better dose selection paradigm, which targets the maximum tolerated dose (MTD), is not suitable for the development of targeted therapies and immunotherapies as the efficacy of these novel therapies may not increase…

Methodology · Statistics 2023-08-31 Peng Yang , Daniel Li , Ruitao Lin , Bo Huang , Ying Yuan
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