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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…

应用统计 · 统计学 2019-09-16 Thevaa Chandereng , Rick Chappell

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…

统计方法学 · 统计学 2022-06-09 David S. Robertson , Kim May Lee , Boryana C. Lopez-Kolkovska , Sofia S. Villar

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.…

统计方法学 · 统计学 2022-08-03 Tianyu Zhan , Lu Cui , Ziqian Geng , Lanju Zhang , Yihua Gu , Ivan S. F. Chan

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…

应用统计 · 统计学 2025-09-16 Rajenki Das , Nina Deliu , Mark Toshner , Sofía S Villar

Response-Adaptive Randomization (RAR) is recognized for its potential to deliver improvements in patient benefit. However, the utility of RAR is contingent on regularization methods to mitigate early instability and preserve statistical…

统计方法学 · 统计学 2025-11-27 Lukas Pin , Stef Baas , Gianmarco Caruso , David S. Robertson , Sofía S. Villar

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,…

统计方法学 · 统计学 2026-02-23 Maria Vittoria Chiaruttini , Lukas Pin , Sofia S. Villar

In clinical trials, response-adaptive randomization (RAR) has the appealing ability to assign more subjects to better-performing treatments based on interim results. The traditional RAR strategy alters the randomization ratio on a…

统计方法学 · 统计学 2021-10-01 David Merrell , Thevaa Chandereng , Yeonhee Park

Response-adaptive randomization (RAR) can increase participant benefit in clinical trials, but also complicates statistical analysis. The burn-in period (a non-adaptive initial stage) is commonly used to mitigate this disadvantage, yet…

统计方法学 · 统计学 2025-10-14 Edwin Y. N. Tang , Stef Baas , Daniel Kaddaj , Lukas Pin , David S. Robertson , Sofía S. Villar

Response-adaptive clinical trial designs allow targeting a given objective by skewing the allocation of participants to treatments based on observed outcomes. Response-adaptive designs face greater regulatory scrutiny due to potential type…

统计方法学 · 统计学 2025-03-19 Stef Baas , Peter Jacko , Sofía S. Villar

This work revisits optimal response-adaptive designs from a type-I error rate perspective, highlighting when and how much these allocations exacerbate type-I error rate inflation - an issue previously undocumented. We explore a range of…

统计方法学 · 统计学 2025-09-09 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…

统计方法学 · 统计学 2024-01-09 Peter Norwood , Marie Davidian , Eric Laber

Response-adaptive designs allow the randomization probabilities to change during the course of a trial based on cumulated response data, so that a greater proportion of patients can be allocated to the better performing treatments. A major…

统计方法学 · 统计学 2020-06-03 David S. Robertson , James M. S. Wason

Covariate adaptive randomization (CAR) procedures are extensively used to reduce the likelihood of covariate imbalances occurring in clinical trials. In literatures, a lot of CAR procedures have been proposed so that the specified…

统计理论 · 数学 2026-03-10 Zhang Li-Xin

Clinical trials are complex and usually involve multiple objectives such as controlling type I error rate, increasing power to detect treatment difference, assigning more patients to better treatment, and more. In literature, both…

统计理论 · 数学 2010-10-20 Hongjian Zhu , Feifang Hu

There has been a split in the statistics community about the need for taking covariates into account in the design phase of a clinical trial. There are many advocates of using stratification and covariate-adaptive randomization to promote…

统计方法学 · 统计学 2011-02-21 William F. Rosenberger , Oleksandr Sverdlov

In developing products for rare diseases, statistical challenges arise due to the limited number of patients available for participation in drug trials and other clinical research. Bayesian adaptive clinical trial designs offer the…

应用统计 · 统计学 2019-09-19 Xiao Wu , Yi Xu , Bradley P. Carlin

Hybrid clinical trials, that borrow real-world data (RWD), are gaining interest, especially for rare diseases. They assume RWD and randomized control arm be exchangeable, but violations can bias results, inflate type I error, or reduce…

Covariate-adjusted response-adaptive (CARA) designs have gained widespread adoption for their clear benefits in enhancing experimental efficiency and participant welfare. These designs dynamically adjust treatment allocations during interim…

统计方法学 · 统计学 2025-12-10 Xinwei Ma , Jingshen Wang , Waverly Wei

Group sequential designs in clinical trials allow for interim efficacy and futility monitoring. Adjustment for baseline covariates can increase power and precision of estimated effects. However, inconsistently applying covariate adjustment…

统计方法学 · 统计学 2023-08-11 Marlena S. Bannick , Sonya L. Heltshe , Noah Simon

To balance the quality and inference cost of a Foundation Model (FM, such as large language models (LLMs)) powered software, people often opt to train a routing model that routes requests to FMs with different sizes and capabilities.…

机器学习 · 计算机科学 2025-06-03 Kirill Vasilevski , Dayi Lin , Ahmed E. Hassan
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