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Multi-arm trials are gaining interest in practice given the statistical and logistical advantages they can offer. The standard approach uses a fixed allocation ratio, but there is a call for making it adaptive and skewing the allocation of…

统计方法学 · 统计学 2026-01-16 Gianmarco Caruso , Pavel Mozgunov

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…

统计方法学 · 统计学 2021-08-31 Elja Arjas , Dario Gasbarra

Selective recruitment designs preferentially recruit individuals that are estimated to be statistically informative onto a clinical trial. Individuals that are expected to contribute less information have a lower probability of recruitment.…

统计理论 · 数学 2017-05-31 James E. Barrett

We propose a multi-metric flexible Bayesian framework to support efficient interim decision-making in multi-arm multi-stage phase II clinical trials. Multi-arm multi-stage phase II studies increase the efficiency of drug development, but…

应用统计 · 统计学 2023-12-15 Suzanne M. Dufault , Angela M. Crook , Katie Rolfe , Patrick P. J. Phillips

For biological experiments aiming at calibrating models with unknown parameters, a good experimental design is crucial, especially for those subject to various constraints, such as financial limitations, time consumption and physical…

应用统计 · 统计学 2014-07-22 Xiao Lin , Gabriel Terejanu

We propose a novel adaptive design for clinical trials with time-to-event outcomes and covariates (which may consist of or include biomarkers). Our method is based on the expected entropy of the posterior distribution of a proportional…

应用统计 · 统计学 2016-03-29 James E. Barrett

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…

统计方法学 · 统计学 2022-11-14 Martin Law

Most Bayesian response-adaptive designs unbalance randomization rates towards the most promising arms with the goal of increasing the number of positive treatment outcomes during the study, even though the primary aim of the trial is…

应用统计 · 统计学 2018-07-02 Steffen Ventz , Matteo Cellamare , Sergio Bacallado , Lorenzo Trippa

Minimizing the number of patients exposed to potentially harmful drugs in early onco logical trials is a major concern during planning. Adaptive designs account for the inherent uncertainty about the true effect size by determining the…

应用统计 · 统计学 2016-05-03 Kevin Kunzmann , Meinhard Kieser

Multi-arm designs provide an effective means of evaluating several treatments within the same clinical trial. Given the large number of treatments now available for testing in many disease areas, it has been argued that their utilisation…

统计计算 · 统计学 2019-06-24 Michael J Grayling , James MS Wason

The principle of allocating an equal number of patients to each arm in a randomized controlled trial remains widely believed to be optimal for maximising statistical power. However, this long-held belief only holds true if the treatment…

统计方法学 · 统计学 2025-10-15 Lukas Pin , Stef Baas , David S. Robertson , Sofía S. Villar

This paper considers the optimal adaptive allocation of measurement effort for identifying the best among a finite set of options or designs. An experimenter sequentially chooses designs to measure and observes noisy signals of their…

机器学习 · 计算机科学 2018-06-11 Daniel Russo

Numerous publications have now addressed the principles of designing, analyzing, and reporting the results of, stepped-wedge cluster randomized trials. In contrast, there is little research available pertaining to the design and analysis of…

统计方法学 · 统计学 2018-06-29 Michael Grayling , Adrian Mander , James Wason

The question of how individual patient data from cohort studies or historical clinical trials can be leveraged for designing more powerful, or smaller yet equally powerful, clinical trials becomes increasingly important in the era of…

统计方法学 · 统计学 2022-09-01 Sandra Siegfried , Stephen Senn , Torsten Hothorn

This paper extends my research applying statistical decision theory to treatment choice with sample data, using maximum regret to evaluate the performance of treatment rules. The specific new contribution is to study as-if optimization…

计量经济学 · 经济学 2021-10-05 Charles F. Manski

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…

统计理论 · 数学 2022-07-13 David Azriel , Yosef Rinott , Martin Posch

This work considers the problem of selective-sampling for best-arm identification. Given a set of potential options $\mathcal{Z}\subset\mathbb{R}^d$, a learner aims to compute with probability greater than $1-\delta$, $\arg\max_{z\in…

机器学习 · 计算机科学 2021-11-03 Romain Camilleri , Zhihan Xiong , Maryam Fazel , Lalit Jain , Kevin Jamieson

Adaptive experiments are used extensively in online platforms, healthcare and biotechnology, and a variety of other settings. In many of these applications, the main goal is not to precisely estimate a treatment effect, but to demonstrate…

When an experimenter has the option of running an adaptive trial, is it admissible to ignore this option and run a non-adaptive trial instead? We provide a negative answer to this question in the best-arm identification problem, where the…

机器学习 · 统计学 2025-06-06 Guido Imbens , Chao Qin , Stefan Wager

Trials enroll a large number of subjects in order to attain power, making them expensive and time-consuming. Sample size calculations are often performed with the assumption of an unadjusted analysis, even if the trial analysis plan…

统计方法学 · 统计学 2021-07-06 Alejandro Schuler
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