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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

Fisher's randomization test (FRT) delivers exact $p$-values under the strong null hypothesis of no treatment effect on any units whatsoever and allows for flexible covariate adjustment to improve the power. Of interest is whether the…

统计方法学 · 统计学 2021-05-03 Anqi Zhao , Peng Ding

Randomized experiments have been the gold standard for assessing the effectiveness of a treatment or policy. The classical complete randomization approach assigns treatments based on a prespecified probability and may lead to inefficient…

统计方法学 · 统计学 2023-10-26 Waverly Wei , Xinwei Ma , Jingshen Wang

Treatment-covariate interaction tests are commonly applied by researchers to examine whether the treatment effect varies across patient subgroups defined by baseline characteristics. The objective of this study is to explore…

统计方法学 · 统计学 2024-03-12 Likun Zhang , Wei Ma

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

Adaptive designs for multi-armed clinical trials have become increasingly popular recently in many areas of medical research because of their potential to shorten development times and to increase patient response. However, developing…

应用统计 · 统计学 2017-03-16 Adam Smith , Sofia S. Villar

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

Adaptive trials are now mainstream science. Recently, researchers have taken the adaptive trial concept to its natural conclusion, proposing what we call "Global Cumulative Treatment Analysis" (GCTA). Similar to the adaptive trial, decision…

应用统计 · 统计学 2013-08-06 Jeff Shrager

Adaptive therapy improves cancer treatment by controlling the competition between sensitive and resistant cells through treatment holidays. This study highlights the critical role of treatment-holiday thresholds in adaptive therapy for…

其他定量生物学 · 定量生物学 2025-07-15 Lanfei Sun , Haifeng Zhang , Kai Kang , Xiaoxin Wang , Leyi Zhang , Yanan Cai , Changjing Zhuge , Lei Zhang

A new approach to adaptive design of clinical trials is proposed in a general multiparameter exponential family setting, based on generalized likelihood ratio statistics and optimal sequential testing theory. These designs are easy to…

统计理论 · 数学 2011-05-25 Jay Bartroff , Tze Leung Lai

In clinical trials, a covariate-adjusted response-adaptive (CARA) design allows a subject newly entering a trial a better chance of being allocated to a superior treatment regimen based on cumulative information from previous subjects, and…

应用统计 · 统计学 2011-06-21 Yuan-chin Ivan Chang , Eunsik Park

While randomized controlled trials (RCTs) are critical for establishing the efficacy of new therapies, there are limitations regarding what comparisons can be made directly from trial data. RCTs are limited to a small number of comparator…

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

统计方法学 · 统计学 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong

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

This paper studies inference in randomized controlled trials with covariate-adaptive randomization when there are multiple treatments. More specifically, we study inference about the average effect of one or more treatments relative to…

计量经济学 · 经济学 2019-01-21 Federico A. Bugni , Ivan A. Canay , Azeem M. Shaikh

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

统计方法学 · 统计学 2018-06-12 Rose Baker , Dan Jackson

Designing experiments often requires balancing between learning about the true treatment effects and earning from allocating more samples to the superior treatment. While optimal algorithms for the Multi-Armed Bandit Problem (MABP) provide…

统计计算 · 统计学 2023-01-04 James K. He , Sofía S. Villar , Lida Mavrogonatou

One size fits all approaches to medicine have become a thing of the past as the understanding of individual differences grows. The paper introduces a test for the presence of heterogeneity in treatment effects in a clinical trial.…

We consider the classical problem of selecting the best of two treatments in clinical trials with binary response. The target is to find the design that maximizes the power of the relevant test. Many papers use a normal approximation to the…

统计理论 · 数学 2011-03-22 David Azriel , Micha Mandel , Yosef Rinott

The field of precision medicine aims to tailor treatment based on patient-specific factors in a reproducible way. To this end, estimating an optimal individualized treatment regime (ITR) that recommends treatment decisions based on patient…

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