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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

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 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.…

应用统计 · 统计学 2017-03-14 Sofia S. Villar , Jack Bowden , James Wason

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

Sequential monitoring in clinical trials is often employed to allow for early stopping and other interim decisions, while maintaining the type I error rate. However, sequential monitoring is typically described only in the context of a…

统计理论 · 数学 2012-05-29 Victoria Plamadeala , William F. Rosenberger

Clustered observations are ubiquitous in controlled and observational studies and arise naturally in multi-centre trials or longitudinal surveys. We present a novel model for the analysis of clustered observations where the marginal…

统计方法学 · 统计学 2022-11-03 Luisa Barbanti , Torsten Hothorn

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

Strategic test allocation plays a major role in the control of both emerging and existing pandemics (e.g., COVID-19, HIV). Widespread testing supports effective epidemic control by (1) reducing transmission via identifying cases, and (2)…

统计方法学 · 统计学 2022-12-06 Ivana Malenica , Jeremy R. Coyle , Mark J. van der Laan , Maya L. Petersen

We consider adaptive designs for a trial involving N individuals that we follow along T time steps. We allow for the variables of one individual to depend on its past and on the past of other individuals. Our goal is to learn a mean…

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

Post-approval safety surveillance of medical products using observational healthcare data can help identify safety issues beyond those found in pre-approval trials. When testing sequentially as data accrue, maximum sequential probability…

统计方法学 · 统计学 2022-07-07 Martijn J. Schuemie , Fan Bu , Akihiko Nishimura , Marc A. Suchard

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

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

A sequential multiple assignment randomized trial (SMART) facilitates comparison of multiple adaptive treatment strategies (ATSs) simultaneously. Previous studies have established a framework to test the homogeneity of multiple ATSs by a…

统计方法学 · 统计学 2022-11-04 Liwen Wu , Junyao Wang , Abdus S. Wahed

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

统计方法学 · 统计学 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

Randomized experiments play a major role in data-driven decision making across many different fields and disciplines. In medicine, for example, randomized controlled trials (RCTs) are the backbone of clinical trial methodology for testing…

应用统计 · 统计学 2016-08-30 Andrew W. Correia

It is common to conduct causal inference in matched observational studies by proceeding as though treatment assignments within matched sets are assigned uniformly at random and using this distribution as the basis for inference. This…

统计方法学 · 统计学 2023-11-14 Samuel D. Pimentel , Yaxuan Huang

Covariate-adaptive randomization (CAR) procedures are frequently used in comparative studies to increase the covariate balance across treatment groups. However, because randomization inevitably uses the covariate information when forming…

统计理论 · 数学 2022-07-08 Wei Ma , Yichen Qin , Yang Li , Feifang Hu

Adaptive clinical trials rely on interim analyses, flexible stopping, and data-dependent design modifications that complicate statistical guarantees when fixed-horizon test statistics are repeatedly inspected or reused after adaptations.…

统计方法学 · 统计学 2026-02-09 Alexandra Sokolova , Vadim Sokolov

Sequential multiple assignment randomized trials mimic the actual treatment processes experienced by physicians and patients in clinical settings and inform the comparative effectiveness of dynamic treatment regimes. In such trials,…

统计方法学 · 统计学 2025-04-07 Zi Wang , Yu Cheng , Abdus S. Wahed
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