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Recently, methodology was presented to facilitate the incorporation of interim analyses in stepped-wedge (SW) cluster randomised trials (CRTs). Here, we extend this previous discussion. We detail how the stopping boundaries, allocation…

统计方法学 · 统计学 2018-03-28 Michael Grayling , David Robertson , James Wason , Adrian Mander

Non-adherence to assigned treatment is a common issue in cluster randomised trials (CRTs). In these settings, the efficacy estimand may be also of interest. Many methodological contributions in recent years have advocated using instrumental…

统计方法学 · 统计学 2018-12-05 Schadrac C. Agbla , Bianca De Stavola , Karla DiazOrdaz

Randomized saturation designs are a family of designs which assign a possibly different treatment proportion to each cluster of a population at random. As a result, they generalize the well-known (stratified) completely randomized designs…

统计方法学 · 统计学 2022-03-21 Chencheng Cai , Jean Pouget-Abadie , Edoardo M. Airoldi

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

统计方法学 · 统计学 2026-03-09 Samuel I. Watson , James Martin

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

统计方法学 · 统计学 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

Accurately estimating the intra-class correlation coefficient (ICC) is crucial for adequately powering clustered randomized trials (CRTs). Challenges arise due to limited prior data on the specific outcome within the target population,…

统计方法学 · 统计学 2025-04-23 Chen Yang , Márcio A. Diniz , Deukwoo Kwon , Madhu Mazumdar

There is a growing literature on design-based methods to estimate average treatment effects for randomized controlled trials (RCTs) using the underpinnings of experiments. In this article, we build on these methods to consider design-based…

统计方法学 · 统计学 2024-01-17 Peter Z Schochet

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

统计方法学 · 统计学 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

To test scientific theories and develop individualized treatment rules, researchers often wish to learn heterogeneous treatment effects that can be consistently found across diverse populations and contexts. We consider the problem of…

统计方法学 · 统计学 2026-05-01 Yi Zhang , Melody Huang , Kosuke Imai

Quantifying treatment effect heterogeneity is a crucial task in many areas of causal inference, e.g. optimal treatment allocation and estimation of subgroup effects. We study the problem of estimating the level sets of the conditional…

统计方法学 · 统计学 2023-07-03 Matteo Bonvini , Edward H. Kennedy , Luke J. Keele

A central goal of causal inference is to detect and estimate the treatment effects of a given treatment or intervention on an outcome variable of interest, where a member known as the heterogeneous treatment effect (HTE) is of growing…

统计理论 · 数学 2020-10-27 Zijun Gao , Yanjun Han

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

统计方法学 · 统计学 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa

To effectively optimize and personalize treatments, it is necessary to investigate the heterogeneity of treatment effects. With the wide range of users being treated over many online controlled experiments, the typical approach of manually…

统计方法学 · 统计学 2022-11-07 John Cai , Weinan Wang

Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

定量方法 · 定量生物学 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

In causal inference, estimating heterogeneous treatment effects (HTE) is critical for identifying how different subgroups respond to interventions, with broad applications in fields such as precision medicine and personalized advertising.…

机器学习 · 计算机科学 2024-07-02 Jiehui Zhou , Linxiao Yang , Xingyu Liu , Xinyue Gu , Liang Sun , Wei Chen

Breakthroughs in cancer biology have defined new research programs emphasizing the development of therapies that target specific pathways in tumor cells. Innovations in clinical trial design have followed with master protocols defined by…

统计方法学 · 统计学 2020-07-09 Alexander M. Kaizer , Joseph S. Koopmeiners , Nan Chen , Brian P. Hobbs

Understanding treatment effect heterogeneity is vital for scientific and policy research. However, identifying and evaluating heterogeneous treatment effects pose significant challenges due to the typically unknown subgroup structure.…

统计方法学 · 统计学 2024-11-05 Kwangho Kim , Jisu Kim , Larry A. Wasserman , Edward H. Kennedy

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

统计方法学 · 统计学 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

Educational research often studies subjects that are in naturally clustered groups of classrooms or schools. When designing a randomized experiment to evaluate an intervention directed at teachers, but with effects on teachers and their…

应用统计 · 统计学 2009-08-17 Brenda Jenney , Sharon Lohr

In randomized controlled trials (RCTs), treatment is often assigned by stratified randomization. I show that among all stratified randomization schemes which treat all units with probability one half, a certain matched-pair design achieves…

计量经济学 · 经济学 2022-06-17 Yuehao Bai