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Sample size determination for cluster randomised trials (CRTs) is challenging as it requires robust estimation of the intra-cluster correlation coefficient (ICC). Typically, the sample size is chosen to provide a certain level of power to…

应用统计 · 统计学 2023-08-23 S. Faye Williamson , Svetlana V. Tishkovskaya , Kevin J. Wilson

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

统计方法学 · 统计学 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

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

Stepped wedge cluster randomized controlled trials are typically analyzed using models that assume the full effect of the treatment is achieved instantaneously. We provide an analytical framework for scenarios in which the treatment effect…

统计方法学 · 统计学 2025-09-25 Avi Kenny , Emily Voldal , Fan Xia , Patrick J. Heagerty , James P. Hughes

Hybrid type 2 studies are gaining popularity for their ability to assess both implementation and health outcomes as co-primary endpoints. Often conducted as cluster-randomized trials (CRTs), five design methods can validly power these…

统计方法学 · 统计学 2026-05-18 Melody Owen , Fan Li , Ruyi Liu , Donna Spiegelman

Cluster-randomized trials (CRTs) are widely used to evaluate interventions delivered at the clinic, practice, or community level. Although standard analyses typically target average treatment effects, such summaries mask potentially…

统计方法学 · 统计学 2026-04-16 Changjun Li , Xi Fang , Michael O. Harhay , Andrew B. Forbes , F. Perry Wilson , Guangyu Tong , Fan Li

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

Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…

统计方法学 · 统计学 2026-04-21 Xi Fang , Guangyu Tong , Yuan Huang , F. Perry Wilson , Patrick J. Heagerty , Fan Li

We address estimation of intervention effects in experimental designs in which (a) interventions are assigned at the cluster level; (b) clusters are selected to form pairs, matched on observed characteristics; and (c) intervention is…

统计方法学 · 统计学 2014-11-24 Zhenke Wu , Constantine E. Frangakis , Thomas A. Louis , Daniel O. Scharfstein

Generalized linear mixed models (GLMM) are commonly used to analyze clustered data, but when the number of clusters is small to moderate, standard statistical tests may produce elevated type I error rates. Small-sample corrections have been…

统计方法学 · 统计学 2023-11-07 Hongxiang Qiu , Andrea J. Cook , Jennifer F. Bobb

Cluster randomized trials with measurements at baseline can improve power over post-test only designs by using difference in difference designs. However, subjects may be lost to follow-up between the baseline and follow-up periods. While…

统计方法学 · 统计学 2019-03-26 Jonathan Moyer , Ken Kleinman

When designing experimental studies with human participants, experimenters must decide how many trials each participant will complete, as well as how many participants to test. Most discussion of statistical power (the ability of a study…

神经元与认知 · 定量生物学 2021-08-30 Daniel H. Baker , Greta Vilidaite , Freya A. Lygo , Anika K. Smith , Tessa R. Flack , Andre D. Gouws , Timothy J. Andrews

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

Paired cluster-randomized experiments (pCRTs) are common across many disciplines because there is often natural clustering of individuals, and paired randomization can help balance baseline covariates to improve experimental precision.…

统计方法学 · 统计学 2024-07-03 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

In the analysis of cluster randomized trials (CRTs), previous work has defined two meaningful estimands: the individual-average treatment effect (iATE) and cluster-average treatment effect (cATE) estimand, to address individual and…

We present an aggregation scheme that increases power in randomized controlled trials and quasi-experiments when the intervention possesses a robust and well-articulated theory of change. Longitudinal data analyzing interventions often…

统计方法学 · 统计学 2022-07-27 Timothy Lycurgus , Ben B. Hansen

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

统计方法学 · 统计学 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Cluster or group randomized trials (CRTs) are increasingly used for both behavioral and system-level interventions, where entire clusters are randomly assigned to a study condition or intervention. Apart from the assigned cluster-level…

统计方法学 · 统计学 2024-11-19 Shubhadeep Chakraborty , Bo Wang , Ram Tiwari , Samiran Ghosh

Staggered rollout cluster randomized experiments (SR-CREs) involve sequential treatment adoption across clusters, requiring analysis methods that address a general class of dynamic causal effects, anticipation, and non-ignorable…

统计方法学 · 统计学 2026-02-02 Xinyuan Chen , Fan Li

Mixed outcome endpoints that combine multiple continuous and discrete components to form co-primary, multiple primary or composite endpoints are often employed as primary outcome measures in clinical trials. There are many advantages to…

统计方法学 · 统计学 2019-12-12 Martina McMenamin , Jessica K. Barrett , Anna Berglind , James M. S. Wason