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The literature on cluster-randomized trials typically allows for interference within but not across clusters. This may be implausible when units are irregularly distributed across space without well-separated communities, as clusters in…

Methodology · Statistics 2025-10-29 Michael P. Leung

Multilevel models (mixed-effect models or hierarchical linear models) are now a standard approach to analysing clustered and longitudinal data in the social, behavioural and medical sciences. This review article focuses on multilevel linear…

Methodology · Statistics 2019-07-16 George Leckie

Causal mediation analysis in cluster-randomized trials (CRTs) is essential for explaining how cluster-level interventions affect individual outcomes, yet it is complicated by interference, post-treatment confounding, and hierarchical…

Methodology · Statistics 2026-03-25 Yuki Ohnishi , Michael J. Daniels , Lei Yang , Fan Li

External controls (ECs) from historical trials or real-world data have gained increasing attention as a way to augment hybrid and single-arm trials, especially when balanced randomization is infeasible. While most existing work has focused…

Methodology · Statistics 2025-12-15 Yujing Gao , Xiang Zhang , Shu Yang

Background A key requirement for a useful power calculation is that the calculation mimic the data analysis that will be performed on the actual data, once it is observed. Close approximations may be difficult to achieve using analytic…

Applications · Statistics 2014-10-15 Ken Kleinman , Susan S. Huang

Cluster randomized trials (CRTs) are popular in public health and in the social sciences to evaluate a new treatment or policy where the new policy is randomly allocated to clusters of units rather than individual units. CRTs often feature…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

Linear mixed models are commonly used in analyzing stepped-wedge cluster randomized trials (SW-CRTs). A key consideration for analyzing a SW-CRT is accounting for the potentially complex correlation structure, which can be achieved by…

Methodology · Statistics 2024-08-21 Yongdong Ouyang , Monica Taljaard , Andrew B Forbes , Fan Li

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…

Methodology · Statistics 2026-02-02 Xinyuan Chen , Fan Li

The ability to accurately estimate the sample size required by a stepped-wedge (SW) cluster randomized trial (CRT) routinely depends upon the specification of several nuisance parameters. If these parameters are mis-specified, the trial…

Methodology · Statistics 2017-10-10 Michael Grayling , Adrian Mander , James Wason

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

The presence of interference, where the outcome of an individual may depend on the treatment assignment and behavior of neighboring nodes, can lead to biased causal effect estimation. Current approaches to network experiment design focus on…

Machine Learning · Computer Science 2024-05-22 Zahra Fatemi , Jean Pouget-Abadie , Elena Zheleva

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…

Methodology · Statistics 2018-12-05 Schadrac C. Agbla , Bianca De Stavola , Karla DiazOrdaz

A/B testing is a standard approach for evaluating the effect of online experiments; the goal is to estimate the `average treatment effect' of a new feature or condition by exposing a sample of the overall population to it. A drawback with…

Social and Information Networks · Computer Science 2013-05-31 Johan Ugander , Brian Karrer , Lars Backstrom , Jon Kleinberg

Power and sample size calculations for Wald tests in generalized linear models (GLMs) are often limited to specific cases like logistic regression. More general methods typically require detailed study parameters that are difficult to…

Methodology · Statistics 2026-01-21 Amy L Cochran , Shijie Yuan , Paul J Rathouz

We abstract the concept of a randomized controlled trial (RCT) as a triple (beta,b,s), where beta is the primary efficacy parameter, b the estimate and s the standard error (s>0). The parameter beta is either a difference of means, a log…

Methodology · Statistics 2020-12-01 Erik van Zwet , Simon Schwab , Stephen Senn

The roles of different nodes within a network are often understood through centrality analysis, which aims to quantify the capacity of a node to influence, or be influenced by, other nodes via its connection topology. Many different…

Social and Information Networks · Computer Science 2020-07-01 Stuart Oldham , Ben Fulcher , Linden Parkes , Aurina Arnatkeviciute , Chao Suo , Alex Fornito

In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the…

Methodology · Statistics 2025-11-06 Bingkai Wang , Fan Li , Rui Wang

Stepped wedge cluster randomized trials (SW-CRTs) have historically been analyzed using immediate treatment (IT) models, which assume the effect of the treatment is immediate after treatment initiation and subsequently remains constant over…

We introduce a novel statistical significance-based approach for clustering hierarchical data using semi-parametric linear mixed-effects models designed for responses with laws in the exponential family (e.g., Poisson and Bernoulli). Within…

Methodology · Statistics 2025-02-04 Alessandra Ragni , Chiara Masci , Francesca Ieva , Anna Maria Paganoni

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye
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