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Stepped-wedge cluster randomised trials (SW-CRTs) increasingly evaluate complex interventions, yet methodological guidance for analysing composite endpoints using generalized pairwise comparisons (GPC)remains limited. This work investigates…

Adaptive interventions, aka dynamic treatment regimens, are sequences of pre-specified decision rules that guide the provision of treatment for an individual given information about their baseline and evolving needs, including in response…

Methodology · Statistics 2024-05-02 Wenchu Pan , Daniel Almirall , Amy M. Kilbourne , Andrew Quanbeck , Lu Wang

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods…

Methodology · Statistics 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…

Methodology · Statistics 2023-07-20 Yunfan Li , Jessica L. Ross , Aaron M. Smith , David P. Miller

Latent variable models (LVMs) are commonly used in psychology and increasingly used for analyzing brain imaging data. Such studies typically involve a small number of participants (n<100), where standard asymptotic results often fail to…

Methodology · Statistics 2020-06-15 Brice Ozenne , Patrick M. Fisher , Esben Budtz-Jørgensen

In longitudinal panels and other regression models with unobserved effects, fixed effects estimation is often paired with cluster-robust variance estimation (CRVE) in order to account for heteroskedasticity and un-modeled dependence among…

Methodology · Statistics 2022-11-08 James E. Pustejovsky , Elizabeth Tipton

For robust statistical inference it is crucial to obtain a good estimator of the variance of the proposed estimator of the statistical estimand. A commonly used estimator of the variance for an asymptotically linear estimator is the sample…

Methodology · Statistics 2025-05-19 Yunwen Ji , Mark van der Laan , Alan Hubbard

Conditional independence tests are crucial across various disciplines in determining the independence of an outcome variable $Y$ from a treatment variable $X$, conditioning on a set of confounders $Z$. The Conditional Randomization Test…

Methodology · Statistics 2024-05-30 Bowen Xu , Yiwen Huang , Chuan Hong , Shuangning Li , Molei Liu

Mediation analysis has been comprehensively studied for independent data but relatively little work has been done for correlated data, especially for the increasingly adopted stepped wedge cluster randomized trials (SW-CRTs). Motivated by…

Methodology · Statistics 2025-04-03 Zhiqiang Cao , Fan Li

Cardiovascular outcome trials commonly face competing risks when non-CV death prevents observation of major adverse cardiovascular events (MACE). While Cox proportional hazards models treat competing events as independent censoring,…

Methodology · Statistics 2026-02-19 Tuo Wang , Yu Du

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is…

Methodology · Statistics 2024-11-18 Joe de Keizer , Rémi Lenain , Raphaël Porcher , Sarah Zoha , Arthur Chatton , Yohann Foucher

This work presents the first large-scale neutral benchmark experiment focused on single-event, right-censored, low-dimensional survival data. Benchmark experiments are essential in methodological research to scientifically compare new and…

Machine Learning · Statistics 2026-04-24 Lukas Burk , John Zobolas , Bernd Bischl , Andreas Bender , Marvin N. Wright , Raphael Sonabend

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…

Methodology · Statistics 2018-03-28 Michael Grayling , David Robertson , James Wason , Adrian Mander

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…

Methodology · Statistics 2024-11-19 Shubhadeep Chakraborty , Bo Wang , Ram Tiwari , Samiran Ghosh

The spatial interaction between two or more classes of points may cause spatial clustering patterns such as segregation or association, which can be tested using a nearest neighbor contingency table (NNCT). A NNCT is constructed using the…

Methodology · Statistics 2008-07-29 Elvan Ceyhan

Over the last five decades, we have seen strong methodological advances in survival analysis, mainly in two separate strands: One strand is based on a parametric approach that assumes some response distribution. More prominent, however, is…

Methodology · Statistics 2025-03-25 Sandra Siegfried , Bálint Tamási , Torsten Hothorn

This paper studies a cluster robust variance estimator proposed by Chiang, Hansen and Sasaki (2024) for linear panels. First, we show algebraically that this variance estimator (CHS estimator, hereafter) is a linear combination of three…

Econometrics · Economics 2024-08-26 Kaicheng Chen , Timothy J. Vogelsang

IMPORTANCE: Feature selection with respect to time-to-event outcomes is one of the fundamental problems in clinical trials and biomarker discovery studies. But it's unclear which statistical methods should be used when sample size is small…

Methodology · Statistics 2022-10-17 Rong Lu

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

The association between multidimensional exposure patterns and outcomes is commonly investigated by first applying cluster analysis algorithms to derive patterns and then estimating the associations. However, errors in the underlying…

Methodology · Statistics 2023-05-31 Timm Intemann , Iris Pigeot