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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

Most cluster randomized trials (CRTs) randomize fewer than 30-40 clusters in total. When performing inference for such ``small'' CRTs, it is important to use methods that appropriately account for the small sample size. When the generalized…

统计方法学 · 统计学 2025-12-01 Shifeng Sun , Xueqi Wang , Zhuoran Hou , Elizabeth L. Turner

Stepped wedge cluster randomized trials (SW-CRTs) are a form of randomized trial whereby clusters are progressively transitioned from control to intervention, with the timing of transition randomized for each cluster. An important task at…

统计方法学 · 统计学 2025-06-27 Mary Ryan Baumann , Denise Esserman , Monica Taljaard , Fan Li

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

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

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

Stepped wedge cluster randomized trials (SW-CRTs) with binary outcomes are increasingly used in prevention and implementation studies. Marginal models represent a flexible tool for analyzing SW-CRTs with population-averaged interpretations,…

统计方法学 · 统计学 2021-01-05 Fan Li , Hengshi Yu , Paul J. Rathouz , Elizabeth L. Turner , John S. Preisser

Difference in proportions is frequently used to measure treatment effect for binary outcomes in randomized clinical trials. The estimation of difference in proportions can be assisted by adjusting for prognostic baseline covariates to…

统计方法学 · 统计学 2023-08-31 Jialuo Liu , Dong Xi

A population-averaged additive subdistribution hazards model is proposed to assess the marginal effects of covariates on the cumulative incidence function and to analyze correlated failure time data subject to competing risks. This approach…

统计方法学 · 统计学 2023-08-23 Xinyuan Chen , Denise Esserman , Fan Li

Cluster-randomized trials (CRTs) are experimental designs where groups or clusters of participants, rather than the individual participants themselves, are randomized to intervention groups. Analyzing CRT requires distinguishing between…

统计方法学 · 统计学 2025-10-10 Xi Fang , Bingkai Wang , Liangyuan Hu , Fan Li

Meta-analyses frequently include trials that report multiple effect sizes based on a common set of study participants. These effect sizes will generally be correlated. Cluster-robust variance-covariance estimators are a fruitful approach…

统计方法学 · 统计学 2022-03-07 Thilo Welz , Wolfgang Viechtbauer , Markus Pauly

Massive sized survival datasets are becoming increasingly prevalent with the development of the healthcare industry. Such datasets pose computational challenges unprecedented in traditional survival analysis use-cases. A popular way for…

统计方法学 · 统计学 2023-05-09 Nir Keret , Malka Gorfine

In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs…

统计方法学 · 统计学 2022-09-07 Xueqi Wang , Elizabeth L. Turner , John S. Preisser , Fan Li

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

应用统计 · 统计学 2019-02-04 Timothy NeCamp , Josh Gardner , Christopher Brooks

Cluster-randomized experiments are increasingly used to evaluate interventions in routine practice conditions, and researchers often adopt model-based methods with covariate adjustment in the statistical analyses. However, the validity of…

统计方法学 · 统计学 2023-12-08 Bingkai Wang , Chan Park , Dylan S. Small , Fan Li

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…

统计方法学 · 统计学 2024-08-21 Yongdong Ouyang , Monica Taljaard , Andrew B Forbes , Fan Li

Modern computational advances have enabled easy parallel implementations of Markov chain Monte Carlo (MCMC). However, almost all work in estimating the variance of Monte Carlo averages, including the efficient batch means (BM) estimator,…

统计方法学 · 统计学 2024-07-23 Kushagra Gupta , Dootika Vats

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

统计方法学 · 统计学 2026-01-22 Xinyuan Chen , Fan Li

The case-cohort design allows analysis of multiple endpoints and only requires covariates to be measured for cases and non-cases in a random subcohort from the cohort. Stratification of subcohort sampling and weight calibration increase…

应用统计 · 统计学 2024-02-15 Lola Etievant , Mitchell H. Gail

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as…

统计方法学 · 统计学 2026-05-19 Chengxin Yang , Bo Liu , Fan Li
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