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Controlled experiments are widely used in many applications to investigate the causal relationship between input factors and experimental outcomes. A completely randomized design is usually used to randomly assign treatment levels to…

统计方法学 · 统计学 2026-05-12 Yiou Li , Lulu Kang , Xiao Huang

This paper considers the problem of inference in cluster randomized experiments when cluster sizes are non-ignorable. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the cluster level. By…

计量经济学 · 经济学 2024-04-11 Federico Bugni , Ivan Canay , Azeem Shaikh , Max Tabord-Meehan

Attrition is a common occurrence in cluster randomised trials (CRTs) which leads to missing outcome data. Two approaches for analysing such trials are cluster-level analysis and individual-level analysis. This paper compares the performance…

统计方法学 · 统计学 2016-03-15 Anower Hossain , Karla Diaz-Ordaz , Jonathan W. Bartlett

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…

统计方法学 · 统计学 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

统计方法学 · 统计学 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Background: Stepped wedge cluster randomized trials (SW-CRTs) involve sequential measurements within clusters over time. Initially, all clusters start in the control condition before crossing over to the intervention on a staggered…

统计方法学 · 统计学 2026-01-21 Jale Basten , Katja Ickstadt , Nina Timmesfeld

Longitudinal cluster randomized trials (L-CRTs) are increasingly used to evaluate the cost-effectiveness of healthcare interventions across multiple assessment periods, yet design methods for powering these trials remain underdeveloped.…

统计方法学 · 统计学 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

The first step towards investigating the effectiveness of a treatment via a randomized trial is to split the population into control and treatment groups then compare the average response of the treatment group receiving the treatment to…

计量经济学 · 经济学 2022-08-30 Hossein Babaei , Sina Alemohammad , Richard Baraniuk

It is crucial to provide compatible treatment schemes for a disease according to various symptoms at different stages. However, most classification methods might be ineffective in accurately classifying a disease that holds the…

机器学习 · 计算机科学 2019-11-26 Jianguo Chen , Kenli Li , Huigui Rong , Kashif Bilal , Nan Yang , Keqin Li

In cluster-randomized trials, generalized linear mixed models and generalized estimating equations have conventionally been the default analytic methods for estimating the average treatment effect as routine practice. However, recent…

统计方法学 · 统计学 2025-09-19 Fan Li , Jiaqi Tong , Xi Fang , Chao Cheng , Brennan C. Kahan , Bingkai Wang

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

Dynamic treatment regimes (DTRs) are personalized, adaptive strategies designed to guide the sequential allocation of treatments based on individual characteristics over time. Before each treatment assignment, covariate information is…

统计方法学 · 统计学 2025-07-24 Kai Chen , Yuqian Zhang

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

Multivariate outcomes are common in pragmatic cluster randomized trials. While sample size calculation procedures for multivariate outcomes exist under parallel assignment, none have been developed for a stepped wedge design. In this…

统计方法学 · 统计学 2022-12-05 Kendra Davis-Plourde , Monica Taljaard , Fan Li

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

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

We consider the problem of model-based clustering in the presence of many correlated, mixed continuous and discrete variables, some of which may have missing values. Discrete variables are treated with a latent continuous variable approach…

The randomization inference literature studying randomized controlled trials (RCTs) assumes that units' potential outcomes are deterministic. This assumption is unlikely to hold, as stochastic shocks may take place during the experiment. In…

计量经济学 · 经济学 2022-12-15 Antoine Deeb , Clément de Chaisemartin

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

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