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In cluster randomized trials, patients are typically recruited after clusters are randomized, and the recruiters and patients may not be blinded to the assignment. This often leads to differential recruitment and consequently systematic…

统计方法学 · 统计学 2021-10-05 Fan Li , Zizhong Tian , Jennifer Bobb , Georgia Papadogeorgou , 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

Evaluating blocked randomized experiments from a potential outcomes perspective has two primary branches of work. The first focuses on larger blocks, with multiple treatment and control units in each block. The second focuses on matched…

统计方法学 · 统计学 2024-05-31 Nicole E. Pashley , Luke W. Miratrix

Recently, there as been an increasing interest in the use of heavily restricted randomization designs which enforces balance on observed covariates in randomized controlled trials. However, when restrictions are strict, there is a risk that…

统计方法学 · 统计学 2021-10-15 Mattias Nordin , Mårten Schultzberg

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

The parallel cluster randomized trial with baseline (PB-CRT) is a common variant of the standard parallel cluster randomized trial (P-CRT). We define two natural estimands in the context of PB-CRTs with informative cluster sizes, the…

统计方法学 · 统计学 2025-05-01 Kenneth Menglin Lee , Fan Li

We study how to efficiently estimate average treatment effects (ATEs) using adaptive experiments. In adaptive experiments, experimenters sequentially assign treatments to experimental units while updating treatment assignment probabilities…

机器学习 · 统计学 2025-02-21 Masahiro Kato , Takuya Ishihara , Junya Honda , Yusuke Narita

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

In randomized controlled trials (RCTs), treatment is often assigned by stratified randomization. I show that among all stratified randomization schemes which treat all units with probability one half, a certain matched-pair design achieves…

计量经济学 · 经济学 2022-06-17 Yuehao Bai

Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation…

统计方法学 · 统计学 2025-01-30 Sky Qiu , Jens Tarp , Andrew Mertens , Mark van der Laan

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In…

统计方法学 · 统计学 2025-08-27 Dane Isenberg , Nandita Mitra , Steven C. Marcus , Rinad S. Beidas , Kristin A. Linn

This paper studies nonparametric identification and estimation of causal effects in centralized school assignment. In many centralized assignment algorithms, students face both lottery-driven variation and regression discontinuity- (RD)…

计量经济学 · 经济学 2025-12-30 Jiafeng Chen

Cluster randomized trials (CRTs) are studies where treatment is randomized at the cluster level but outcomes are typically collected at the individual level. When CRTs are employed in pragmatic settings, baseline population characteristics…

统计方法学 · 统计学 2023-06-22 Mary M. Ryan , Denise Esserman , Fan Li

In cluster randomized trials, the average treatment effect among individuals (i-ATE) can be different from the cluster average treatment effect (c-ATE) when informative cluster size is present, i.e., when treatment effects or participant…

统计方法学 · 统计学 2025-10-02 Bryan S. Blette , Zhe Chen , Brennan C. Kahan , Andrew Forbes , Michael O. Harhay , Fan Li

Given the unconfoundedness assumption, we propose new nonparametric estimators for the reduced dimensional conditional average treatment effect (CATE) function. In the first stage, the nuisance functions necessary for identifying CATE are…

计量经济学 · 经济学 2021-07-26 Qingliang Fan , Yu-Chin Hsu , Robert P. Lieli , Yichong Zhang

This study designs an adaptive experiment for efficiently estimating average treatment effects (ATEs). In each round of our adaptive experiment, an experimenter sequentially samples an experimental unit, assigns a treatment, and observes…

统计方法学 · 统计学 2024-06-21 Masahiro Kato , Akihiro Oga , Wataru Komatsubara , Ryo Inokuchi

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

统计方法学 · 统计学 2026-03-09 Samuel I. Watson , James Martin

Randomized controlled trials (RCTs) face inherent limitations, such as ethical or resource constraints, which lead to a limited number of study participants. To address these limitations, recent research endeavors have sought to incorporate…

统计方法学 · 统计学 2025-02-20 Peijin Wang , Hwanhee Hong , Kyungeun Jeon , Laine Elliott Thomas

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

Randomized controlled trials often enroll participants whose characteristics differ from those of a target population, which can limit the generalizability of the estimated treatment effects when effect modifiers differ across populations.…

统计方法学 · 统计学 2026-05-15 Lan Wen , Issa J. Dahabreh , Yu-Han Chiu