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Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

统计方法学 · 统计学 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Randomized controlled trials (RCTs) in oncology often allow control group participants to crossover to experimental treatments, a practice that, while often ethically necessary, complicates the accurate estimation of long-term treatment…

统计方法学 · 统计学 2025-02-12 Harlan Campbell , Nicholas Latimer , Jeroen P Jansen , Shannon Cope

Randomized controlled trials (RCTs) are often underpowered to detect treatment heterogeneity in subgroups defined by cross-classifications of multiple covariates, due to sparse sample sizes in some strata. External RCT data can help, but…

统计方法学 · 统计学 2026-04-23 Youqi Yang , Walter Dempsey , Bhramar Mukherjee

The least trimmed squares (LTS) estimator is a renowned robust alternative to the classic least squares estimator and is popular in location, regression, machine learning, and AI literature. Many studies exist on LTS, including its…

机器学习 · 统计学 2025-01-10 Yijun Zuo

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

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

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

机器学习 · 统计学 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

In multi-site randomized trials with many sites and few randomization units per site, an Empirical-Bayes estimator can be used to estimate the variance of the treatment effect across sites. When this estimator indicates that treatment…

计量经济学 · 经济学 2024-12-12 Clément de Chaisemartin , Antoine Deeb

We consider the estimation of the average treatment effect in the treated as a function of baseline covariates, where there is a valid (conditional) instrument. We describe two doubly robust (DR) estimators: a locally efficient g-estimator,…

统计方法学 · 统计学 2019-06-11 Karla DiazOrdaz , Rhian Daniel , Noemi Kreif

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

Under treatment effect heterogeneity, an instrument identifies the instrument-specific local average treatment effect (LATE). With multiple instruments, two-stage least squares (2SLS) estimand is a weighted average of different LATEs. What…

计量经济学 · 经济学 2026-02-03 Seojeong Lee

Stepped-wedge cluster randomized trials (SW-CRTs) evaluate interventions rolled out across clusters over time. Standard analyses typically use immediate-treatment (IT) models, which assume effects begin at crossover and remain constant…

统计方法学 · 统计学 2026-04-21 Yongdong Ouyang , Monica Taljaard , James P. Hughes , Fan Li

This paper studies inference in cluster randomized trials where treatment status is determined according to a "matched pairs" design. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the level of the…

计量经济学 · 经济学 2025-08-14 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

This paper studies inference in two-stage randomized experiments under covariate-adaptive randomization. In the initial stage of this experimental design, clusters (e.g., households, schools, or graph partitions) are stratified and randomly…

计量经济学 · 经济学 2026-01-16 Jizhou Liu

The micro-randomized trial (MRT) is a sequential randomized experimental design to empirically evaluate the effectiveness of mobile health (mHealth) intervention components that may be delivered at hundreds or thousands of decision points.…

统计方法学 · 统计学 2021-12-14 Jieru Shi , Zhenke Wu , Walter Dempsey

We propose and discuss a Bayesian procedure to estimate the average treatment effect (ATE) for multilevel observations in the presence of confounding. We focus on situations where the confounders may be latent (e.g., spatial latent…

统计方法学 · 统计学 2023-04-26 Widemberg S. Nobre , Alexandra M. Schmidt , Erica E. M. Moodie , David A. Stephens

In the analysis of cluster randomized trials (CRTs), previous work has defined two meaningful estimands: the individual-average treatment effect (iATE) and cluster-average treatment effect (cATE) estimand, to address individual and…

We address estimation of intervention effects in experimental designs in which (a) interventions are assigned at the cluster level; (b) clusters are selected to form pairs, matched on observed characteristics; and (c) intervention is…

统计方法学 · 统计学 2014-11-24 Zhenke Wu , Constantine E. Frangakis , Thomas A. Louis , Daniel O. Scharfstein

In a cluster-randomized experiment, treatment is assigned to clusters of individual units of interest--households, classrooms, villages, etc.--instead of the units themselves. The number of clusters sampled and the number of units sampled…

统计方法学 · 统计学 2020-02-20 Yeng Xiong , Michael J. Higgins

In matched-pairs experiments in which one cluster per pair of clusters is assigned to treatment, to estimate treatment effects, researchers often regress their outcome on a treatment indicator and pair fixed effects, clustering standard…

计量经济学 · 经济学 2023-06-29 Clément de Chaisemartin , Jaime Ramirez-Cuellar