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Cluster randomized trials (CRTs) often enroll large numbers of participants, but due to logistical and fiscal challenges, only a subset of participants may be selected for measurement of certain outcomes, and those sampled may, purposely or…

统计方法学 · 统计学 2023-05-16 Joshua R. Nugent , Carina Marquez , Edwin D. Charlebois , Rachel Abbott , Laura B. Balzer

There is a growing literature on design-based methods to estimate average treatment effects for randomized controlled trials (RCTs) using the underpinnings of experiments. In this article, we build on these methods to consider design-based…

统计方法学 · 统计学 2024-01-17 Peter Z Schochet

Competing risk is a common phenomenon when dealing with time-to-event outcomes in biostatistical applications. An attractive estimand in this setting is the "number of life-years lost due to a specific cause of death", Andersen et al.…

统计方法学 · 统计学 2025-02-04 Simon Christoffer Ziersen , Torben Martinussen

Randomized experiments (or A/B tests) are widely used to evaluate interventions in dynamic systems such as recommendation platforms, marketplaces, and digital health. In these settings, interventions affect both current and future system…

统计方法学 · 统计学 2025-10-08 Ramesh Johari , Tianyi Peng , Wenqian Xing

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

统计方法学 · 统计学 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

Cluster randomized trails (CRT) have been widely employed in medical and public health research. Many clinical count outcomes, such as the number of falls in nursing homes, exhibit excessive zero values. In the presence of zero inflation,…

应用统计 · 统计学 2020-09-23 Zhengyang Zhou , Dateng Li , Song Zhang

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…

统计方法学 · 统计学 2025-04-03 Zhiqiang Cao , Fan Li

There are a number of available methods for selecting whom to prioritize for treatment, including ones based on treatment effect estimation, risk scoring, and hand-crafted rules. We propose rank-weighted average treatment effect (RATE)…

统计方法学 · 统计学 2023-11-30 Steve Yadlowsky , Scott Fleming , Nigam Shah , Emma Brunskill , Stefan Wager

The effects of treatments on continuous outcomes can be estimated by the mean difference (i.e. by measurement units) and the relative effect scales (i.e. by percentages), both of which provide only a single effect size estimate over the…

应用统计 · 统计学 2025-11-27 Harri Hemilä , Matti Pirinen

Common causal estimands include the average treatment effect (ATE), the average treatment effect of the treated (ATT), and the average treatment effect on the controls (ATC). Using augmented inverse probability weighting methods, parametric…

统计方法学 · 统计学 2022-09-23 Roland A. Matsouaka , Yi Liu , Yunji Zhou

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

Existing weighting methods for treatment effect estimation are often built upon the idea of propensity scores or covariate balance. They usually impose strong assumptions on treatment assignment or outcome model to obtain unbiased…

机器学习 · 计算机科学 2023-05-09 Dongcheng Zhang , Kunpeng Zhang

This study examines the problem of determining whether to treat individuals based on observed covariates. The most common decision rule is the conditional empirical success (CES) rule proposed by Manski (2004), which assigns individuals to…

计量经济学 · 经济学 2025-10-08 Takuya Ishihara , Daisuke Kurisu

Generalization methods offer a powerful solution to one of the key drawbacks of randomized controlled trials (RCTs): their limited representativeness. By enabling the transport of treatment effect estimates to target populations subject to…

统计方法学 · 统计学 2025-05-20 Ahmed Boughdiri , Clément Berenfeld , Julie Josse , Erwan Scornet

Recurrent events often serve as key endpoints in clinical studies but may be prematurely truncated by terminal events such as death, creating selection bias and complicating causal inference. To address this challenge, we develop a Bayesian…

统计方法学 · 统计学 2026-03-18 Yuki Ohnishi , Michael O. Harhay , Guangyu Tong , Fan Li

Causal inference is best understood using potential outcomes. This use is particularly important in more complex settings, that is, observational studies or randomized experiments with complications such as noncompliance. The topic of this…

统计理论 · 数学 2007-06-13 Donald B. Rubin

In the presence of treatment effect heterogeneity, the average treatment effect (ATE) in a randomized controlled trial (RCT) may differ from the average effect of the same treatment if applied to a target population of interest. If all…

统计方法学 · 统计学 2017-05-02 Trang Quynh Nguyen , Cyrus Ebnesajjad , Stephen R. Cole , Elizabeth A. Stuart

Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…

统计方法学 · 统计学 2026-04-21 Xi Fang , Guangyu Tong , Yuan Huang , F. Perry Wilson , Patrick J. Heagerty , Fan Li

In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the…

统计方法学 · 统计学 2025-11-06 Bingkai Wang , Fan Li , Rui Wang

Standard approaches in generalizability often focus on generalizing the intent-to-treat (ITT). However, in practice, a more policy-relevant quantity is the generalized impact of an intervention across compliers. While instrumental variable…

统计方法学 · 统计学 2025-06-03 Zhongren Chen , Melody Huang