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In pragmatic cluster randomized controlled trials (PCRCTs), healthcare providers are randomized while both providers and patients may deviate from the assigned intervention. In many PCRCTs, cluster-level implementation is measured using…

Applications · Statistics 2026-04-29 Anthony Sisti , Ellen McCreedy , Roee Gutman

Motivated by a potential-outcomes perspective, the idea of principal stratification has been widely recognized for its relevance in settings susceptible to posttreatment selection bias such as randomized clinical trials where treatment…

Applications · Statistics 2011-11-08 Corwin M. Zigler , Thomas R. Belin

Accurately estimating personalized treatment effects within a study site (e.g., a hospital) has been challenging due to limited sample size. Furthermore, privacy considerations and lack of resources prevent a site from leveraging…

Machine Learning · Statistics 2022-06-17 Xiaoqing Tan , Chung-Chou H. Chang , Ling Zhou , Lu Tang

Clustering explores meaningful patterns in the non-labeled data sets. Cluster Ensemble Selection (CES) is a new approach, which can combine individual clustering results for increasing the performance of the final results. Although CES can…

Machine Learning · Computer Science 2016-04-26 Muhammad Yousefnezhad , Daoqiang Zhang

The primary analysis of randomized screening trials for cancer typically adheres to the intention-to-screen principle, measuring cancer-specific mortality reductions between screening and control arms. These mortality reductions result from…

Methodology · Statistics 2021-07-30 Sudipta Saha , Zhihui Liu , Olli Saarela

Treatment effect heterogeneity plays an important role in many areas of causal inference and within recent years, estimation of the conditional average treatment effect (CATE) has received much attention in the statistical community. While…

Methodology · Statistics 2024-12-17 Simon Christoffer Ziersen , Torben Martinussen

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…

Econometrics · Economics 2022-12-15 Antoine Deeb , Clément de Chaisemartin

Dropout is common in clinical studies, with up to half of patients leaving early due to side effects or other reasons. When dropout is informative (i.e., dependent on survival time), it introduces censoring bias, because of which treatment…

Machine Learning · Computer Science 2026-05-12 Yuxin Wang , Dennis Frauen , Jonas Schweisthal , Maresa Schröder , Stefan Feuerriegel

Propensity score weighting is a tool for causal inference to adjust for measured confounders in observational studies. In practice, data often present complex structures, such as clustering, which make propensity score modeling and…

Methodology · Statistics 2017-03-20 Shu Yang

In this study, we focus on estimating the heterogeneous treatment effect (HTE) for survival outcome. The outcome is subject to censoring and the number of covariates is high-dimensional. We utilize data from both the randomized controlled…

Methodology · Statistics 2025-03-21 Xin Ye , Shu Yang , Xiaofei Wang , Yanyan Liu

Confounding bias and selection bias bring two significant challenges to the validity of conclusions drawn from applied causal inference. The latter can stem from informative missingness, such as in cases of attrition. We introduce the…

Methodology · Statistics 2025-03-25 Johan de Aguas , Johan Pensar , Tomás Varnet Pérez , Guido Biele

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

Methodology · Statistics 2019-02-27 Anthony D. Scotina , Roee Gutman

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

Artificial Intelligence · Computer Science 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Outcome-dependent sampling designs are extensively utilized in various scientific disciplines, including epidemiology, ecology, and economics, with retrospective case-control studies being specific examples of such designs. Additionally, if…

Methodology · Statistics 2023-09-22 Min Zeng , Zeyang Jia , Zijian Sui , Jinfeng Xu , Hong Zhang

When estimating the treatment effect in an observational study, we use a semiparametric locally efficient dimension reduction approach to assess both the treatment assignment mechanism and the average responses in both treated and…

Methodology · Statistics 2020-10-26 Trinetri Ghosh , Yanyuan Ma , Xavier de Luna

We extend recently proposed design-based capture-recapture (CRC) methods for prevalence estimation among registry participants, in order to enhance treatment effect evaluation among a trial-eligible target population. The so-called ``anchor…

Methodology · Statistics 2025-10-28 Lin Ge , Yuzi Zhang , Lance A. Waller , Robert H. Lyles

The weighted average treatment effect (WATE) defines a versatile class of causal estimands for populations characterized by propensity score weights, including the average treatment effect (ATE), treatment effect on the treated (ATT), on…

Methodology · Statistics 2025-09-23 Yiming Wang , Yi Liu , Shu Yang

Stepped wedge cluster randomized trials (SWCRTs) often face challenges with potential confounding by time trends. Traditional frequentist methods can fail to provide adequate coverage of the intervention's true effect using confidence…

Methodology · Statistics 2024-02-02 Danni Wu , Hyung G. Park , Corita R. Grudzen , Keith S. Goldfeld

We extend methods for finite-sample inference about the average treatment effect (ATE) in randomized experiments with binary outcomes to accommodate stratification (blocking). We present three valid methods that differ in their…

Methodology · Statistics 2025-08-07 Jiaxun Li , Jacob Spertus , Philip B. Stark

Estimating conditional average treatment effects (CATE) from randomized controlled trials (RCTs) and generalizing them to broader populations is essential for personalizing treatment rules but is complicated by selection bias due to trial…

Methodology · Statistics 2026-05-15 Rikuta Hamaya , Etsuji Suzuki , Konan Hara