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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…

Methodology · Statistics 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

Despite their cost, randomized controlled trials (RCTs) are widely regarded as gold-standard evidence in disciplines ranging from social science to medicine. In recent decades, researchers have increasingly sought to reduce the resource…

Methodology · Statistics 2026-02-24 Guilherme Duarte

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…

Methodology · Statistics 2025-05-20 Ahmed Boughdiri , Clément Berenfeld , Julie Josse , Erwan Scornet

Synthetic Control methods have recently gained considerable attention in applications with only one treated unit. Their popularity is partly based on the key insight that we can predict good synthetic counterfactuals for our treated unit.…

Econometrics · Economics 2025-07-15 Tzvetan Moev

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

One of the major challenges in estimating conditional potential outcomes and conditional average treatment effects (CATE) is the presence of hidden confounders. Since testing for hidden confounders cannot be accomplished only with…

Machine Learning · Computer Science 2025-06-17 Ahmed Aloui , Juncheng Dong , Ali Hasan , Vahid Tarokh

In empirical studies with time-to-event outcomes, investigators often leverage observational data to conduct causal inference on the effect of exposure when randomized controlled trial data is unavailable. Model misspecification and lack of…

Methodology · Statistics 2023-05-05 Shenbo Xu , Bang Zheng , Bowen Su , Stan Finkelstein , Roy Welsch , Kenney Ng , Ioanna Tzoulaki , Zach Shahn

Progressive multi-state survival outcomes are common in trials with recurrent or sequential events and require treatment effect estimands that remain interpretable without proportional intensity or Markov assumptions. The restricted mean…

Methodology · Statistics 2026-01-22 Xi Fang , Bingkai Wang , Guangyu Tong , Liangyuan Hu , Shuangge Ma , Fan Li

We study causal effect estimation from a mixture of observational and interventional data in a confounded linear regression model with multivariate treatments. We show that the statistical efficiency in terms of expected squared error can…

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…

Methodology · Statistics 2024-01-17 Peter Z Schochet

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

Randomized control trials (RCTs) have been the gold standard to evaluate the effectiveness of a program, policy, or treatment on an outcome of interest. However, many RCTs assume that study participants are willing to share their…

Applications · Statistics 2021-12-07 Manjusha Kancharla , Hyunseung Kang

Covariate adjustment is widely recommended to improve statistical efficiency in randomized clinical trials (RCTs), yet empirical evidence comparing available strategies remains limited. This lack of real-world evaluation leaves unresolved…

Applications · Statistics 2026-02-03 Yulin Shao , Liangbo Lyu , Menggang Yu , Bingkai Wang

Many social and environmental phenomena are associated with macroscopic changes in the built environment, captured by satellite imagery on a global scale and with daily temporal resolution. While widely used for prediction, these images and…

Methodology · Statistics 2024-07-25 Connor T. Jerzak , Ritwik Vashistha , Adel Daoud

Although increasingly used for research, electronic health records (EHR) often lack gold-standard assessment of key data elements. Linking EHRs to other data sources with higher-quality measurements can improve statistical inference, but…

Methodology · Statistics 2025-03-05 Jenny Shen , Dane Isenberg , Kristin A. Linn , Rebecca A. Hubbard

Predicting risks of chronic diseases has become increasingly important in clinical practice. When a prediction model is developed in a given source cohort, there is often a great interest to apply the model to other cohorts. However, due to…

Methodology · Statistics 2020-03-05 Zheng Jiayin , Zheng Yingye , Hsu Li

Stepped-wedge cluster-randomized trials (SW-CRTs) are widely used in healthcare and implementation science, providing an ethical advantage by ensuring all clusters eventually receive the intervention. The staggered rollout of treatment…

Methodology · Statistics 2026-04-03 Xi Fang , Xueqi Wang , Patrick J. Heagerty , Bingkai Wang , Fan Li

Patient care may be improved by recommending treatments based on patient characteristics when there is treatment effect heterogeneity. Recently, there has been a great deal of attention focused on the estimation of optimal treatment rules…

Methodology · Statistics 2024-01-29 Michael Jetsupphasuk , Michael G. Hudgens , Jessie K. Edwards , Stephen R. Cole

The interpretation of randomised clinical trial results is often complicated by intercurrent events. For instance, rescue medication is sometimes given to patients in response to worsening of their disease, either in addition to the…

Methodology · Statistics 2021-02-12 Hege Michiels , Cristina Sotto , An Vandebosch , Stijn Vansteelandt

To take sample biases and skewness in the observations into account, practitioners frequently weight their observations according to some marginal distribution. The present paper demonstrates that such weighting can indeed improve the…

Methodology · Statistics 2018-11-05 Tobias Niebuhr , Mathias Trabs