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To estimate casual treatment effects, we propose a new matching approach based on the reduced covariates obtained from sufficient dimension reduction. Compared to the original covariates and the propensity score, which are commonly used for…

Methodology · Statistics 2017-02-03 Wei Luo , Yeying Zhu

This study investigates treatment effect estimation in the semi-supervised setting, also can be interpreted as prediction-powered inference. In our setting, we can use not only the standard triple of covariates, treatment indicator, and…

Machine Learning · Statistics 2026-05-05 Masahiro Kato

Unblinded sample size re-estimation (SSR) is often planned in a clinical trial when there is large uncertainty about the true treatment effect. For Proof-of Concept (PoC) in a Phase II dose finding study, contrast test can be adopted to…

Methodology · Statistics 2022-11-11 Qingyang Liu , Guanyu Hu , Binqi Ye , Susan Wang , Yaoshi Wu

Two commonly used methods for improving precision and power in clinical trials are stratified randomization and covariate adjustment. However, many trials do not fully capitalize on the combined precision gains from these two methods, which…

Stepped wedge cluster randomized trials (SW-CRTs) have become increasingly popular and are used for a variety of interventions and outcomes, often chosen for their feasibility advantages. SW-CRTs must account for time trends in the outcome…

Methodology · Statistics 2024-07-16 Lee Kennedy-Shaffer , Victor De Gruttola , Marc Lipsitch

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…

Methodology · Statistics 2022-03-07 Thilo Welz , Wolfgang Viechtbauer , Markus Pauly

Unmeasured confounding is a threat to causal inference in observational studies. In recent years, use of negative controls to mitigate unmeasured confounding has gained increasing recognition and popularity. Negative controls have a…

Methodology · Statistics 2019-09-05 Xu Shi , Wang Miao , Jennifer C. Nelson , Eric J. Tchetgen Tchetgen

Loss of power and clear description of treatment differences are key issues in designing and analyzing a clinical trial where non-proportional hazard is a possibility. A log-rank test may be very inefficient and interpretation of the hazard…

Applications · Statistics 2021-01-13 Satrajit Roychoudhury , Keaven M Anderson , Jiabu Ye , Pralay Mukhopadhyay

Rerandomization is a modern experimental design technique that repeatedly randomizes treatment assignments until covariates are deemed balanced between treatment groups. This enhances the precision and coherence of causal effect estimators,…

Methodology · Statistics 2025-12-08 Antônio Carlos Herling Ribeiro Junior , Zach Branson

When treating depression, clinicians are interested in determining the optimal treatment for a given patient, which is challenging given the amount of treatments available. To advance individualized treatment allocation, integrating data…

In most nonrandomized observational studies, differences between treatment groups may arise not only due to the treatment but also because of the effect of confounders. Therefore, causal inference regarding the treatment effect is not as…

Methodology · Statistics 2018-07-04 Debashis Ghosh

Hybrid randomized controlled trials (hybrid RCTs) integrate external control data, such as historical or concurrent data, with data from randomized trials. While numerous frequentist and Bayesian methods, such as the test-then-pool and…

Methodology · Statistics 2025-10-07 Han Chang Chiam , Franz König , Martin Posch

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

Methodology · Statistics 2022-02-08 Melody Huang

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

Borrowing external data can improve estimation efficiency but may introduce bias when populations differ in covariate distributions or outcome variability. A proper balance needs to be maintained between the two datasets to justify the…

Randomized experiments are the "gold standard" for estimating causal effects, yet often in practice, chance imbalances exist in covariate distributions between treatment groups. If covariate data are available before units are exposed to…

Statistics Theory · Mathematics 2012-07-25 Kari Lock Morgan , Donald B. Rubin

There is a growing literature on design-based methods to estimate average treatment effects (ATEs) for randomized controlled trials (RCTs) for full sample analyses. This article extends these methods to estimate ATEs for discrete subgroups…

Methodology · Statistics 2023-10-16 Peter Z. Schochet

Random-effects meta-analyses are widely used for evidence synthesis in medical research. However, conventional methods based on large-sample approximations often exhibit poor performance in case of very few studies (e.g., 2 to 4), which is…

Methodology · Statistics 2025-11-20 Ao Huang , Christian Röver , Tim Friede

Causal decomposition analysis aims to assess the effect of modifying risk factors on reducing social disparities in outcomes. Recently, this analysis has incorporated individual characteristics when modifying risk factors by utilizing…

Machine Learning · Statistics 2025-09-16 Soojin Park , Suyeon Kang , Chioun Lee

We develop estimators that improve precision of heterogeneous treatment effect estimates that allow borrowing information from observational studies when the available covariates in each data source do not perfectly match. Standard…

Methodology · Statistics 2026-03-19 Samhita Pal , Jared D. Huling , Amir Asiaee