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The first step towards investigating the effectiveness of a treatment via a randomized trial is to split the population into control and treatment groups then compare the average response of the treatment group receiving the treatment to…

Econometrics · Economics 2022-08-30 Hossein Babaei , Sina Alemohammad , Richard Baraniuk

Regression adjustment is broadly applied in randomized trials under the premise that it usually improves the precision of a treatment effect estimator. However, previous work has shown that this is not always true. To further understand…

Methodology · Statistics 2022-10-11 Katarzyna Reluga , Ting Ye , Qingyuan Zhao

A crucial task for a randomized controlled trial (RCT) is to specify a statistical method that can yield an efficient estimator and powerful test for the treatment effect. A novel and effective strategy to obtain efficient and powerful…

Randomized clinical trials are the gold standard when estimating the average treatment effect. However, they are usually not a random sample from the real-world population because of the inclusion/exclusion rules. Meanwhile, observational…

Methodology · Statistics 2024-12-11 Kuan Jiang , Wenjie Hu , Shu Yang , Xinxing Lai , Xiaohua Zhou

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

Paired cluster-randomized experiments (pCRTs) are common across many disciplines because there is often natural clustering of individuals, and paired randomization can help balance baseline covariates to improve experimental precision.…

Methodology · Statistics 2024-07-03 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

Baseline covariates in randomized experiments are often used in the estimation of treatment effects, for example, when estimating treatment effects within covariate-defined subgroups. In practice, however, covariate values may be missing…

Methodology · Statistics 2023-08-29 Gauri Kamat , Jerome P. Reiter

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

We propose the covariate-balanced-and-adjusted response-adaptive randomization (CBARA) procedure for adaptive design in clinical trials, which integrates the complementary strengths of covariate-adjusted response-adaptive randomization…

Methodology · Statistics 2026-05-08 Hengjia Fang , Wei Ma

The purpose of this work is to improve the efficiency in estimating the average causal effect (ACE) on the survival scale where right-censoring exists and high-dimensional covariate information is available. We propose new estimators using…

Methodology · Statistics 2021-06-29 Ran Dai , Cheng Zheng , Mei-Jie Zhang

In the context of precision medicine, covariate-adjusted response-adaptive randomization (CARA) has garnered much attention from both academia and industry due to its benefits in providing ethical and tailored treatment assignments based on…

Methodology · Statistics 2024-11-26 Jiahui Xin , Wei Ma

Delayed separation of survival curves is a common occurrence in confirmatory studies in immuno-oncology. Many novel statistical methods that aim to efficiently capture potential long-term survival improvements have been proposed in recent…

Methodology · Statistics 2022-01-26 Dominic Magirr , José L. Jiménez

Ordering the expected outcomes across a collection of clusters after performing a covariate adjustment commonly arises in many applied settings, such as healthcare provider evaluation. Regression parameters in such covariate adjustment…

Methodology · Statistics 2025-11-21 Nicholas C. Henderson , Nicholas Hartman

This paper studies inference on the average treatment effect in experiments in which treatment status is determined according to "matched pairs" and it is additionally desired to adjust for observed, baseline covariates to gain further…

Econometrics · Economics 2023-10-20 Yuehao Bai , Liang Jiang , Joseph P. Romano , Azeem M. Shaikh , Yichong Zhang

This paper studies the use of highly stratified designs for the efficient estimation of a large class of treatment effect parameters that arise in the analysis of experiments. By a "highly stratified" design, we mean experiments in which…

Econometrics · Economics 2026-03-30 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

Empirical researchers often use diagnostic checks to assess the plausibility of their modeling assumptions, such as testing for covariate balance in RCTs, pre-trends in event studies, or instrument validity in IV designs. While these checks…

Econometrics · Economics 2026-04-21 Reca Sarfati , Vod Vilfort

When investigators seek to estimate causal effects, they often assume that selection into treatment is based only on observed covariates. Under this identification strategy, analysts must adjust for observed confounders. While basic…

Applications · Statistics 2019-01-09 Luke Keele , Dylan Small

Randomized experiments are the gold standard for estimating treatment effects, and randomization serves as a reasoned basis for inference. In widely used stratified randomized experiments, randomization-based finite-population asymptotic…

Statistics Theory · Mathematics 2026-05-20 Haoyang Yu , Ke Zhu , Hanzhong Liu

Modern social and biomedical scientific publications require the reporting of covariate balance tables with not only covariate means by treatment group but also the associated $p$-values from significance tests of their differences. The…

Methodology · Statistics 2023-06-06 Anqi Zhao , Peng Ding

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…