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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…

Machine Learning · Computer Science 2023-05-09 Dongcheng Zhang , Kunpeng Zhang

In cluster randomized trials, patients are typically recruited after clusters are randomized, and the recruiters and patients may not be blinded to the assignment. This often leads to differential recruitment and consequently systematic…

Methodology · Statistics 2021-10-05 Fan Li , Zizhong Tian , Jennifer Bobb , Georgia Papadogeorgou , Fan Li

There are many settings where researchers are interested in estimating average treatment effects and are willing to rely on the unconfoundedness assumption, which requires that the treatment assignment be as good as random conditional on…

Methodology · Statistics 2018-02-02 Susan Athey , Guido W. Imbens , Stefan Wager

Reliable uncertainty estimates are an important tool for helping autonomous agents or human decision makers understand and leverage predictive models. However, existing approaches to estimating uncertainty largely ignore the possibility of…

Machine Learning · Computer Science 2020-05-22 Sangdon Park , Osbert Bastani , James Weimer , Insup Lee

Covariate-adjusted randomization (CAR) can reduce the risk of covariate imbalance and, when accounted for in analysis, increase the power of a trial. Despite CAR advances, stratified randomization remains the most common CAR method. Matched…

Methodology · Statistics 2023-07-13 Jonathan J. Chipman , Lindsay Mayberry , Robert A. Greevy

Covariate adjustment is a general method for improving precision when estimating treatment effects in randomized trials and is recommended by the FDA in its 2023 guidance when baseline variables are prognostic for the primary outcome. We…

A basic principle in the design of observational studies is to approximate the randomized experiment that would have been conducted under controlled circumstances. Now, linear regression models are commonly used to analyze observational…

Methodology · Statistics 2022-07-08 Ambarish Chattopadhyay , Jose R. Zubizarreta

We present machine learning estimators for causal and predictive parameters under covariate shift, where covariate distributions differ between training and target populations. One such parameter is the average effect of a policy that…

Methodology · Statistics 2025-09-23 Victor Chernozhukov , Michael Newey , Whitney K Newey , Rahul Singh , Vasilis Syrgkanis

Causal inferences from a randomized controlled trial (RCT) may not pertain to a target population where some effect modifiers have a different distribution. Prior work studies generalizing the results of a trial to a target population with…

Machine Learning · Statistics 2024-06-06 Ilker Demirel , Ahmed Alaa , Anthony Philippakis , David Sontag

Estimation of the average treatment effect (ATE) is a central problem in causal inference. In recent times, inference for the ATE in the presence of high-dimensional covariates has been extensively studied. Among the diverse approaches that…

Statistics Theory · Mathematics 2022-11-01 Kuanhao Jiang , Rajarshi Mukherjee , Subhabrata Sen , Pragya Sur

We propose generalized additive partial linear models for complex data which allow one to capture nonlinear patterns of some covariates, in the presence of linear components. The proposed method improves estimation efficiency and increases…

Statistics Theory · Mathematics 2014-05-26 Li Wang , Lan Xue , Annie Qu , Hua Liang

Randomized controlled trials (RCTs) are the gold standard for estimating heterogeneous treatment effects, yet they are often underpowered for detecting effect heterogeneity. Large observational studies (OS) can supplement RCTs for…

Machine Learning · Computer Science 2026-04-07 Amir Asiaee , Samhita Pal

Inferring individualised treatment effects from observational data can unlock the potential for targeted interventions. It is, however, hard to infer these effects from observational data. One major problem that can arise is covariate shift…

Machine Learning · Computer Science 2024-04-25 Damian Machlanski , Spyros Samothrakis , Paul Clarke

In this article, we study nonparametric inference for a covariate-adjusted regression function. This parameter captures the average association between a continuous exposure and an outcome after adjusting for other covariates. In…

Methodology · Statistics 2023-12-18 Kenta Takatsu , Ted Westling

Background: In clinical research, the Bland-Altman analysis is commonly used to assess agreement of metric measurements made by two or more techniques, devices or methods. The approach can also deal with repeated measurements per subject or…

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Analysts are often confronted with censoring, wherein some variables are not observed at their true value, but rather at a value that is known to fall above or below that truth. While much attention has been given to the analysis of…

Methodology · Statistics 2024-05-31 Sarah C. Lotspeich , Kyle F. Grosser , Tanya P. Garcia

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power…

Covariate adjustment has the potential to increase power in the analysis of randomised trials, but mis-specification of the adjustment model could cause error. We explore what error is possible when the adjustment model omits a covariate by…

Methodology · Statistics 2021-07-16 Ian R. White , Tim P Morris , Elizabeth Williamson

Causal inference methods have been applied in various fields where researchers want to estimate treatment effects. In traditional causal inference settings, one assumes that the outcome of a unit does not depend on treatments of other…

Methodology · Statistics 2022-09-05 Kelly Kung , Daniel L. Sussman
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