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Beyond conditional average treatment effects, treatments may impact the entire outcome distribution in covariate-dependent ways, for example, by altering the variance or tail risks for specific subpopulations. We propose a novel estimand to…

Machine Learning · Statistics 2026-03-18 Saksham Jain , Alex Luedtke

In observational studies, weighting methods that directly optimize the balance between treatment and covariates have received much attention lately; however these have mainly focused on binary treatments. Inspired by domain adaptation, we…

Statistics Theory · Mathematics 2020-02-27 Guillaume Martinet

In most clinical trials, patients are randomized with equal probability among treatments to obtain an unbiased estimate of the treatment effect. Response-adaptive randomization (RAR) has been proposed for ethical reasons, where the…

Applications · Statistics 2019-09-16 Thevaa Chandereng , Rick Chappell

Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately…

Randomized Controlled Trials (RCTs) may suffer from limited scope. In particular, samples may be unrepresentative: some RCTs over- or under- sample individuals with certain characteristics compared to the target population, for which one…

Methodology · Statistics 2024-03-15 Bénédicte Colnet , Julie Josse , Gaël Varoquaux , Erwan Scornet

Rerandomization, a design that utilizes pretreatment covariates and improves their balance between different treatment groups, has received attention recently in both theory and practice. From a survey by Bruhn and McKenzie (2009), there…

Methodology · Statistics 2025-09-17 Yuhao Wang , Xinran Li

In the absence of unobserved confounders, matching and weighting methods are widely used to estimate causal quantities including the Average Treatment Effect on the Treated (ATT). Unfortunately, these methods do not necessarily achieve…

Methodology · Statistics 2016-05-03 Chad Hazlett

Complete randomization allows for consistent estimation of the average treatment effect based on the difference in means of the outcomes without strong modeling assumptions on the outcome-generating process. Appropriate use of the…

Methodology · Statistics 2021-08-03 Anqi Zhao , Peng Ding

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

Methodology · Statistics 2026-03-31 Zhe Chen , Xinran Li

The design of experiments involves a compromise between covariate balance and robustness. This paper provides a formalization of this trade-off and describes an experimental design that allows experimenters to navigate it. The design is…

Methodology · Statistics 2023-11-16 Christopher Harshaw , Fredrik Sävje , Daniel Spielman , Peng Zhang

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

We give an approach for characterizing interference by lower bounding the number of units whose outcome depends on selected groups of treated individuals, such as depending on the treatment of others, or others who are at least a certain…

Methodology · Statistics 2025-11-04 David Choi

Background: Subgroup analyses are frequently conducted in randomized clinical trials to assess evidence of heterogeneous treatment effect across patient subpopulations. Although randomization balances covariates within subgroups in…

Methodology · Statistics 2021-05-27 Siyun Yang , Fan Li , Laine E. Thomas , Fan Li

Covariate adjustment aims to improve the statistical efficiency of randomized trials by incorporating information from baseline covariates. Popular methods for covariate adjustment include analysis of covariance for continuous endpoints and…

Methodology · Statistics 2025-05-09 Zhiwei Zhang , Ya Wang , Dong Xi

We consider learning personalized assignments to one of many treatment arms from a randomized controlled trial. Standard methods that estimate heterogeneous treatment effects separately for each arm may perform poorly in this case due to…

Machine Learning · Statistics 2023-11-02 Rahul Ladhania , Jann Spiess , Lyle Ungar , Wenbo Wu

Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

Methodology · Statistics 2026-05-01 Elizabeth Tipton , Michalis Mamakos

In observational studies, propensity scores are commonly estimated by maxi- mum likelihood but may fail to balance high-dimensional pre-treatment covariates even after specification search. We introduce a general framework that unifies and…

Methodology · Statistics 2017-03-22 Qingyuan Zhao

A common concern in observational studies focuses on properly evaluating the causal effect, which usually refers to the average treatment effect or the average treatment effect on the treated. In this paper, we propose a data preprocessing…

Methodology · Statistics 2021-01-12 Xialing Wen , Ying Yan , Wenliang Pan , Xianyang Zhang

In recent years, real-world external controls have grown in popularity as a tool to empower randomized placebo-controlled trials, particularly in rare diseases or cases where balanced randomization is unethical or impractical. However, as…

Methodology · Statistics 2024-11-14 Chenyin Gao , Shu Yang , Mingyang Shan , Wenyu Ye , Ilya Lipkovich , Douglas Faries

We study optimal sample allocation between treatment and control groups under Bayesian linear models. We derive an analytic expression for the Bayes risk, which depends jointly on sample size and covariate mean balance across groups. Under…

Statistics Theory · Mathematics 2025-09-03 André A. F. Fumis , Victor Fossaluza , Rafael B. Stern