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Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

Randomized controlled trials are not only the golden standard in medicine and vaccine trials but have spread to many other disciplines like behavioral economics, making it an important interdisciplinary tool for scientists. When designing…

Methodology · Statistics 2021-11-30 Tassilo Schwarz

This paper provides asymptotically valid tests for the null hypothesis of no treatment effect heterogeneity. Importantly, I consider the presence of heterogeneity that is not explained by observed characteristics, or so-called idiosyncratic…

Econometrics · Economics 2023-04-04 Jaime Ramirez-Cuellar

We study the problem of treatment effect estimation in randomized experiments with high-dimensional covariate information, and show that essentially any risk-consistent regression adjustment can be used to obtain efficient estimates of the…

Methodology · Statistics 2022-06-08 Stefan Wager , Wenfei Du , Jonathan Taylor , Robert Tibshirani

We extend Fisher's randomization test (FRT) to test conditional independence between observed outcomes and treatments given covariates in both randomized experiments and observational studies, with no restriction on the variable type of…

Methodology · Statistics 2025-06-12 Zhen Zhong

Treatment-covariate interaction tests are commonly applied by researchers to examine whether the treatment effect varies across patient subgroups defined by baseline characteristics. The objective of this study is to explore…

Methodology · Statistics 2024-03-12 Likun Zhang , Wei Ma

This article studies randomization inference for treatment effects in randomized controlled trials with attrition, where outcomes are observed for only a subset of units. We assume monotonicity in reporting behavior as in…

Econometrics · Economics 2026-03-30 Haoge Chang , Zeyang Yu

A growing number of methods aim to assess the challenging question of treatment effect variation in observational studies. This special section of "Observational Studies" reports the results of a workshop conducted at the 2018 Atlantic…

Methodology · Statistics 2019-09-17 Carlos Carvalho , Avi Feller , Jared Murray , Spencer Woody , David Yeager

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Methodology · Statistics 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

In causal analysis, understanding the causal mechanisms through which an intervention or treatment affects an outcome is often of central interest. We propose a test to evaluate (i) whether the causal effect of a treatment that is randomly…

Econometrics · Economics 2026-03-05 Martin Huber , Kevin Kloiber , Lukáš Lafférs

Attrition is a common and potentially important threat to internal validity in treatment effect studies. We extend the changes-in-changes approach to identify the average treatment effect for respondents and the entire study population in…

Econometrics · Economics 2024-03-29 Dalia Ghanem , Sarojini Hirshleifer , Désiré Kédagni , Karen Ortiz-Becerra

Treatment effect heterogeneity is of a great concern when evaluating policy impact: "is the treatment Pareto-improving?", "what is the proportion of people who are better off under the treatment?", etc. However, even in the simple case of a…

Econometrics · Economics 2025-09-18 Myungkou Shin

Large observational data are increasingly available in disciplines such as health, economic and social sciences, where researchers are interested in causal questions rather than prediction. In this paper, we examine the problem of…

Methodology · Statistics 2021-11-24 Alberto Caron , Gianluca Baio , Ioanna Manolopoulou

A growing statistical literature focuses on causal inference in the context of experiments where the target of inference is the average treatment effect in a finite population and random assignment determines which subjects are allocated to…

Methodology · Statistics 2025-09-04 Jonas M. Mikhaeil , Donald P. Green

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

This paper considers conducting inference about the effect of a treatment (or exposure) on an outcome of interest. In the ideal setting where treatment is assigned randomly, under certain assumptions the treatment effect is identifiable…

Methodology · Statistics 2015-03-06 Amy Richardson , Michael G. Hudgens , Peter B. Gilbert , Jason P. Fine

In randomized experiments, covariates are often used to reduce variance and improve the precision of treatment effect estimates. However, in many real-world settings, interference between units, where one unit's treatment affects another's…

Methodology · Statistics 2026-04-10 Xinyi Wang , Shuangning Li

Randomization inference is a powerful tool in early phase vaccine trials when estimating the causal effect of a regimen against a placebo or another regimen. Randomization-based inference often focuses on testing either Fisher's sharp null…

Methodology · Statistics 2024-02-27 Zhe Chen , Xinran Li , Bo Zhang

Consider a situation with two treatments, the first of which is randomized but the second is not, and the multifactor version of this. Interest is in treatment effects, defined using standard factorial notation. We define estimators for the…

Tests for paired censored outcomes have been extensively studied, with some justified in the context of randomization-based inference. These tests are primarily designed to detect an overall treatment effect across the entire follow-up…

Methodology · Statistics 2025-06-10 Sangjin Lee , Kwonsang Lee