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Effect modification means the size of a treatment effect varies with an observed covariate. Generally speaking, a larger treatment effect with more stable error terms is less sensitive to bias. Thus, we might be able to conclude that a…

Methodology · Statistics 2026-05-19 Yijun Fan , Dylan S. Small

There is effect modification if the magnitude or stability of a treatment effect varies systematically with the level of an observed covariate. A larger or more stable treatment effect is typically less sensitive to bias from unmeasured…

Applications · Statistics 2016-12-12 Kwonsang Lee , Dylan S. Small , Jesse Y. Hsu , Jeffrey H. Silber , Paul R. Rosenbaum

Understanding effect modification -- how treatment effects vary across subpopulations -- is practically important in observational studies, as it helps identify which subgroups are likely to benefit from a given treatment. In this paper, we…

Methodology · Statistics 2026-05-12 Yu Gui , Dylan S Small , Zhimei Ren

There is strong interest in estimating how the magnitude of treatment effects of an intervention vary across sub-groups of the population of interest. In our paper, we propose a two-study approach to first propose and then test…

Methodology · Statistics 2020-06-23 Rahul Ladhania , Amelia Haviland , Neeraj Sood , Edward Kennedy , Ateev Mehrotra

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

Methodology · Statistics 2018-06-12 Rose Baker , Dan Jackson

One fundamental statistical question for research areas such as precision medicine and health disparity is about discovering effect modification of treatment or exposure by observed covariates. We propose a semiparametric framework for…

Methodology · Statistics 2020-08-04 Muxuan Liang , Menggang Yu

Effect modification occurs when the effect of the treatment on an outcome varies according to the level of other covariates and often has important implications in decision making. When there are tens or hundreds of covariates, it becomes…

Methodology · Statistics 2021-11-23 Qingyuan Zhao , Dylan S. Small , Ashkan Ertefaie

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

Suppose we have a binary treatment used to influence an outcome. Given data from an observational or controlled study, we wish to determine whether or not there exists some subset of observed covariates in which the treatment is more…

Methodology · Statistics 2016-03-22 Alexander R. Luedtke , Mark J. van der Laan

Estimating causal effects from observational data is not always possible due to confounding. Identifying a set of appropriate covariates (adjustment set) and adjusting for their influence can remove confounding bias; however, such a set is…

Methodology · Statistics 2020-11-19 Sofia Triantafillou , Gregory Cooper

Studies have shown that exposure to air pollution, even at low levels, significantly increases mortality. As regulatory actions are becoming prohibitively expensive, robust evidence to guide the development of targeted interventions to…

Applications · Statistics 2018-02-20 Kwonsang Lee , Dylan S. Small , Francesca Dominici

Causal effect moderation investigates how the effect of interventions (or treatments) on outcome variables changes based on observed characteristics of individuals, known as potential effect moderators. With advances in data collection,…

Methodology · Statistics 2024-11-26 Soham Bakshi , Walter Dempsey , Snigdha Panigrahi

Random-effects meta-analyses of observational studies can produce biased estimates if the synthesized studies are subject to unmeasured confounding. We propose sensitivity analyses quantifying the extent to which unmeasured confounding of…

Methodology · Statistics 2017-10-10 Maya B. Mathur , Tyler J. VanderWeele

A fundamental limitation of causal inference in observational studies is that perceived evidence for an effect might instead be explained by factors not accounted for in the primary analysis. Methods for assessing the sensitivity of a…

Methodology · Statistics 2018-09-14 Colin B. Fogarty

For testing the statistical significance of a treatment effect, we usually compare between two parts of a population, one is exposed to the treatment, and the other is not exposed to it. Standard parametric and nonparametric two-sample…

Computation · Statistics 2012-11-02 Bikram Karmakar , Kumaresh Dhara , Kushal Kumar Dey , Analabha Basu , Anil Ghosh

Identifying causal treatment (or exposure) effects in observational studies requires the data to satisfy the unconfoundedness assumption which is not testable using the observed data. With sensitivity analysis, one can determine how the…

Methodology · Statistics 2023-01-31 Yang Ou , Lu Tang , Chung-Chou H. Chang

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

We provide an approach to exploratory data analysis in matched observational studies with a single intervention and multiple endpoints. In such settings, the researcher would like to explore evidence for actual treatment effects among these…

Methodology · Statistics 2025-12-10 Mengqi Lin , Colin Fogarty

Matching is a widely used causal inference design that aims to approximate a randomized experiment using observational data by forming matched sets of treated and control units based on similarities in their covariates. Ideally, treated…

Methodology · Statistics 2026-04-06 Jianan Zhu , Jeffrey Zhang , Zijian Guo , Siyu Heng

Inferring the causal effect of a treatment on an outcome in an observational study requires adjusting for observed baseline confounders to avoid bias. However, adjusting for all observed baseline covariates, when only a subset are…

Methodology · Statistics 2021-02-04 Wen Wei Loh , Stijn Vansteelandt
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