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Mendelian randomization (MR) is a widely-used method to estimate the causal relationship between a risk factor and disease. A fundamental part of any MR analysis is to choose appropriate genetic variants as instrumental variables.…

Methodology · Statistics 2023-04-26 Ashish Patel , Francis J. DiTraglia , Verena Zuber , Stephen Burgess

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

In a cluster-randomized experiment, treatment is assigned to clusters of individual units of interest--households, classrooms, villages, etc.--instead of the units themselves. The number of clusters sampled and the number of units sampled…

Methodology · Statistics 2020-02-20 Yeng Xiong , Michael J. Higgins

The method of instrumental variables provides a fundamental and practical tool for causal inference in many empirical studies where unmeasured confounding between the treatments and the outcome is present. Modern data such as the genetical…

Methodology · Statistics 2022-10-28 Ziang Niu , Yuwen Gu , Wei Li

Instrumental Variables provide a way of addressing bias due to unmeasured confounding when estimating treatment effects using observational data. As instrument prescription preference of individual healthcare providers has been proposed.…

Methodology · Statistics 2023-08-17 Laura M. Güdemann , Beverley M. Shields , John M. Dennis , Jack Bowden

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

Inference for causal effects can benefit from the availability of an instrumental variable (IV) which, by definition, is associated with the given exposure, but not with the outcome of interest other than through a causal exposure effect.…

Methodology · Statistics 2012-01-13 Stijn Vansteelandt , Jack Bowden , Manoochehr Babanezhad , Els Goetghebeur

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In…

Methodology · Statistics 2025-08-27 Dane Isenberg , Nandita Mitra , Steven C. Marcus , Rinad S. Beidas , Kristin A. Linn

Instrumental variables (IVs) are widely used for estimating causal effects in the presence of unmeasured confounding. Under the standard IV model, however, the average treatment effect (ATE) is only partially identifiable. To address this,…

Methodology · Statistics 2018-01-08 Linbo Wang , Eric Tchetgen Tchetgen

Uncertainty in the estimation of the causal effect in observational studies is often due to unmeasured confounding, i.e., the presence of unobserved covariates linking treatments and outcomes. Instrumental Variables (IV) are commonly used…

Methodology · Statistics 2019-07-30 M. Usaid Awan , Yameng Liu , Marco Morucci , Sudeepa Roy , Cynthia Rudin , Alexander Volfovsky

In causal inference, encouragement designs (EDs) are widely used to analyze causal effects, when randomized controlled trials (RCTs) are impractical or compliance to treatment cannot be perfectly enforced. Unlike RCTs, which directly…

Machine Learning · Computer Science 2024-12-20 Anpeng Wu , Kun Kuang , Ruoxuan Xiong , Xiangwei Chen , Zexu Sun , Fei Wu , Kun Zhang

In semi-logarithmic regressions, treatment coefficients are often interpreted as approximations of the average treatment effect (ATE) in percentage points. This paper highlights the overlooked bias of this approximation under treatment…

Econometrics · Economics 2026-02-04 Ying Zeng

Variable selection for optimal treatment regime in a clinical trial or an observational study is getting more attention. Most existing variable selection techniques focused on selecting variables that are important for prediction, therefore…

Methodology · Statistics 2014-05-22 Ailin Fan , Wenbin Lu , Rui Song

The micro-randomized trial (MRT) is a sequential randomized experimental design to empirically evaluate the effectiveness of mobile health (mHealth) intervention components that may be delivered at hundreds or thousands of decision points.…

Methodology · Statistics 2021-12-14 Jieru Shi , Zhenke Wu , Walter Dempsey

The most widely discussed methods for estimating the Average Causal Effect/Average Treatment Effect are those for intervention in discrete binary variables whose value represents intervention/non-intervention groups. On the other hand,…

Machine Learning · Statistics 2022-03-21 Yoshiaki Kitazawa

Measurement error can often be harmful when estimating causal effects. Two scenarios in which this is the case are in the estimation of (a) the average treatment effect when confounders are measured with error and (b) the natural indirect…

Methodology · Statistics 2024-06-04 Caleb H. Miles , Linda Valeri , Brent Coull

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Estimating the causal effect of a treatment or health policy with observational data can be challenging due to an imbalance of and a lack of overlap between treated and control covariate distributions. In the presence of limited overlap,…

Methodology · Statistics 2025-03-24 Martha Barnard , Jared D. Huling , Julian Wolfson

This article proposes different tests for treatment effect heterogeneity when the outcome of interest, typically a duration variable, may be right-censored. The proposed tests study whether a policy 1) has zero distributional (average)…

Methodology · Statistics 2020-02-19 Pedro H. C. Sant'Anna

It is common in instrumental variable studies for instrument values to be missing, for example when the instrument is a genetic test in Mendelian randomization studies. In this paper we discuss two apparent paradoxes that arise in so-called…

Methodology · Statistics 2018-04-19 Edward H. Kennedy , Dylan S. Small