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Intercurrent events, such as treatment switching, rescue medication, dropout, or truncation by death, frequently complicate intention-to-treat analyses in randomized clinical trials. Existing causal inference frameworks typically target…

Methodology · Statistics 2026-03-12 Georgi Baklicharov , Kelly Van Lancker , Stijn Vansteelandt

For obtaining causal inferences that are objective, and therefore have the best chance of revealing scientific truths, carefully designed and executed randomized experiments are generally considered to be the gold standard. Observational…

Applications · Statistics 2008-11-12 Donald B. Rubin

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying…

Methodology · Statistics 2024-07-18 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

Causal inference in connected populations is non-trivial, because the treatment assignments of units can affect the outcomes of other units via treatment and outcome spillover. Since outcome spillover induces dependence among outcomes,…

Methodology · Statistics 2025-12-25 Subhankar Bhadra , Michael Schweinberger

The primary analysis of randomized screening trials for cancer typically adheres to the intention-to-screen principle, measuring cancer-specific mortality reductions between screening and control arms. These mortality reductions result from…

Methodology · Statistics 2021-07-30 Sudipta Saha , Zhihui Liu , Olli Saarela

Many research questions concern treatment effects on outcomes that can recur several times in the same individual. For example, medical researchers are interested in treatment effects on hospitalizations in heart failure patients and sports…

Methodology · Statistics 2024-12-31 Matias Janvin , Jessica G. Young , Pål C. Ryalen , Mats J. Stensrud

Causal inference from observational data requires assumptions. These assumptions range from measuring confounders to identifying instruments. Traditionally, causal inference assumptions have focused on estimation of effects for a single…

Machine Learning · Statistics 2019-03-04 Rajesh Ranganath , Adler Perotte

In clinical trials, it is often of interest to understand the principal causal effect (PCE), the average treatment effect for a principal stratum (a subset of patients defined by the potential outcomes of one or more post-baseline…

Methodology · Statistics 2022-10-06 Yongming Qu , Ilya Lipkovich , Stephen J. Ruberg

Pathogens usually exist in heterogeneous variants, like subtypes and strains. Quantifying treatment effects on the different variants is important for guiding prevention policies and treatment development. Here we ground analyses of…

Applications · Statistics 2024-08-15 Gellert Perenyi , Mats J. Stensrud

How should researchers conduct causal inference when the outcome of interest is latent and measured imperfectly by multiple indicators? We develop a general nonparametric framework for identifying and estimating average treatment effects on…

Methodology · Statistics 2026-04-22 Jiawei Fu , Donald P. Green

In causal mediation analysis, identification of existing causal direct or indirect effects requires untestable assumptions in which potential outcomes and potential mediators are independent. This paper defines a new causal direct and…

Methodology · Statistics 2020-09-29 Takahiro Hoshino

We study the causal effect with a functional treatment variable, where practical applications often arise in neuroscience, biomedical sciences, etc. Previous research concerning the effect of a functional variable on an outcome is typically…

Methodology · Statistics 2025-05-20 Ruoxu Tan , Wei Huang , Zheng Zhang , Guosheng Yin

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

In the absence of data from a randomized trial, researchers often aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the…

Methodology · Statistics 2021-06-15 Ted Westling , Alex Luedtke , Peter Gilbert , Marco Carone

The sufficient cause framework has been used for decades to improve our understanding of both basic and more complex causal concepts in epidemiology, such as mediation and interaction. Here, we make use of this framework to provide a…

Methodology · Statistics 2026-04-08 Bronner P. Gonçalves , Eiji Yamamoto , Etsuji Suzuki

Causal inference concerns not only the average effect of the treatment on the outcome but also the underlying mechanism through an intermediate variable of interest. Principal stratification characterizes such a mechanism by targeting…

Methodology · Statistics 2022-03-29 Zhichao Jiang , Shu Yang , Peng Ding

Meta-analysis is commonly used to combine results from multiple clinical trials, but traditional meta-analysis methods do not refer explicitly to a population of individuals to whom the results apply and it is not clear how to use their…

Effect modification occurs when a covariate alters the relative effectiveness of treatment compared to control. It is widely understood that, when effect modification is present, treatment recommendations may vary by population and by…

Causal inference on multiple non-independent outcomes raises serious challenges, because multivariate techniques that properly account for the outcome's dependence structure need to be considered. We focus on the case of binary outcomes…

Methodology · Statistics 2018-05-11 Monia Lupparelli , Alessandra Mattei

In this chapter, we review the class of causal effects based on incremental propensity scores interventions proposed by Kennedy [2019]. The aim of incremental propensity score interventions is to estimate the effect of increasing or…

Methodology · Statistics 2021-10-22 Matteo Bonvini , Alec McClean , Zach Branson , Edward H. Kennedy