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In the absence of randomized controlled and natural experiments, it is necessary to balance the distributions of (observable) covariates of the treated and control groups in order to obtain an unbiased estimate of a causal effect of…

Methodology · Statistics 2022-03-02 Martin Cousineau , Vedat Verter , Susan A. Murphy , Joelle Pineau

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

Methodology · Statistics 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

With an increasing focus on precision medicine in medical research, numerous studies have been conducted in recent years to clarify the relationship between treatment effects and patient characteristics. The treatment effects for patients…

Methodology · Statistics 2023-09-22 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

Regression discontinuity designs are widely used when treatment assignment is determined by whether a running variable exceeds a predefined threshold. However, most research focuses on estimating local causal effects at the threshold,…

Methodology · Statistics 2025-01-06 Xinqin Feng , Wenjie Hu , Pu Yang , Tingyu Li , Xiao-Hua Zhou

Traditional causal inference approaches leverage observational study data to estimate the difference in observed and unobserved outcomes for a potential treatment, known as the Conditional Average Treatment Effect (CATE). However, CATE…

Machine Learning · Statistics 2022-09-22 Tianhui Zhou , William E Carson , David Carlson

Causal decomposition analysis provides a way to identify mediators that contribute to health disparities between marginalized and non-marginalized groups. In particular, the degree to which a disparity would be reduced or remain after…

Methodology · Statistics 2021-09-16 Soojin Park , Suyeon Kang , Chioun Lee

Multiple and combined endpoints involving also non-normal outcomes appear in many clinical trials in various areas in medicine. In some cases, the outcome can be observed only on an ordinal or dichotomous scale. Then the success of two…

Methodology · Statistics 2020-02-24 Edgar Brunner

Estimating causal effects for survival outcomes in the high-dimensional setting is an extremely important topic for many biomedical applications as well as areas of social sciences. We propose a new orthogonal score method for treatment…

Methodology · Statistics 2024-12-04 Jue Hou , Jelena Bradic , Ronghui Xu

We propose a novel method for estimating heterogeneous treatment effects based on the fused lasso. By first ordering samples based on the propensity or prognostic score, we match units from the treatment and control groups. We then run the…

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

Methodology · Statistics 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

In settings where units' outcomes are affected by others' treatments, there has been a proliferation of ways to quantify effects of treatments on outcomes, including via indirect exposure to other units' treatments. Here we consider two…

Methodology · Statistics 2026-03-10 Sahil Loomba , Dean Eckles

Causal inference analysis is the estimation of the effects of actions on outcomes. In the context of healthcare data this means estimating the outcome of counter-factual treatments (i.e. including treatments that were not observed) on a…

Methodology · Statistics 2018-03-21 Yishai Shimoni , Chen Yanover , Ehud Karavani , Yaara Goldschmnidt

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

Heterogeneous treatment effect (HTE) estimation is critical in medical research. It provides insights into how treatment effects vary among individuals, which can provide statistical evidence for precision medicine. While most existing…

Machine Learning · Statistics 2025-04-25 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

Randomized Controlled Trials are one of the pillars of science; nevertheless, they rely on hand-crafted hypotheses and expensive analysis. Such constraints prevent causal effect estimation at scale, potentially anchoring on popular yet…

Machine Learning · Computer Science 2026-01-07 Tommaso Mencattini , Riccardo Cadei , Francesco Locatello

The aim of clinical effectiveness research using repositories of electronic health records is to identify what health interventions 'work best' in real-world settings. Since there are several reasons why the net benefit of intervention may…

Methodology · Statistics 2020-06-19 Jie Zhu , Blanca Gallego

In most nonrandomized observational studies, differences between treatment groups may arise not only due to the treatment but also because of the effect of confounders. Therefore, causal inference regarding the treatment effect is not as…

Methodology · Statistics 2018-07-04 Debashis Ghosh

Investigators often use multi-source data (e.g., multi-center trials, meta-analyses of randomized trials, pooled analyses of observational cohorts) to learn about the effects of interventions in subgroups of some well-defined target…

Methodology · Statistics 2024-02-06 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

Composite endpoints are widely used in cardiovascular clinical trials to improve statistical efficiency while preserving clinical relevance. The Win Ratio (WR) measure and more general frameworks of Win Statistics have emerged as…

Methodology · Statistics 2025-11-24 Yunhan Mou , Fan Li , Denise Esserman , Yuan Huang

A central goal of modern causal inference is estimating heterogeneous treatment effects to answer questions like "how does an intervention affect each unit," rather than only on average. We study this problem with panel-data where we…

Machine Learning · Statistics 2026-05-29 Anay Mehrotra , Phuc Tran , Van H. Vu , Manolis Zampetakis