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When estimating heterogeneous treatment effects, missing outcome data can complicate treatment effect estimation, causing certain subgroups of the population to be poorly represented. In this work, we discuss this commonly overlooked…

Machine Learning · Statistics 2025-04-15 Matthew Pryce , Karla Diaz-Ordaz , Ruth H. Keogh , Stijn Vansteelandt

Estimating treatment effects from observational data is challenging due to two main reasons: (a) hidden confounding, and (b) covariate mismatch (control and treatment groups not having identical distributions). Long lines of works exist…

Machine Learning · Computer Science 2025-04-30 Praharsh Nanavati , Ranjitha Prasad , Karthikeyan Shanmugam

Unmeasured confounding is a threat to causal inference in observational studies. In recent years, use of negative controls to mitigate unmeasured confounding has gained increasing recognition and popularity. Negative controls have a…

Methodology · Statistics 2019-09-05 Xu Shi , Wang Miao , Jennifer C. Nelson , Eric J. Tchetgen Tchetgen

We propose Causal Interaction Trees for identifying subgroups of participants that have enhanced treatment effects using observational data. We extend the Classification and Regression Tree algorithm by using splitting criteria that focus…

Methodology · Statistics 2021-12-08 Jiabei Yang , Issa J. Dahabreh , Jon A. Steingrimsson

Efficiently and flexibly estimating treatment effect heterogeneity is an important task in a wide variety of settings ranging from medicine to marketing, and there are a considerable number of promising conditional average treatment effect…

Machine Learning · Statistics 2023-05-10 Erik Sverdrup , Yifan Cui

Causal inference for observational longitudinal studies often requires the accurate estimation of treatment effects on time-to-event outcomes in the presence of time-dependent patient history and time-dependent covariates. To tackle this…

Machine Learning · Statistics 2022-06-17 Jie Zhu , Blanca Gallego

Learning the response of single-cells to various treatments offers great potential to enable targeted therapies. In this context, neural optimal transport (OT) has emerged as a principled methodological framework because it inherently…

Machine Learning · Computer Science 2025-04-14 Alice Driessen , Benedek Harsanyi , Marianna Rapsomaniki , Jannis Born

Disaggregated evaluation across subgroups is critical for assessing the fairness of machine learning models, but its uncritical use can mislead practitioners. We show that equal performance across subgroups is an unreliable measure of…

We study hybrid control trials (HCTs), in which a randomized controlled trial (RCT) is augmented with external control patients. Existing approaches for HCTs typically assume conditional exchangeability of the concurrent and external…

Methodology · Statistics 2025-11-21 Alissa Gordon , Emilie Højbjerre-Frandsen , Alejandro Schuler

Several problems in statistics involve the combination of high-variance unbiased estimators with low-variance estimators that are only unbiased under strong assumptions. A notable example is the estimation of causal effects while combining…

Methodology · Statistics 2023-05-25 Michael Oberst , Alexander D'Amour , Minmin Chen , Yuyan Wang , David Sontag , Steve Yadlowsky

Image-based precision medicine aims to personalize treatment decisions based on an individual's unique imaging features so as to improve their clinical outcome. Machine learning frameworks that integrate uncertainty estimation as part of…

Machine Learning · Computer Science 2023-08-11 Joshua Durso-Finley , Jean-Pierre Falet , Raghav Mehta , Douglas L. Arnold , Nick Pawlowski , Tal Arbel

The estimation of Conditional Average Treatment Effects (CATE) is crucial for understanding the heterogeneity of treatment effects in clinical trials. We evaluate the performance of common methods, including causal forests and various…

Methodology · Statistics 2024-07-12 Oshri Machluf , Tzviel Frostig , Gal Shoham , Tomer Milo , Elad Berkman , Raviv Pryluk

The bulk of causal inference studies rule out the presence of interference between units. However, in many real-world scenarios, units are interconnected by social, physical, or virtual ties, and the effect of the treatment can spill from…

Methodology · Statistics 2023-11-03 Falco J. Bargagli-Stoffi , Costanza Tortù , Laura Forastiere

Recursive decision trees are widely used to estimate heterogeneous causal treatment effects in experimental and observational studies. These methods are typically implemented using CART-type recursive partitioning and are often viewed as…

Statistics Theory · Mathematics 2026-03-19 Matias D. Cattaneo , Jason M. Klusowski , Ruiqi Rae Yu

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

Causal inference in observational studies can be challenging when confounders are subject to missingness. Generally, the identification of causal effects is not guaranteed even under restrictive parametric model assumptions when confounders…

Methodology · Statistics 2023-03-23 Jian Sun , Bo Fu

In causal mediation studies that decompose an average treatment effect into a natural indirect effect (NIE) and a natural direct effect (NDE), examples of post-treatment confounding are abundant. Past research has generally considered it…

Methodology · Statistics 2021-07-26 Guanglei Hong , Fan Yang , Xu Qin

Interpretability research now offers a variety of techniques for identifying abstract internal mechanisms in neural networks. Can such techniques be used to predict how models will behave on out-of-distribution examples? In this work, we…

Machine Learning · Computer Science 2025-11-12 Jing Huang , Junyi Tao , Thomas Icard , Diyi Yang , Christopher Potts

In observational research treatment effects, the average treatment effect (ATE) estimator may be biased if a confounding variable is misclassified. We discuss the impact of classification error in a dichotomous confounding variable in…

Methodology · Statistics 2019-12-13 Linda Nab , Rolf H. H. Groenwold , Maarten van Smeden , Ruth H. Keogh

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li
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