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In cohort studies, non-random medication use can pose barriers to estimation of the natural history trend in a mean biomarker value (namely, the association between a predictor of interest and a biomarker outcome that would be observed in…

Methodology · Statistics 2021-01-25 Andrew J. Spieker , Joseph A. C. Delaney , Robyn L. McClelland

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

Methodology · Statistics 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

The paper proposes a causal supervised machine learning algorithm to uncover treatment effect heterogeneity in sharp and fuzzy regression discontinuity (RD) designs. We develop a criterion for building an honest ``regression discontinuity…

Econometrics · Economics 2025-09-01 Ágoston Reguly

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

Over the past few years, several approaches utilizing score-based diffusion have been proposed to sample from probability distributions, that is without having access to exact samples and relying solely on evaluations of unnormalized…

Machine Learning · Statistics 2025-04-15 Maxence Noble , Louis Grenioux , Marylou Gabrié , Alain Oliviero Durmus

The Minimum Covariance Determinant (MCD) approach robustly estimates the location and scatter matrix using the subset of given size with lowest sample covariance determinant. Its main drawback is that it cannot be applied when the dimension…

Methodology · Statistics 2021-01-13 Kris Boudt , Peter J. Rousseeuw , Steven Vanduffel , Tim Verdonck

Post-randomization events, also known as intercurrent events, such as treatment noncompliance and censoring due to a terminal event, are common in clinical trials. Principal stratification is a framework for causal inference in the presence…

Methodology · Statistics 2023-01-19 Bo Liu , Lisa Wruck , Fan Li

Variable selection has played a critical role in modern statistical learning and scientific discoveries. Numerous regularization and Bayesian variable selection methods have been developed in the past two decades for variable selection, but…

Methodology · Statistics 2024-03-04 Travis Canida , Hongjie Ke , Shuo Chen , Zhenayo Ye , Tianzhou Ma

This paper presents a novel nonlinear regression model for estimating heterogeneous treatment effects from observational data, geared specifically towards situations with small effect sizes, heterogeneous effects, and strong confounding.…

Methodology · Statistics 2019-11-14 P. Richard Hahn , Jared S. Murray , Carlos Carvalho

Cluster-randomized trials (CRTs) are widely used to evaluate interventions delivered at the clinic, practice, or community level. Although standard analyses typically target average treatment effects, such summaries mask potentially…

Methodology · Statistics 2026-04-16 Changjun Li , Xi Fang , Michael O. Harhay , Andrew B. Forbes , F. Perry Wilson , Guangyu Tong , Fan Li

Instrumental variable methods are widely used in medical and social science research to draw causal conclusions when the treatment and outcome are confounded by unmeasured confounding variables. One important feature of such studies is that…

Methodology · Statistics 2021-05-25 Bo Zhang , Siyu Heng , Emily J. MacKay , Ting Ye

Covariate-adjusted randomization (CAR) can reduce the risk of covariate imbalance and, when accounted for in analysis, increase the power of a trial. Despite CAR advances, stratified randomization remains the most common CAR method. Matched…

Methodology · Statistics 2023-07-13 Jonathan J. Chipman , Lindsay Mayberry , Robert A. Greevy

Causal mediation analysis of observational data is an important tool for investigating the potential causal effects of medications on disease-related risk factors, and on time-to-death (or disease progression) through these risk factors.…

This paper presents a novel approach that leverages domain variability to learn representations that are conditionally invariant to unwanted variability or distractors. Our approach identifies both spurious and invariant latent features…

Machine Learning · Computer Science 2023-07-04 Hananeh Aliee , Ferdinand Kapl , Soroor Hediyeh-Zadeh , Fabian J. Theis

We investigate the finite sample performance of causal machine learning estimators for heterogeneous causal effects at different aggregation levels. We employ an Empirical Monte Carlo Study that relies on arguably realistic data generation…

Econometrics · Economics 2021-10-19 Michael C. Knaus , Michael Lechner , Anthony Strittmatter

Due to the challenge posed by multi-source and heterogeneous data collected from diverse environments, causal relationships among features can exhibit variations influenced by different time spans, regions, or strategies. This diversity…

Machine Learning · Computer Science 2025-02-11 Lu Liu , Yang Tang , Kexuan Zhang , Qiyu Sun

Causal systems often exhibit variations of the underlying causal mechanisms between the variables of the system. Often, these changes are driven by different environments or internal states in which the system operates, and we refer to…

Machine Learning · Computer Science 2025-03-28 Wiebke Günther , Oana-Iuliana Popescu , Martin Rabel , Urmi Ninad , Andreas Gerhardus , Jakob Runge

The Cox regression model and its associated hazard ratio (HR) are frequently used for summarizing the effect of treatments on time to event outcomes. However, the HR's interpretation strongly depends on the assumed underlying survival…

Methodology · Statistics 2021-08-10 Pablo Martinez-Camblor , Todd A. MacKenzie , A. James O'Malley

Multilevel network meta-regression (ML-NMR) enables population-adjusted indirect treatment comparisons by combining individual patient data (IPD) with aggregate data. When individual-level covariates are unavailable, ML-NMR marginalizes…

Methodology · Statistics 2026-03-12 Harlan Campbell , Charles C. Margossian , Jeroen P. Jansen , Paul Gustafson