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We consider estimation of an optimal individualized treatment rule from observational and randomized studies when a high-dimensional vector of baseline variables is available. Our optimality criterion is with respect to delaying expected…

Methodology · Statistics 2017-11-09 Iván Díaz , Oleksandr Savenkov , Karla Ballman

Background: Phase I dose-finding trials increasingly encounter delayed-onset toxicities, especially with immunotherapies and targeted agents. The time-to-event continual reassessment method (TITE-CRM) handles incomplete follow-up using…

Methodology · Statistics 2026-02-24 Robert Amevor , Emmanuel Kubuafor , Dennis Baidoo

Targeted maximum likelihood estimation (TMLE) is a general method for estimating parameters in semiparametric and nonparametric models. Each iteration of TMLE involves fitting a parametric submodel that targets the parameter of interest. We…

Methodology · Statistics 2014-06-03 Iván Díaz , Michael Rosenblum

Treatment effect estimation is essential for informed decision-making in many fields such as healthcare, economics, and public policy. While flexible machine learning models have been widely applied for estimating heterogeneous treatment…

Machine Learning · Computer Science 2025-09-29 Pascal Memmesheimer , Vincent Heuveline , Jürgen Hesser

Estimating the effect of a treatment on a given outcome, conditioned on a vector of covariates, is central in many applications. However, learning the impact of a treatment on a continuous temporal response, when the covariates suffer…

Machine Learning · Computer Science 2019-06-11 Guangyi Zhang , Reza Ashrafi , Anne Juuti , Kirsi Pietiläinen , Pekka Marttinen

This thesis develops methods for causal inference and causal representation learning (CRL) in high-dimensional, time-varying data. The first contribution introduces the Causal Dynamic Variational Autoencoder (CDVAE), a model for estimating…

Machine Learning · Statistics 2025-12-05 Mouad EL Bouchattaoui

We address the practical construction of asymptotic confidence intervals for smooth (i.e., path-wise differentiable), real-valued statistical parameters by targeted learning from independent and identically distributed data in contexts…

Statistics Theory · Mathematics 2016-07-01 Patrice Bertail , Antoine Chambaz , Emilien Joly

Covariate-specific treatment effects (CSTEs) represent heterogeneous treatment effects across subpopulations defined by certain selected covariates. In this article, we consider marginal structural models where CSTEs are linearly…

Methodology · Statistics 2021-05-25 Peng Wu , Zhiqiang Tan , Wenjie Hu , Xiao-Hua Zhou

Identifying a biomarker or treatment-dose threshold that marks a specified level of risk is an important problem, especially in clinical trials. This risk, viewed as a function of thresholds and possibly adjusted for covariates, we call the…

Methodology · Statistics 2023-03-22 Lars van der Laan , Wenbo Zhang , Peter B. Gilbert

The Average Treatment Effect (ATE) is a global measure of the effectiveness of an experimental treatment intervention. Classical methods of its estimation either ignore relevant covariates or do not fully exploit them. Moreover, past work…

Methodology · Statistics 2013-11-05 Emil Pitkin , Richard Berk , Lawrence Brown , Andreas Buja , Ed George , Kai Zhang , Linda Zhao

The Targeted Maximum Likelihood Estimation (TMLE) statistical data analysis framework integrates machine learning, statistical theory, and statistical inference to provide a least biased, efficient and robust strategy for estimation and…

Objective: In randomized clinical trials, prediction models can be used to explore the relationships between patients' variables (e.g., clinical, pathological, or lifestyle variables, and also biomarker or genomic data) and treatment effect…

Quantitative Methods · Quantitative Biology 2026-02-03 Elvire Roblin , Paul-Henry Cournède , Stefan Michiels

Across research disciplines, cluster randomized trials (CRTs) are commonly implemented to evaluate interventions delivered to groups of participants, such as communities and clinics. Despite advances in the design and analysis of CRTs,…

Covariate adjustment is a ubiquitous method used to estimate the average treatment effect (ATE) from observational data. Assuming a known graphical structure of the data generating model, recent results give graphical criteria for optimal…

Statistics Theory · Mathematics 2025-12-08 Alexander Mangulad Christgau , Anton Rask Lundborg , Niels Richard Hansen

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

Machine learning (ML) holds great potential for accurately forecasting treatment outcomes over time, which could ultimately enable the adoption of more individualized treatment strategies in many practical applications. However, a…

Machine Learning · Statistics 2023-06-08 Toon Vanderschueren , Alicia Curth , Wouter Verbeke , Mihaela van der Schaar

Estimation of the Average Treatment Effect (ATE) is a core problem in causal inference with strong connections to Off-Policy Evaluation in Reinforcement Learning. This paper considers the problem of adaptively selecting the treatment…

Machine Learning · Statistics 2024-11-22 Ojash Neopane , Aaditya Ramdas , Aarti Singh

We study how to efficiently estimate average treatment effects (ATEs) using adaptive experiments. In adaptive experiments, experimenters sequentially assign treatments to experimental units while updating treatment assignment probabilities…

Machine Learning · Statistics 2025-02-21 Masahiro Kato , Takuya Ishihara , Junya Honda , Yusuke Narita

Many practical decision-making problems in economics and healthcare seek to estimate the average treatment effect (ATE) from observational data. The Double/Debiased Machine Learning (DML) is one of the prevalent methods to estimate ATE in…

Econometrics · Economics 2022-12-07 Yiyan Huang , Cheuk Hang Leung , Xing Yan , Qi Wu , Shumin Ma , Zhiri Yuan , Dongdong Wang , Zhixiang Huang

Recently, many researchers have advanced data-driven methods for modeling heterogeneous treatment effects (HTEs). Even still, estimation of HTEs is a difficult task -- these methods frequently over- or under-estimate the treatment effects,…

Methodology · Statistics 2022-03-28 Yizhe Xu , Steve Yadlowsky
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