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In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

Predicting time-to-event outcomes in large databases can be a challenging but important task. One example of this is in predicting the time to a clinical outcome for patients in intensive care units (ICUs), which helps to support critical…

Computation · Statistics 2019-08-06 Yingying Xu , Joon Lee , Joel A. Dubin

Individualized treatment rules aim to identify if, when, which, and to whom treatment should be applied. A globally aging population, rising healthcare costs, and increased access to patient-level data have created an urgent need for…

Methodology · Statistics 2019-01-04 Ying-Qi Zhao , Eric B. Laber , Yang Ning , Sumona Saha , Bruce Sands

Tree ensemble models such as random forests and boosted trees are among the most widely used and practically successful predictive models in applied machine learning and business analytics. Although such models have been used to make…

Optimization and Control · Mathematics 2019-10-11 Velibor V. Mišić

The increasing availability of individual-level data has led to numerous applications of individualized (or personalized) treatment rules (ITRs). Policy makers often wish to empirically evaluate ITRs and compare their relative performance…

Applications · Statistics 2021-05-06 Kosuke Imai , Michael Lingzhi Li

Dynamic treatment regimes (DTRs) are used in medicine to tailor sequential treatment decisions to patients by considering patient heterogeneity. Common methods for learning optimal DTRs, however, have shortcomings: they are typically based…

Machine Learning · Statistics 2023-06-21 Theresa Blümlein , Joel Persson , Stefan Feuerriegel

The randomized controlled trial (RCT) is the gold standard for estimating the average treatment effect (ATE) of a medical intervention but requires 100s-1000s of subjects, making it expensive and difficult to implement. While a cross-over…

Signal Processing · Electrical Eng. & Systems 2023-05-10 Sayeri Lala , Niraj K. Jha

Individualized treatment rules (ITRs) are deterministic decision rules that recommend treatments to individuals based on their characteristics. Though ubiquitous in medicine, ITRs are hardly ever evaluated in randomized controlled trials.…

Methodology · Statistics 2023-08-22 François Grolleau , Francois Petit , Raphaël Porcher

Precision medicine is an emerging scientific topic for disease treatment and prevention that takes into account individual patient characteristics. It is an important direction for clinical research, and many statistical methods have been…

Methodology · Statistics 2017-02-17 Jingxiang Chen , Haoda Fu , Xuanyao He , Michael R. Kosorok , Yufeng Liu

Recurrent events, characterized by the repeated occurrence of the same event in an individual, are a common type of data in medical research. Motivated by cancer recurrences, we aim to estimate the optimal individualized treatment regime…

Methodology · Statistics 2025-02-18 Zi-Shu Zhan , Jin-Lun Zhang , Chen Shi , Xiao-Han Xu , Chun-Quan Ou

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

Methodology · Statistics 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

Medical treatments tailored to a patient's baseline characteristics hold the potential of improving patient outcomes while reducing negative side effects. Learning individualized treatment rules (ITRs) often requires aggregation of multiple…

Machine Learning · Statistics 2022-12-15 Jay Jojo Cheng , Jared D. Huling , Guanhua Chen

In recent years, there has been growing interest in causal machine learning estimators for quantifying subject-specific effects of a binary treatment on time-to-event outcomes. Estimation approaches have been proposed which attenuate the…

Methodology · Statistics 2026-03-30 Matthew Pryce , Karla Diaz-Ordaz , Ruth H. Keogh , Stijn Vansteelandt

We illustrate use of nonparametric statistical methods to compare alternative treatments for a particular disease or condition on both their relative effectiveness and their relative cost. These Incremental Cost Effectiveness (ICE) methods…

Methodology · Statistics 2023-12-04 Robert L. Obenchain

Estimating the Individual Treatment Effect from observational data, defined as the difference between outcomes with and without treatment or intervention, while observing just one of both, is a challenging problems in causal learning. In…

Machine Learning · Computer Science 2020-05-07 Céline Beji , Michaël Bon , Florian Yger , Jamal Atif

The ability to predict individualized treatment effects (ITEs) based on a given patient's profile is essential for personalized medicine. We propose a hypothesis testing approach to choosing between two potential treatments for a given…

Methodology · Statistics 2020-08-11 Tianxi Cai , Tony Cai , Zijian Guo

Optimal treatment regimes are personalized policies for making a treatment decision based on subject characteristics, with the policy chosen to maximize some value. It is common to aim to maximize the mean outcome in the population, via a…

Methodology · Statistics 2022-02-28 Liu Leqi , Edward H. Kennedy

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

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

Estimation of heterogeneous treatment effects (HTE) is of prime importance in many disciplines, ranging from personalized medicine to economics among many others. Random forests have been shown to be a flexible and powerful approach to HTE…

Methodology · Statistics 2025-10-07 Susanne Dandl , Torsten Hothorn , Heidi Seibold , Erik Sverdrup , Stefan Wager , Achim Zeileis