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Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises…

Applications · Statistics 2022-03-15 Jina Park , Wenjing Hu , Ick Hoon Jin , Hao Liu , Yong Zang

Estimating the ratio of two probability densities from a finite number of observations is a central machine learning problem. A common approach is to construct estimators using binary classifiers that distinguish observations from the two…

Machine Learning · Computer Science 2025-01-28 Werner Zellinger

Objective: To compare different risk-based methods for optimal prediction of treatment effects. Methods: We simulated RCT data using diverse assumptions for the average treatment effect, a baseline prognostic index of risk (PI), the shape…

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

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

In healthcare, there is much interest in estimating policies, or mappings from covariates to treatment decisions. Recently, there is also interest in constraining these estimated policies to the standard of care, which generated the…

Methodology · Statistics 2026-02-17 Samuel J. Weisenthal , Sally W. Thurston , Ashkan Ertefaie

Warfarin, an anticoagulant medication, is formulated to prevent and address conditions associated with abnormal blood clotting, making it one of the most prescribed drugs globally. However, determining the suitable dosage remains…

Machine Learning · Computer Science 2024-02-20 Yong Huang , Charles A. Downs , Amir M. Rahmani

Evaluating the performance of an ongoing policy plays a vital role in many areas such as medicine and economics, to provide crucial instructions on the early-stop of the online experiment and timely feedback from the environment. Policy…

Machine Learning · Statistics 2024-08-05 Ye Shen , Hengrui Cai , Rui Song

Individualizing treatment assignment can improve outcomes for diseases with patient-to-patient variability in comparative treatment effects. When a clinical trial demonstrates that some patients improve on treatment while others do not, it…

Methodology · Statistics 2022-11-02 Nina Galanter , Marco Carone , Ronald C. Kessler , Alex Luedtke

In this work, we consider causal inference in various high-dimensional treatment settings, including for single multi-valued treatments and vector treatments with binary or continuous components, when the number of treatments can be…

Statistics Theory · Mathematics 2026-02-26 Patrick Kramer , Edward H. Kennedy , Isaac M. Opper

We suggest double/debiased machine learning estimators of direct and indirect quantile treatment effects under a selection-on-observables assumption. This permits disentangling the causal effect of a binary treatment at a specific outcome…

Econometrics · Economics 2023-07-04 Yu-Chin Hsu , Martin Huber , Yu-Min Yen

Personalized medicine seeks to identify the causal effect of treatment for a particular patient as opposed to a clinical population at large. Most investigators estimate such personalized treatment effects by regressing the outcome of a…

Machine Learning · Statistics 2021-09-02 Eric V. Strobl , Shyam Visweswaran

Interval designs are a class of phase I trial designs for which the decision of dose assignment is determined by comparing the observed toxicity rate at the current dose with a prespecified (toxicity tolerance) interval. If the observed…

Methodology · Statistics 2013-09-20 Suyu Liu , Ying Yuan

With the increasing adoption of electronic health records, there is an increasing interest in developing individualized treatment rules, which recommend treatments according to patients' characteristics, from large observational data.…

Methodology · Statistics 2021-05-05 Muxuan Liang , Young-Geun Choi , Yang Ning , Maureen A Smith , Ying-Qi Zhao

Recent years have experienced increasing utilization of complex machine learning models across multiple sources of data to inform more generalizable decision-making. However, distribution shifts across data sources and privacy concerns…

Methodology · Statistics 2024-05-16 Yi Liu , Alexander W. Levis , Sharon-Lise Normand , Larry Han

In many mobile health interventions, treatments should only be delivered in a particular context, for example when a user is currently stressed, walking or sedentary. Even in an optimal context, concerns about user burden can restrict which…

Machine Learning · Computer Science 2018-12-04 Sabina Tomkins , Predrag Klasnja , Susan Murphy

We propose a novel regression adjustment method designed for estimating distributional treatment effect parameters in randomized experiments. Randomized experiments have been extensively used to estimate treatment effects in various…

Econometrics · Economics 2024-07-24 Undral Byambadalai , Tatsushi Oka , Shota Yasui

Predicated on the increasing abundance of electronic health records, we investi- gate the problem of inferring individualized treatment effects using observational data. Stemming from the potential outcomes model, we propose a novel multi-…

Machine Learning · Computer Science 2017-05-30 Ahmed M. Alaa , Mihaela van der Schaar

We investigate the task of estimating the conditional average causal effect of treatment-dosage pairs from a combination of observational data and assumptions on the causal relationships in the underlying system. This has been a…

Machine Learning · Computer Science 2022-05-31 Alexis Bellot , Anish Dhir , Giulia Prando

In machine learning applications, predictive models are trained to serve future queries across the entire data distribution. Real-world data often demands excessively complex models to achieve competitive performance, however, sacrificing…

Machine Learning · Computer Science 2025-09-22 Jizhou Huang , Brendan Juba