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Precision medicine for chronic diseases such as multiple sclerosis (MS) involves choosing a treatment which best balances efficacy and side effects/preferences for individual patients. Making this choice as early as possible is important,…

Image and Video Processing · Electrical Eng. & Systems 2022-09-23 Joshua Durso-Finley , Jean-Pierre R. Falet , Brennan Nichyporuk , Douglas L. Arnold , Tal Arbel

A dynamic treatment regime is a sequence of decision rules in which each decision rule recommends treatment based on features of patient medical history such as past treatments and outcomes. Existing methods for estimating optimal dynamic…

Methodology · Statistics 2015-05-22 Kristin A. Linn , Eric B. Laber , Leonard A. Stefanski

In clinical trials with recurrent events, such as repeated hospitalizations terminating with death, it is important to consider the patient events overall history for a thorough assessment of treatment effects. The occurrence of fewer…

Methodology · Statistics 2025-09-08 Alessandra Ragni , Torben Martinussen , Thomas Scheike

Provider profiling has the goal of identifying healthcare providers with exceptional patient outcomes. When evaluating providers, adjustment is necessary to control for differences in case-mix between different providers. Direct and…

Methodology · Statistics 2024-10-28 Herbert Susmann , Yiting Li , Mara A. McAdams-DeMarco , Iván Díaz , Wenbo Wu

Targeted maximum likelihood estimation is a general methodology combining flexible ensemble learning and semiparametric efficiency theory in a two-step procedure for estimation of causal parameters. Proposed targeted maximum likelihood…

Methodology · Statistics 2021-06-22 Helene Charlotte Wiese Rytgaard , Frank Eriksson , Mark van der Laan

Large observational data are increasingly available in disciplines such as health, economic and social sciences, where researchers are interested in causal questions rather than prediction. In this paper, we examine the problem of…

Methodology · Statistics 2021-11-24 Alberto Caron , Gianluca Baio , Ioanna Manolopoulou

We address causal estimation in semi-competing risks settings, where a non-terminal event may be precluded by one or more terminal events. We define a principal-stratification causal estimand for treatment effects on the non-terminal event,…

Methodology · Statistics 2025-06-27 Karina Gelis-Cadena , Michael Daniels , Juned Siddique

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

An accurate model of a patient's individual survival distribution can help determine the appropriate treatment for terminal patients. Unfortunately, risk scores (e.g., from Cox Proportional Hazard models) do not provide survival…

Machine Learning · Computer Science 2020-07-08 Humza Haider , Bret Hoehn , Sarah Davis , Russell Greiner

Patient care may be improved by recommending treatments based on patient characteristics when there is treatment effect heterogeneity. Recently, there has been a great deal of attention focused on the estimation of optimal treatment rules…

Methodology · Statistics 2024-01-29 Michael Jetsupphasuk , Michael G. Hudgens , Jessie K. Edwards , Stephen R. Cole

Individualized treatment recommendation (ITR) is an important analytic framework for precision medicine. The goal is to assign proper treatments to patients based on their individual characteristics. From the machine learning perspective,…

Machine Learning · Statistics 2020-04-07 Haomiao Meng , Ying-Qi Zhao , Haoda Fu , Xingye Qiao

Many scientific and engineering challenges -- ranging from personalized medicine to customized marketing recommendations -- require an understanding of treatment effect heterogeneity. In this paper, we develop a non-parametric causal forest…

Methodology · Statistics 2017-07-11 Stefan Wager , Susan Athey

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

Sequential multiple assignment randomized trials (SMARTs) provide a systematic framework for constructing and evaluating dynamic treatment regimens (DTRs). In clinical studies, longitudinal biomarkers are routinely collected to monitor…

Methodology · Statistics 2026-05-06 Zhengxi Chen , Holly Hartman

There is a growing literature on finding so-called optimal treatment rules, which are rules by which to assign treatment to individuals based on an individual's characteristics, such that a desired outcome is maximized. A related goal…

Applications · Statistics 2021-01-22 Kara E. Rudolph , Ivan Diaz

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

Methodology · Statistics 2026-03-31 Zhe Chen , Xinran Li

As cancer patient survival improves, late effects from treatment are becoming the next clinical challenge. Chemotherapy and radiotherapy, for example, potentially increase the risk of both morbidity and mortality from second malignancies…

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Competing risk analysis considers event times due to multiple causes, or of more than one event types. Commonly used regression models for such data include 1) cause-specific hazards model, which focuses on modeling one type of event while…

Applications · Statistics 2017-04-27 Jiayi Hou , Anthony Paravati , Ronghui Xu , James Murphy

Clinical trials involving novel immuno-oncology (IO) therapies frequently exhibit survival profiles which violate the proportional hazards assumption due to a delay in treatment effect, and in such settings, the survival curves in the two…

Methodology · Statistics 2021-02-02 Nicholas C. Henderson , Kijoeng Nam , Dai Feng