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The dearth of prescribing guidelines for physicians is one key driver of the current opioid epidemic in the United States. In this work, we analyze medical and pharmaceutical claims data to draw insights on characteristics of patients who…

Machine Learning · Computer Science 2020-05-01 Chirag Nagpal , Dennis Wei , Bhanukiran Vinzamuri , Monica Shekhar , Sara E. Berger , Subhro Das , Kush R. Varshney

It has recently become popular to define treatment effects for subsets of the target population characterized by variables not observable at the time a treatment decision is made. Characterizing and estimating such treatment effects is…

Statistics Theory · Mathematics 2007-08-30 Marshall M. Joffe , Dylan Small , Chi-Yuan Hsu

In many applied fields, researchers are often interested in tailoring treatments to unit-level characteristics in order to optimize an outcome of interest. Methods for identifying and estimating treatment policies are the subject of the…

Methodology · Statistics 2020-04-06 Eli Sherman , David Arbour , Ilya Shpitser

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

In personalised decision making, evidence is required to determine whether an action (treatment) is suitable for an individual. Such evidence can be obtained by modelling treatment effect heterogeneity in subgroups. The existing…

Methodology · Statistics 2022-06-24 Jiuyong Li , Lin Liu , Shisheng Zhang , Saisai Ma , Thuc Duy Le , Jixue Liu

We propose a new modeling and estimation approach to select the optimal treatment regime from different options through constructing a robust estimating equation. The method is protected against misspecification of the propensity score…

Methodology · Statistics 2022-11-15 Trinetri Ghosh , Yanyuan Ma , Wensheng Zhu , Yuanjia Wang

Appropriate treatment regimens play a vital role in improving patient health status. Although some achievements have been made, few of the recent studies of learning treatment regimens have exploited different kinds of patient information…

Computers and Society · Computer Science 2018-06-21 Khanh-Hung Hoang , Tu-Bao Ho

Pragmatic trials increasingly define outcomes using real-world data such as electronic health records, where assessments are collected during routine care rather than at fixed timepoints. Consequently, these uncontrolled assessments may be…

Big data and business analytics are critical drivers of business and societal transformations. Uplift models support a firm's decision-making by predicting the change of a customer's behavior due to a treatment. Prior work examines models…

Machine Learning · Computer Science 2021-01-12 Robin M. Gubela , Stefan Lessmann

An individualized treatment rule (ITR) is a decision rule that aims to improve individual patients health outcomes by recommending optimal treatments according to patients specific information. In observational studies, collected data may…

Methodology · Statistics 2023-10-03 Zeyu Bian , Erica EM Moodie , Susan M Shortreed , Sylvie D Lambert , Sahir Bhatnagar

Precision medicine involves developing individualized treatment regimes (ITRs) which allow for treatment decisions to be tailored to patient characteristics. Naturally, the identification of the optimal regime, that is, the rule which…

Methodology · Statistics 2025-09-30 Misha Dolmatov , Erica E. M. Moodie , David A. Stephens , Dipankar Bandyopadhyay

Learning interpretable models has become a major focus of machine learning research, given the increasing prominence of machine learning in socially important decision-making. Among interpretable models, rule lists are among the best-known…

Machine Learning · Computer Science 2024-06-19 Leonardo Pellegrina , Fabio Vandin

Randomized controlled trials typically analyze the effectiveness of treatments with the goal of making treatment recommendations for patient subgroups. With the advance of electronic health records, a great variety of data has been…

Machine Learning · Computer Science 2021-03-31 Zhiliang Wu , Yinchong Yang , Yunpu Ma , Yushan Liu , Rui Zhao , Michael Moor , Volker Tresp

Multistate process data are common in studies of chronic diseases such as cancer. These data are ideal for precision medicine purposes as they can be leveraged to improve more refined health outcomes, compared to standard survival outcomes,…

Methodology · Statistics 2022-11-28 Giorgos Bakoyannis

A fundamental principle of clinical medicine is that a treatment should only be administered to those patients who would benefit from it. Treatment strategies that assign treatment to patients as a function of their individual…

Applications · Statistics 2025-06-13 Nicholas Williams , Kara Rudolph , Iván Díaz

The current work is motivated by the need for robust statistical methods for precision medicine; as such, we address the need for statistical methods that provide actionable inference for a single unit at any point in time. We aim to learn…

Statistics Theory · Mathematics 2021-07-02 Ivana Malenica , Aurelien Bibaut , Mark J. van der Laan

One primary goal of precision medicine is to estimate the individualized treatment rules (ITRs) that optimize patients' health outcomes based on individual characteristics. Health studies with multiple treatments are commonly seen in…

Methodology · Statistics 2025-05-07 Xuqiao Li , Qiuyan Zhou , Ying Wu , Ying Yan

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

The concept of personalised medicine in cancer therapy is becoming increasingly important. There already exist drugs administered specifically for patients with tumours presenting well-defined mutations. However, the field is still in its…

Biomolecules · Quantitative Biology 2024-08-26 Abbi Abdel-Rehim , Oghenejokpeme Orhobor , Gareth Griffiths , Larisa Soldatova , Ross D. King

This paper presents the foundations of a computer oriented approach for preparing a list of random treatment assignments to be adopted in randomised controlled trials. Software is presented which can be applied in the earliest stage of…

Applications · Statistics 2015-02-12 N. S. Santos-Magalhaes , H. M. de Oliveira , A. J. Alves
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