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Decision makers, such as doctors and judges, make crucial decisions such as recommending treatments to patients, and granting bails to defendants on a daily basis. Such decisions typically involve weighting the potential benefits of taking…

Machine Learning · Statistics 2016-11-24 Himabindu Lakkaraju , Cynthia Rudin

Data-driven individualized decision making has recently received increasing research interests. Most existing methods rely on the assumption of no unmeasured confounding, which unfortunately cannot be ensured in practice especially in…

Methodology · Statistics 2022-12-26 Zhengling Qi , Rui Miao , Xiaoke Zhang

The identification of patient subgroups with differential treatment effects is the first step towards individualised treatments. A current draft guideline by the EMA discusses potentials and problems in subgroup analyses and formulated…

Methodology · Statistics 2020-01-22 Heidi Seibold , Achim Zeileis , Torsten Hothorn

Approving and assessing new drugs is complex because multiple criteria must be considered simultaneously. A common approach is benefit-risk analysis, often conducted within a Bayesian framework to account for uncertainty and combine data…

Network Meta-Analysis (NMA) plays a pivotal role in synthesizing evidence from various sources and comparing multiple interventions. At its core, NMA relies on integrating both direct evidence from head-to-head comparisons and indirect…

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Quantifying causal effects in the presence of complex and multivariate outcomes remains a key challenge in treatment evaluation. For hierarchical multivariate outcomes, the FDA recommends the Win Ratio and Generalized Pairwise Comparisons…

Methodology · Statistics 2026-03-24 Mathieu Even , Julie Josse

The appearance of a new dangerous and contagious disease requires the development of a drug therapy faster than what is foreseen by usual mechanisms. Many drug therapy developments consist in investigating through different clinical trials…

Quantitative Methods · Quantitative Biology 2020-03-31 Ezequiel Alvarez , Federico Lamagna , Manuel Szewc

While many novel therapies have been approved in recent years for treating patients with multiple myeloma, there is still no established curative regimen, especially for patients with high risk disease. In this work, we use a mathematical…

Optimization and Control · Mathematics 2022-11-18 Mahya Aghaee , Urszula Ledzewicz , Michael Robbins , Natalie Bezman , Hearn Jay Cho , Helen Moore

In order to identify important variables that are involved in making optimal treatment decision, Lu et al. (2013) proposed a penalized least squared regression framework for a fixed number of predictors, which is robust against the…

Machine Learning · Statistics 2015-10-16 Chengchun Shi , Rui Song , Wenbin Lu

In pharmaceutical R&D, predicting the efficacy of a pharmaceutical in treating a particular disease prior to clinical testing or any real-world use has been challenging. In this paper, we propose a flexible and modular machine…

Machine Learning · Computer Science 2025-09-10 Adrien Couetoux , Thomas Devenyns , Lise Diagne , David Champagne , Pierre-Yves Mousset , Chris Anagnostopoulos

Variable selection for optimal treatment regime in a clinical trial or an observational study is getting more attention. Most existing variable selection techniques focused on selecting variables that are important for prediction, therefore…

Methodology · Statistics 2014-05-22 Ailin Fan , Wenbin Lu , Rui Song

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…

In medicine, treatments often influence multiple, interdependent outcomes, such as primary endpoints, complications, adverse events, or other secondary endpoints. Hence, to make optimal treatment decisions, clinicians are interested in…

Machine Learning · Computer Science 2025-06-03 Yuchen Ma , Jonas Schweisthal , Hengrui Zhang , Stefan Feuerriegel

The integration of artificial intelligence [AI] into clinical trials has revolutionized the process of drug development and personalized medicine. Among these advancements, deep learning and predictive modelling have emerged as…

Machine Learning · Computer Science 2024-12-11 Sydney Anuyah , Mallika K Singh , Hope Nyavor

A treatment regime is a function that maps individual patient information to a recommended treatment, hence explicitly incorporating the heterogeneity in need for treatment across individuals. Patient responses are dichotomous and can be…

Machine Learning · Statistics 2016-07-07 Yingfei Wang , Warren Powell

The move towards personalized treatment and digital twins for cancer therapy requires a complete understanding of the mathematical models upon which these optimized simulation-based strategies are formulated. This study investigates the…

Quantitative Methods · Quantitative Biology 2025-11-21 Changin Oh , Kathleen P. Wilkie

Precision medicine leverages patient heterogeneity to estimate individualized treatment regimens, formalized, data-driven approaches designed to match patients with optimal treatments. In the presence of competing events, where multiple…

Radiation therapy outcomes are decided by two key parameters, dose and timing, whose best values vary substantially across patients. This variability is especially critical in the treatment of brain cancer, where fractionated or staged…

Medical Physics · Physics 2025-06-24 Hao Peng , Steve Jiang , Robert Timmerman

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