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Objective: In randomized clinical trials, prediction models can be used to explore the relationships between patients' variables (e.g., clinical, pathological, or lifestyle variables, and also biomarker or genomic data) and treatment effect…

Quantitative Methods · Quantitative Biology 2026-02-03 Elvire Roblin , Paul-Henry Cournède , Stefan Michiels

A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the…

Methodology · Statistics 2025-10-14 David S. Robertson , Thomas Jaki

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Clinical prediction models (CPMs) are used to predict clinically relevant outcomes or events. Typically, prognostic CPMs are derived to predict the risk of a single future outcome. However, with rising emphasis on the prediction of…

Methodology · Statistics 2020-10-29 Glen P. Martin , Matthew Sperrin , Kym I. E. Snell , Iain Buchan , Richard D. Riley

In domains where transparency and trustworthiness are crucial, such as healthcare, rule-based systems are widely used and often preferred over black-box models for decision support systems due to their inherent interpretability. However, as…

Machine Learning · Computer Science 2025-06-18 Christel Sirocchi , Damiano Verda

The goal of uplift modeling is to recommend actions that optimize specific outcomes by determining which entities should receive treatment. One common approach involves two steps: first, an inference step that estimates conditional average…

Machine Learning · Computer Science 2025-05-21 Simon De Vos , Christopher Bockel-Rickermann , Stefan Lessmann , Wouter Verbeke

INTRODUCTION: The pharmacological treatment of Major Depressive Disorder (MDD) relies on a trial-and-error approach. We introduce an artificial intelligence (AI) model aiming to personalize treatment and improve outcomes, which was deployed…

Researchers commonly use difference-in-differences (DiD) designs to evaluate public policy interventions. While methods exist for estimating effects in the context of binary interventions, policies often result in varied exposures across…

Methodology · Statistics 2025-02-07 Gary Hettinger , Youjin Lee , Nandita Mitra

The aim is to create a method for accurately estimating the duration of post-cancer treatment, particularly focused on chemotherapy, to optimize patient care and recovery. This initiative seeks to improve the effectiveness of cancer…

Computers and Society · Computer Science 2025-04-24 Joyee Chakraborty , Mazahrul Islam Tohin , Danbir Rashid , Tanjil Ahmed Tanmoy , Md. Jehadul Islam Mony

Problem definition: Personalized medicine (PM) seeks the best treatment for each patient among a set of available treatment methods. Since a specific treatment does not work well on all patients, traditionally, the best treatment was…

Optimization and Control · Mathematics 2023-08-03 Jianzhong Du , Siyang Gao , Chun-Hung Chen

It is common to compare individualized treatment rules based on the value function, which is the expected potential outcome under the treatment rule. Although the value function is not point-identified when there is unmeasured confounding,…

Methodology · Statistics 2020-09-25 Bo Zhang , Jordan Weiss , Dylan S Small , Qingyuan Zhao

Studies often report estimates of the average treatment effect. While the ATE summarizes the effect of a treatment on average, it does not provide any information about the effect of treatment within any individual. A treatment strategy…

Methodology · Statistics 2025-06-13 Nicholas T. Williams , Katherine L. Hoffman Iván Díaz , Kara E. Rudolph

With an increasing focus on precision medicine in medical research, numerous studies have been conducted in recent years to clarify the relationship between treatment effects and patient characteristics. The treatment effects for patients…

Methodology · Statistics 2023-09-22 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

Network meta-analysis (NMA) allow combining efficacy information from multiple comparisons from trials assessing different therapeutic interventions for a given disease and to estimate unobserved comparisons from a network of observed…

Methodology · Statistics 2016-04-08 Victoria Nyaga , Marc Aerts , Marc Arbyn

Restricted mean survival time (RMST) models have gained popularity when analyzing time-to-event outcomes because RMST models offer more straightforward interpretations of treatment effects with fewer assumptions than hazard ratios commonly…

Methodology · Statistics 2023-10-23 Kaiyuan Hua , Xiaofei Wang , Hwanhee Hong

Dynamic treatment regimes have been proposed to personalize treatment decisions by utilizing historical patient data, but they may not always improve on the current standard of care. It is thus meaningful to integrate the standard of care…

Applications · Statistics 2025-12-11 Johannes Hruza , Arvid Sjölander , Erin Gabriel , Samir Bhatt , Michael Sachs

A treatment benefit predictor (TBP) is a function that maps patient characteristics to an estimate of the treatment benefit for that patient. Such predictors support optimizing individualized treatment decisions, which are central to…

Methodology · Statistics 2025-09-30 Yuan Xia , Mohsen Sadatsafavi , Paul Gustafson

Machine learning (ML) estimates of conditional average treatment effects (CATE) can guide policy decisions, either by allowing targeting of individuals with beneficial CATE estimates, or as inputs to decision trees that optimise overall…

Econometrics · Economics 2023-10-04 Julia Hatamyar , Noemi Kreif

We develop a prediction-based prescriptive model for learning optimal personalized treatments for patients based on their Electronic Health Records (EHRs). Our approach consists of: (i) predicting future outcomes under each possible therapy…

Machine Learning · Statistics 2018-12-06 Ruidi Chen , Ioannis Paschalidis

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

Methodology · Statistics 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong