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Related papers: Individual Treatment Effect Prediction for ALS Pat…

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In recent years, research interest in personalised treatments has been growing. However, treatment effect heterogeneity and possibly time-varying treatment effects are still often overlooked in clinical studies. Statistical tools are needed…

Methodology · Statistics 2023-10-27 Caterina Gregorio , Giovanni Baj , Giulia Barbati , Francesca Ieva

Precision medicine seeks to match patients with treatments that produce the greatest benefit. The Predicted Individual Treatment Effect (PITE)-the difference between predicted outcomes under treatment and control-quantifies this benefit but…

Applications · Statistics 2026-02-09 Pamela M. Chiroque-Solano , M Lee Van Horn , Thomas Jaki

Amyotrophic lateral sclerosis (ALS) is a severe disease with a typical survival of 3-5 years after symptom onset. Current treatments offer only limited life extension, and the variability in patient responses highlights the need for…

Machine Learning · Computer Science 2025-11-05 Guillaume Tejedor , Veronika Peralta , Nicolas Labroche , Patrick Marcel , Hélène Blasco , Hugo Alarcan

Given two possible treatments, there may exist subgroups who benefit greater from one treatment than the other. This problem is relevant to the field of marketing, where treatments may correspond to different ways of selling a product. It…

Machine Learning · Statistics 2016-05-16 Derek Feng , Xiaofei Wang

In biomedical science, analyzing treatment effect heterogeneity plays an essential role in assisting personalized medicine. The main goals of analyzing treatment effect heterogeneity include estimating treatment effects in clinically…

Methodology · Statistics 2022-12-06 Waverly Wei , Maya Petersen , Mark J van der Laan , Zeyu Zheng , Chong Wu , Jingshen Wang

Effect modification occurs while the effect of the treatment is not homogeneous across the different strata of patient characteristics. When the effect of treatment may vary from individual to individual, precision medicine can be improved…

Treatment effects vary across different patients and estimation of this variability is important for clinical decisions. The aim is to develop a model to estimate the benefit of alternative treatment options for individual patients. Hence,…

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

Machine Learning · Statistics 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

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

Assessing treatment effect heterogeneity (TEH) in clinical trials is crucial, as it provides insights into the variability of treatment responses among patients, influencing important decisions related to drug development. Furthermore, it…

Applications · Statistics 2026-02-06 Konstantinos Sechidis , Cong Zhang , Sophie Sun , Yao Chen , Asher Spector , Björn Bornkamp

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

In this paper we present a data-adaptive estimation procedure for estimation of average treatment effects in a time-to-event setting based on generalized random forests. In these kinds of settings, the definition of causal effect parameters…

Methodology · Statistics 2021-04-28 Helene C. W. Rytgaard , Claus T. Ekstrøm , Lars V. Kessing , Thomas A. Gerds

Because different patients may response quite differently to the same drug or treatment, there is increasing interest in discovering individualized treatment rule. In particular, people are eager to find the optimal individualized treatment…

Methodology · Statistics 2016-04-14 Wei Xiao , Hao Helen Zhang , Wenbin Lu

Practitioners in medicine, business, political science, and other fields are increasingly aware that decisions should be personalized to each patient, customer, or voter. A given treatment (e.g. a drug or advertisement) should be…

Machine Learning · Statistics 2018-06-15 Alejandro Schuler , Michael Baiocchi , Robert Tibshirani , Nigam Shah

Some patients benefit from a treatment while others may do so less or do not benefit at all. We have previously developed a two-stage network meta-regression prediction model that synthesized randomized trials and evaluates how treatment…

To promote precision medicine, individualized treatment regimes (ITRs) are crucial for optimizing the expected clinical outcome based on patient-specific characteristics. However, existing ITR research has primarily focused on scenarios…

Methodology · Statistics 2024-02-20 Chang Wang , Lu Wang

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

In this paper, we propose the use of causal inference techniques for survival function estimation and prediction for subgroups of the data, upto individual units. Tree ensemble methods, specifically random forests were modified for this…

Econometrics · Economics 2018-03-23 Vikas Ramachandra

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease characterized by a rapid motor decline, leading to respiratory failure and subsequently to death. In this context, researchers have sought for models to automatically…

Machine Learning · Computer Science 2019-07-31 Telma Pereira , Sofia Pires , Marta Gromicho , Susana Pinto , Mamede de Carvalho , Sara C. Madeira