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Prediction models developed before the introduction of a new treatment may be used to estimate treatment effects of newly introduced treatments. One approach, known as model-based clinical evaluation in radiotherapy, does this by comparing…

For many complex diseases, there is a wide variety of ways in which an individual can manifest the disease. The challenge of personalized medicine is to develop tools that can accurately predict the trajectory of an individual's disease,…

机器学习 · 统计学 2016-01-22 Peter Schulam , Suchi Saria

In many medical and business applications, researchers are interested in estimating individualized treatment effects using data from a randomized experiment. For example in medical applications, doctors learn the treatment effects from…

统计方法学 · 统计学 2022-03-01 Kevin Wu Han , Han Wu

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

统计方法学 · 统计学 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

Precision medicine is an emerging scientific topic for disease treatment and prevention that takes into account individual patient characteristics. It is an important direction for clinical research, and many statistical methods have been…

统计方法学 · 统计学 2017-02-17 Jingxiang Chen , Haoda Fu , Xuanyao He , Michael R. Kosorok , Yufeng Liu

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…

统计方法学 · 统计学 2023-09-22 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

We propose a novel method for estimating heterogeneous treatment effects based on the fused lasso. By first ordering samples based on the propensity or prognostic score, we match units from the treatment and control groups. We then run the…

Decision curve analysis can be used to determine whether a personalized model for treatment benefit would lead to better clinical decisions. Decision curve analysis methods have been described to estimate treatment benefit using data from a…

统计方法学 · 统计学 2023-03-01 Konstantina Chalkou , Andrew J. Vickers , Fabio Pellegrini , Andrea Manca , Georgia Salanti

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

统计方法学 · 统计学 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

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…

统计方法学 · 统计学 2022-06-24 Jiuyong Li , Lin Liu , Shisheng Zhang , Saisai Ma , Thuc Duy Le , Jixue Liu

Much evidence in comparative effectiveness research is based on observational studies. Researchers who conduct observational studies typically assume that there are no unobservable differences between the treated and control groups.…

应用统计 · 统计学 2020-01-23 Luke Keele , Stephen O'Neill , Richard Grieve

The burden of diseases is rising worldwide, with unequal treatment efficacy for patient populations that are underrepresented in clinical trials. Healthcare, however, is driven by the average population effect of medical treatments and,…

机器学习 · 计算机科学 2024-02-08 Ghadeer O. Ghosheh , Moritz Gögl , Tingting Zhu

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…

统计方法学 · 统计学 2020-08-11 Tianxi Cai , Tony Cai , Zijian Guo

In observational studies, the recorded treatment assignment is not purely random, but it is influenced by external factors such as patient characteristics, reimbursement policies, and existing guidelines. Therefore, the treatment effect can…

统计方法学 · 统计学 2024-09-02 Sara Poletto , Enrico Longato , Erica Tavazzi , Martina Vettoretti

The effectiveness of personalized oncology treatments ultimately depends on whether outcomes can be causally attributed to the treatment. Advances in precision oncology have improved molecular profiling of individuals, and tailored…

应用统计 · 统计学 2025-09-29 Lydia Jang , Stefan Konigorski

In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the…

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…

Treating children with autism spectrum disorders (ASD) with behavioral interventions, such as Pivotal Response Treatment (PRT), has shown promise in recent studies. However, deciding which therapy is best for a given patient is largely by…

In many practical tasks it is needed to estimate an effect of treatment on individual level. For example, in medicine it is essential to determine the patients that would benefit from a certain medicament. In marketing, knowing the persons…

统计方法学 · 统计学 2019-02-28 Aleksey Buzmakov

We propose Causal Interaction Trees for identifying subgroups of participants that have enhanced treatment effects using observational data. We extend the Classification and Regression Tree algorithm by using splitting criteria that focus…

统计方法学 · 统计学 2021-12-08 Jiabei Yang , Issa J. Dahabreh , Jon A. Steingrimsson