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In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Many policies involve dynamics in their treatment assignments, where individuals receive sequential interventions over multiple stages. We study estimation of an optimal dynamic treatment regime that guides the optimal treatment assignment…

Econometrics · Economics 2024-09-04 Shosei Sakaguchi

Clinicians and researchers alike are increasingly interested in how best to personalize interventions. A dynamic treatment regimen (DTR) is a sequence of pre-specified decision rules which can be used to guide the delivery of a sequence of…

Recent development in the data-driven decision science has seen great advances in individualized decision making. Given data with individual covariates, treatment assignments and outcomes, policy makers best individualized treatment rule…

Machine Learning · Statistics 2020-06-29 Weibin Mo , Zhengling Qi , Yufeng Liu

Machine learning methods for estimating treatment effect heterogeneity promise greater flexibility than existing methods that test a few pre-specified hypotheses. However, one problem these methods can have is that it can be challenging to…

Econometrics · Economics 2024-08-05 Patrick Rehill

Accurately estimating personalized treatment effects within a study site (e.g., a hospital) has been challenging due to limited sample size. Furthermore, privacy considerations and lack of resources prevent a site from leveraging…

Machine Learning · Statistics 2022-06-17 Xiaoqing Tan , Chung-Chou H. Chang , Ling Zhou , Lu Tang

Survival analysis studies and predicts the time of death, or other singular unrepeated events, based on historical data, while the true time of death for some instances is unknown. Survival trees enable the discovery of complex nonlinear…

Machine Learning · Computer Science 2024-01-10 Tim Huisman , Jacobus G. M. van der Linden , Emir Demirović

A core component of precision medicine research involves optimizing individualized treatment rules (ITRs) based on patient characteristics. Many studies used to estimate ITRs are longitudinal in nature, collecting outcomes over time. Yet,…

Methodology · Statistics 2024-05-29 Lanqiu Yao , Thaddeus Tarpey

Causal inference has gained much popularity in recent years, with interests ranging from academic, to industrial, to educational, and all in between. Concurrently, the study and usage of neural networks has also grown profoundly (albeit at…

Machine Learning · Statistics 2024-05-07 Demetrios Papakostas , Andrew Herren , P. Richard Hahn , Francisco Castillo

In previous research, we developed methods to train decision trees (DT) as agents for reinforcement learning tasks, based on deep reinforcement learning (DRL) networks. The samples from which the DTs are built, use the environment's state…

Machine Learning · Computer Science 2024-12-09 Raphael C. Engelhardt , Marcel J. Meinen , Moritz Lange , Laurenz Wiskott , Wolfgang Konen

The field of precision medicine aims to tailor treatment based on patient-specific factors in a reproducible way. To this end, estimating an optimal individualized treatment regime (ITR) that recommends treatment decisions based on patient…

We developed a study design for rare disease clinical trials (RDTs) that efficiently evaluate treatments, promotes access to new treatments during treatment development, and optimizes healthcare resource utilization for future treatment…

Applications · Statistics 2016-07-04 Jian Yong , Sohaib H. Mohammad , Yan Yuan

Clinical trials are critical for drug development but often suffer from expensive and inefficient patient recruitment. In recent years, machine learning models have been proposed for speeding up patient recruitment via automatically…

Machine Learning · Computer Science 2023-07-20 Brandon Theodorou , Cao Xiao , Jimeng Sun

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

Decision Trees (DTs) are commonly used for many machine learning tasks due to their high degree of interpretability. However, learning a DT from data is a difficult optimization problem, as it is non-convex and non-differentiable.…

Machine Learning · Computer Science 2024-08-20 Sascha Marton , Stefan Lüdtke , Christian Bartelt , Heiner Stuckenschmidt

In recent years, the field of precision medicine has seen many advancements. Significant focus has been placed on creating algorithms to estimate individualized treatment rules (ITR), which map from patient covariates to the space of…

Methodology · Statistics 2021-12-09 Kushal S. Shah , Haoda Fu , Michael R. Kosorok

We consider Dynamic Treatment Regimes (DTRs) with One Sided Noncompliance that arise in applications such as digital recommendations and adaptive medical trials. These are settings where decision makers encourage individuals to take…

Econometrics · Economics 2025-05-06 Ravi B. Sojitra , Vasilis Syrgkanis

A dynamic treatment regime is a sequence of medical decisions that adapts to the evolving clinical status of a patient over time. To facilitate personalized care, it is crucial to assess the probability of each available treatment option…

Methodology · Statistics 2024-11-05 Jiefeng Bi , Matteo Borrotti , Bernardo Nipoti

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

Decision-making across various fields, such as medicine, heavily relies on conditional average treatment effects (CATEs). Practitioners commonly make decisions by checking whether the estimated CATE is positive, even though the…

Machine Learning · Computer Science 2025-05-23 Dennis Frauen , Valentyn Melnychuk , Jonas Schweisthal , Mihaela van der Schaar , Stefan Feuerriegel
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