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Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li

Dynamic Treatment Regimes (DTRs) provide a systematic framework for optimizing sequential decision-making in chronic disease management, where therapies must adapt to patients' evolving clinical profiles. Inverse probability weighting (IPW)…

Methodology · Statistics 2026-03-26 Chloe Si , David A. Stephens , Erica E. M. Moodie

In a sequential multiple-assignment randomized trial (SMART), a sequence of treatments is given to a patient over multiple stages. In each stage, randomization may be done to allocate patients to different treatment groups. Even though…

Methodology · Statistics 2024-01-09 Rik Ghosh , Bibhas Chakraborty , Inbal Nahum-Shani , Megan E. Patrick , Palash Ghosh

Causal effect estimation for dynamic treatment regimes (DTRs) contributes to sequential decision making. However, censoring and time-dependent confounding under DTRs are challenging as the amount of observational data declines over time due…

Machine Learning · Statistics 2021-09-27 Adi Lin , Jie Lu , Junyu Xuan , Fujin Zhu , Guangquan Zhang

Recent advances in dynamic treatment regimes (DTRs) facilitate the search for optimal treatments, which are tailored to individuals' specific needs and able to maximize their expected clinical benefits. However, existing algorithms relying…

Machine Learning · Statistics 2024-10-18 Hanwen Ye , Wenzhuo Zhou , Ruoqing Zhu , Annie Qu

Joint modelling of longitudinal and survival data is increasingly used in clinical trials on cancer. In prostate cancer for example, these models permit to account for the link between longitudinal measures of prostate-specific antigen…

Dynamic treatment regimes (DTRs) are used in medicine to tailor sequential treatment decisions to patients by considering patient heterogeneity. Common methods for learning optimal DTRs, however, have shortcomings: they are typically based…

Machine Learning · Statistics 2023-06-21 Theresa Blümlein , Joel Persson , Stefan Feuerriegel

A dynamic treatment regime (DTR) is an approach to delivering precision medicine that uses patient characteristics to guide treatment decisions for optimal health outcomes. Numerous methods have been proposed for DTR estimation, including…

Methodology · Statistics 2025-02-03 Adel Ahmadi Nadi , Michael Wallace

The optimal dynamic treatment rule (ODTR) framework offers an approach for understanding which kinds of patients respond best to specific treatments -- in other words, treatment effect heterogeneity. Recently, there has been a proliferation…

Objectives To investigate the use of a Bayesian joint modelling approach to predict overall survival (OS) from immature clinical trial data using an intermediate biomarker. To compare the results with a typical parametric approach of…

Estimating optimal dynamic treatment regimes (DTRs) using observational data is often challenged by nonignorable missing covariates arsing from informative monitoring of patients in clinical practice. To address nonignorable missingness of…

Methodology · Statistics 2025-07-01 Jian Sun , Bo Fu , Li Su

Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the…

Methodology · Statistics 2024-06-11 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

Sequential, multiple assignment, randomized trial (SMART) designs have become increasingly popular in the field of precision medicine by providing a means for comparing sequences of treatments tailored to the individual patient, i.e.,…

Applications · Statistics 2018-04-13 William J. Artman , Inbal Nahum-Shani , Tianshuang Wu , James R. McKay , Ashkan Ertefaie

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

Methodology · Statistics 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

The optimal prophylaxis, and treatment if the prophylaxis fails, for a disease may be best evaluated using a sequential multiple assignment randomised trial (SMART). A SMART is a multi-stage study that randomises a participant to an initial…

Methodology · Statistics 2022-03-25 Robert K. Mahar , Katherine J. Lee , Bibhas Chakraborty , Agus Salim , Julie A. Simpson

Repeated measures of biomarkers have the potential of explaining hazards of survival outcomes. In practice, these measurements are intermittently measured and are known to be subject to substantial measurement error. Joint modelling of…

Applications · Statistics 2019-12-12 Lisa McFetridge , Ozgur Asar , Jonas Wallin

Large health care data repositories such as electronic health records (EHR) open new opportunities to derive individualized treatment strategies for complicated diseases such as sepsis. In this paper, we consider the problem of estimating…

Statistics Theory · Mathematics 2023-10-03 Nilanjana Laha , Aaron Sonabend-W , Rajarshi Mukherjee , Tianxi Cai

The sequential multiple assignment randomized trial (SMART) is the gold standard trial design to generate data for the evaluation of multi-stage treatment regimes. As with conventional (single-stage) randomized clinical trials, interim…

Methodology · Statistics 2023-09-13 Cole Manschot , Eric Laber , Marie Davidian

Sequential multiple assignment randomized trials (SMARTs) have grown in popularity in recent years, and many of their study protocols propose conducting a cost effectiveness analysis of the adaptive strategies embedded within them. The cost…

In this work, we examine recently developed methods for Bayesian inference of optimal dynamic treatment regimes (DTRs). DTRs are a set of treatment decision rules aimed at tailoring patient care to patient-specific characteristics, thereby…

Methodology · Statistics 2023-03-28 Daniel Rodriguez Duque , Erica E. M. Moodie , David A. Stephens