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Non-adherence to assigned treatment is common in randomised controlled trials (RCTs). Recently, there has been an increased interest in estimating causal effects of treatment received, for example the so-called local average treatment…

Methodology · Statistics 2018-12-05 Karla DiazOrdaz , James Carpenter

In this paper, a Bayesian approach is developed for simultaneously comparing multiple experimental treatments with a common control treatment in an exploratory clinical trial. The sample size is set to ensure that, at the end of the study,…

Statistics Theory · Mathematics 2019-11-14 John Whitehead , Faye Cleary , Amanda Turner

Although there is an extensive statistical literature showing the disadvantages of discretizing continuous variables, categorization is a common practice in clinical research which results in substantial loss of information. A large…

Methodology · Statistics 2017-08-17 Márcio Augusto Diniz , Mourad Tighiouart , André Rogatko

The conditional average treatment effect (CATE) is the best measure of individual causal effects given baseline covariates. However, the CATE only captures the (conditional) average, and can overlook risks and tail events, which are…

Machine Learning · Statistics 2025-06-05 Nathan Kallus , Miruna Oprescu

In precision medicine, identifying optimal sequences of decision rules, termed dynamic treatment regimes (DTRs), is an important undertaking. One approach investigators may take to infer about optimal DTRs is via Bayesian dynamic Marginal…

Methodology · Statistics 2022-06-09 Daniel Rodriguez Duque , David A. Stephens , Erica E. M. Moodie

Purpose: LATTICE, a form of spatially fractionated radiation therapy that delivers high-dose peaks and low-dose valleys within the target, has been clinically utilized for treating bulky tumors. However, its application to…

Medical Physics · Physics 2025-09-08 Nimita Shinde , Weijie Zhang , Yuting Lin , Hao Gao

A mathematical model for time development of metastases and their distribution in size and carrying capacity is presented. The model is used to theoretically investigate anti-cancer therapies such as surgery and chemical treatments…

Tissues and Organs · Quantitative Biology 2013-06-21 Sebastien Benzekry , Dominique Barbolosi , Assia Benabdallah , Florence Hubert , Philip Hahnfeldt

Recently there has been much work on early phase cancer designs that incorporate both toxicity and efficacy data, called Phase I-II designs because they combine elements of both phases. However, they do not explicitly address the Phase II…

Methodology · Statistics 2014-02-12 Jay Bartroff , Tze Leung Lai , Balasubramanian Narasimhan

In precision medicine, Dynamic Treatment Regimes (DTRs) are treatment protocols that adapt over time in response to a patient's observed characteristics. A DTR is a set of decision functions that takes an individual patient's information as…

Methodology · Statistics 2022-03-17 Cong Jiang , Michael Wallace , Mary Thompson

Drug discovery is a multi-stage process that comprises two costly major steps: pre-clinical research and clinical trials. Among its stages, lead optimization easily consumes more than half of the pre-clinical budget. We propose a combined…

Machine Learning · Computer Science 2020-11-30 Leili Zhang , Giacomo Domeniconi , Chih-Chieh Yang , Seung-gu Kang , Ruhong Zhou , Guojing Cong

One of the main goals of sequential, multiple assignment, randomized trials (SMART) is to find the most efficacious design embedded dynamic treatment regimes. The analysis method known as multiple comparisons with the best (MCB) allows…

Methodology · Statistics 2020-08-07 William J. Artman , Ashkan Ertefaie , Kevin G. Lynch , James R. McKay

In clinical trials, patients may discontinue treatments prematurely, breaking the initial randomization and, thus, challenging inference. Stakeholders in drug development are generally interested in going beyond the Intention-To-Treat (ITT)…

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

Immunotherapy has transformed cancer treatment, yet its delayed therapeutic effects often lead to non-proportional hazards, rendering many conventional phase II designs underpowered and prone to type I error inflation. To address this…

Methodology · Statistics 2025-09-03 Zhongheng Cai , Haitao Pan

Model informed precision dosing (MIPD) is a Bayesian framework to individualize drug therapy based on prior knowledge and patient-specific monitoring data. Typically, prior knowledge results from controlled clinical trials with a more…

Computation · Statistics 2025-05-27 Franziska Thoma , Niklas Hartung , Manfred Opper , Wilhelm Huisinga

Model-informed precision dosing (MIPD) is a quantitative dosing framework that combines prior knowledge on the drug-disease-patient system with patient data from therapeutic drug/ biomarker monitoring (TDM) to support individualized dosing…

Optimization and Control · Mathematics 2021-06-08 Corinna Maier , Jana de Wiljes , Niklas Hartung , Charlotte Kloft , Wilhelm Huisinga

Diffusion Language Models (DLMs) have recently emerged as a strong alternative to autoregressive language models (LMs). DLMs offer comparable accuracy with faster inference speed via parallel decoding. However, standard DLM decoding…

Machine Learning · Computer Science 2025-11-27 Hengyu Fu , Baihe Huang , Virginia Adams , Charles Wang , Venkat Srinivasan , Jiantao Jiao

Precision medicine promises to transform health care by offering individualised treatments that dramatically improve clinical outcomes. A necessary prerequisite is to identify subgroups of patients who respond differently to different…

Machine Learning · Computer Science 2026-03-03 Adam Marcus , Paul Agapow

For large libraries of small molecules, exhaustive combinatorial chemical screens become infeasible to perform when considering a range of disease models, assay conditions, and dose ranges. Deep learning models have achieved state of the…

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
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