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Clinical trials constitute a critical yet exceptionally challenging and costly stage of drug development (\$2.6B per drug), where protocols are encoded as complex natural language documents, motivating the use of AI systems beyond manual…

Artificial Intelligence · Computer Science 2026-04-06 Sixue Xing , Kerui Wu , Xuanye Xia , Meng Jiang , Jintai Chen , Tianfan Fu

High-dimensional biomarkers such as genomics are increasingly being measured in randomized clinical trials. Consequently, there is a growing interest in developing methods that improve the power to detect biomarker-treatment interactions.…

Methodology · Statistics 2021-04-30 Jixiong Wang , Ashish Patel , James M. S. Wason , Paul J. Newcombe

The primary goal of a two-stage Phase I/II trial is to identify the optimal dose for the following large-scale Phase III trial. Recently, Phase I dose-finding designs have shifted from identifying the maximum tolerated dose (MTD) to the…

Methodology · Statistics 2025-01-16 Hao Sun , Jerry Li

The regression discontinuity (RD) design is widely used for program evaluation with observational data. The primary focus of the existing literature has been the estimation of the local average treatment effect at the existing treatment…

Methodology · Statistics 2024-09-05 Yi Zhang , Eli Ben-Michael , Kosuke Imai

The primary objective of phase I cancer clinical trials is to evaluate the safety of a new experimental treatment and to find the maximum tolerated dose (MTD). We show that the MTD estimation problem can be regarded as a level set…

Machine Learning · Statistics 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

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

Nowadays, more and more clinical trials choose combinational agents as the intervention to achieve better therapeutic responses. However, dose-finding for combinational agents is much more complicated than single agent as the full order of…

Applications · Statistics 2022-08-05 Shu Wang , Ji-Hyun Lee

It is possible to find the optimized radiation dose per session for a radiotherapy (RT) treatment, using a population dynamics model. This has already been done in a previous work for a protocol with 30 sessions and a fixed dose per…

Practical machine learning systems often operate in multiple sequential stages, as seen in ranking and recommendation systems, which typically include a retrieval phase followed by a ranking phase. Effectively assessing prediction…

Information Retrieval · Computer Science 2025-02-04 Yunpeng Xu , Mufang Ying , Wenge Guo , Zhi Wei

A dynamic treatment regime is a sequence of treatment decision rules tailored to an individual's evolving status over time. In precision medicine, much focus has been placed on finding an optimal dynamic treatment regime which, if followed…

Other Statistics · Statistics 2025-10-13 Chunyu Wang , Brian DM Tom

Current clinical decision support systems (DSS) are trained and validated on observational data from the target clinic. This is problematic for treatments validated in a randomized clinical trial (RCT), but not yet introduced in any clinic.…

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

The clinical trial process, a critical phase in drug development, is essential for developing new treatments. The primary goal of interventional clinical trials is to evaluate the safety and efficacy of drug-based treatments for specific…

Machine Learning · Computer Science 2024-07-19 Ling Yue , Jonathan Li , Sixue Xing , Md Zabirul Islam , Bolun Xia , Tianfan Fu , Jintai Chen

Individualizing treatment assignment can improve outcomes for diseases with patient-to-patient variability in comparative treatment effects. When a clinical trial demonstrates that some patients improve on treatment while others do not, it…

Methodology · Statistics 2022-11-02 Nina Galanter , Marco Carone , Ronald C. Kessler , Alex Luedtke

An optimal dynamic treatment regime (DTR) is a sequence of decision rules aimed at providing the best course of treatments individualized to patients. While conventional DTR estimation uses longitudinal data, such data can also be…

Methodology · Statistics 2025-02-06 Larry Dong , Eleanor Pullenayegum , Rodolphe Thiébaut , Olli Saarela

Technological advancements in mobile devices have made it possible to deliver mobile health interventions to individuals. A novel intervention framework that emerges from such advancements is the just-in-time adaptive intervention (JITAI),…

Methodology · Statistics 2020-07-29 Jing Xu , Xiaoxi Yan , Caroline Figueroa , Joseph Jay Williams , Bibhas Chakraborty

The Stepped Wedge Design (SWD) is a form of cluster randomized trial, usually comparing two treatments, which is divided into time periods and sequences, with clusters allocated to sequences. Typically all sequences start with the standard…

Methodology · Statistics 2020-11-06 John N. S. Matthews

Empirical studies using Regression Discontinuity (RD) designs often explore heterogeneous treatment effects based on pretreatment covariates, even though no formal statistical methods exist for such analyses. This has led to the widespread…

This paper presents the first deep reinforcement learning (DRL) framework to estimate the optimal Dynamic Treatment Regimes from observational medical data. This framework is more flexible and adaptive for high dimensional action and state…

Artificial Intelligence · Computer Science 2018-01-30 Ning Liu , Ying Liu , Brent Logan , Zhiyuan Xu , Jian Tang , Yanzhi Wang

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

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