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A common problem faced in clinical studies is that of estimating the effect of the most effective (e.g., the one having the largest mean) treatment among $k~(\geq2)$ available treatments. The most effective treatment is adjudged based on…

Statistics Theory · Mathematics 2022-09-20 Masihuddin , Neeraj Misra

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

Response-adaptive randomisation (RAR) can considerably improve the chances of a successful treatment outcome for patients in a clinical trial by skewing the allocation probability towards better performing treatments as data accumulates.…

Applications · Statistics 2017-03-14 Sofia S. Villar , Jack Bowden , James Wason

Respondent-driven sampling (RDS) is a widely used method for sampling from hard-to-reach human populations, especially groups most at-risk for HIV/AIDS. Data are collected through a peer-referral process in which current sample members…

Methodology · Statistics 2012-09-28 Krista J. Gile , Lisa G. Johnston , Matthew J. Salganik

The success of many RL techniques heavily relies on human-engineered dense rewards, which typically demand substantial domain expertise and extensive trial and error. In our work, we propose DrS (Dense reward learning from Stages), a novel…

Machine Learning · Computer Science 2024-04-26 Tongzhou Mu , Minghua Liu , Hao Su

This paper proposes a novel criterion for the allocation of patients in Phase~I dose-escalation clinical trials aiming to find the maximum tolerated dose (MTD). Conventionally, using a model-based approach the next patient is allocated to…

Methodology · Statistics 2018-07-17 Pavel Mozgunov , Thomas Jaki

We propose Locally Optimal Restricted Designs (LORDs) for phase I/II dose-finding studies that focus on both efficacy and toxicity outcomes. As an illustrative application, we find various LORDs for a 4-parameter continuation-ratio (CR)…

Methodology · Statistics 2025-09-09 Oleksandr Sverdlov , Yevgen Ryeznik , Weng Kee Wong

A main research goal in various studies is to use an observational data set and provide a new set of counterfactual guidelines that can yield causal improvements. Dynamic Treatment Regimes (DTRs) are widely studied to formalize this…

Machine Learning · Computer Science 2023-06-06 Soroush Saghafian

Randomised Controlled Trials (RCTs) are the gold standard for estimating treatment effects across many fields of science. Technology companies have adopted A/B-testing methods as a modern RCT counterpart, where end-users are randomly…

Social and Information Networks · Computer Science 2024-09-20 Olivier Jeunen

One common approach for dose optimization is a two-stage design, which initially conducts dose escalation to identify the maximum tolerated dose (MTD), followed by a randomization stage where patients are assigned to two or more doses to…

Methodology · Statistics 2024-11-11 Yixuan Zhao , Rachael Liu , Jianchang Lin , Ying Yuan

The paper presents an approach for building consistent and applicable clinical decision support systems (CDSSs) using a data-driven predictive model aimed at resolving the problem of low applicability and scalability of CDSSs in real-world…

Artificial Intelligence · Computer Science 2022-02-18 Sergey V. Kovalchuk , Georgy D. Kopanitsa , Ilia V. Derevitskii , Daria A. Savitskaya

The traditional more-is-better dose selection paradigm, developed based on cytotoxic chemotherapeutics, is often problematic When applied to the development of novel molecularly targeted agents (e.g., kinase inhibitors, monoclonal…

Methodology · Statistics 2022-11-04 Liyun Jiang , Ying Yuan

Reinforcement learning (RL) has garnered increasing recognition for its potential to optimise dynamic treatment regimes (DTRs) in personalised medicine, particularly for drug dosage prescriptions and medication recommendations. However, a…

Machine Learning · Computer Science 2024-05-30 Zhiyao Luo , Mingcheng Zhu , Fenglin Liu , Jiali Li , Yangchen Pan , Jiandong Zhou , Tingting Zhu

The goal of precision medicine is to provide individualized treatment at each stage of chronic diseases, a concept formalized by Dynamic Treatment Regimes (DTR). These regimes adapt treatment strategies based on decision rules learned from…

Methodology · Statistics 2025-06-09 Sophia Yazzourh , Nicolas Savy , Philippe Saint-Pierre , Michael R. Kosorok

Recently, diabetic retinopathy (DR) screening utilizing ultra-wide optical coherence tomography angiography (UW-OCTA) has been used in clinical practices to detect signs of early DR. However, developing a deep learning-based DR analysis…

Image and Video Processing · Electrical Eng. & Systems 2022-10-19 Gitaek Kwon , Eunjin Kim , Sunho Kim , Seongwon Bak , Minsung Kim , Jaeyoung Kim

Subgroup analysis of treatment effects plays an important role in applications from medicine to public policy to recommender systems. It allows physicians (for example) to identify groups of patients for whom a given drug or treatment is…

Machine Learning · Statistics 2020-10-20 Hyun-Suk Lee , Yao Zhang , William Zame , Cong Shen , Jang-Won Lee , Mihaela van der Schaar

A master protocol trial uses a single overarching protocol to test multiple therapies, often across several diseases or subtypes. Although such trials offer considerable flexibility and efficiency, their constrained and non-uniform…

Methodology · Statistics 2026-04-29 Shiyu Wan , Yuhan Qian , Yanyao Yi , Nicole Mayer-Hamblett , Patrick J. Heagerty , Ting Ye

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

Methodology · Statistics 2018-07-24 Ruitao Lin , Ying Yuan

Many multi-genic systemic diseases such as neurological disorders, inflammatory diseases, and the majority of cancers do not have effective treatments yet. Reinforcement learning powered systems pharmacology is a potentially effective…

Biomolecules · Quantitative Biology 2022-02-25 Ryan K. Tan , Yang Liu , Lei Xie

This article presents the general framework of theranostic digital twins (TDTs) in computational nuclear medicine, designed to support clinical decision-making and improve cancer patient prognosis through personalized radiopharmaceutical…

Other Quantitative Biology · Quantitative Biology 2025-11-07 Marc Ryhiner , Yangmeihui Song , Babak Saboury , Gerhard Glatting , Arman Rahmim , Kuangyu Shi