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Response-Adaptive Randomization (RAR) is part of a wider class of data-dependent sampling algorithms, for which clinical trials are typically used as a motivating application. In that context, patient allocation to treatments is determined…

Methodology · Statistics 2022-06-09 David S. Robertson , Kim May Lee , Boryana C. Lopez-Kolkovska , Sofia S. Villar

Although optimal control theory has been used for the theoretical study of anti-cancerous drugs scheduling optimization, with the aim of reducing the primary tumor volume, the effect on metastases is often ignored. Here, we use a previously…

Tissues and Organs · Quantitative Biology 2013-06-21 Sebastien Benzekry , Philip Hahnfeldt

Personalized medicine has gained much popularity recently as a way of providing better healthcare by tailoring treatments to suit individuals. Our research, motivated by the UK INTERVAL blood donation trial, focuses on estimating the…

Methodology · Statistics 2023-02-24 Yuejia Xu , Angela M. Wood , David J. Roberts , Brian D. M. Tom

We propose an adaptive sequential framework for testing two simple hypotheses that analytically ensures finite exposure to the less effective treatment. Our proposed procedure employs a likelihood ratio-driven adaptive allocation rule,…

Statistics Theory · Mathematics 2025-11-26 Sampurna Kundu , Jayant Jha , Subir Kumar Bhandari

Given n experiment subjects with potentially heterogeneous covariates and two possible treatments, namely active treatment and control, this paper addresses the fundamental question of determining the optimal accuracy in estimating the…

Machine Learning · Statistics 2024-11-13 Jiachun Li , David Simchi-Levi , Yunxiao Zhao

A treatment regime is a deterministic function that dictates personalized treatment based on patients' individual prognostic information. There is a fast-growing interest in finding optimal treatment regimes to maximize expected long-term…

Statistics Theory · Mathematics 2016-11-25 Runchao Jiang , Wenbin Lu , Rui Song , Marie Davidian

Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. The main goal is to make the process of introducing new medical interventions to patients more efficient by reducing the cost and the…

Machine Learning · Computer Science 2014-11-17 Ognjen Arandjelovic

Health policy decisions regarding patient treatment strategies require consideration of both treatment effectiveness and cost. Optimizing treatment rules with respect to effectiveness may result in prohibitively expensive strategies; on the…

Methodology · Statistics 2021-10-20 Nicholas Illenberger , Andrew J. Spieker , Nandita Mitra

Clinical trials usually target average treatment effects, but treatment decisions are made for individuals. This tension motivates a common criticism of evidence-based medicine: a treatment that is beneficial on average may be inappropriate…

Applications · Statistics 2026-05-29 Zach Shahn , Mats Stensrud

The question of how individual patient data from cohort studies or historical clinical trials can be leveraged for designing more powerful, or smaller yet equally powerful, clinical trials becomes increasingly important in the era of…

Methodology · Statistics 2022-09-01 Sandra Siegfried , Stephen Senn , Torsten Hothorn

Recent shifts in global health priorities have positioned Population Health Management (PHM) as a central area of focus. However, optimizing PHM strategies presents several challenges: managing high-dimensional patient covariates, tracking…

Optimization and Control · Mathematics 2026-04-10 Daniel Adelman , Alba V Olivares-Nadal , Miaolan Xie

Trials enroll a large number of subjects in order to attain power, making them expensive and time-consuming. Sample size calculations are often performed with the assumption of an unadjusted analysis, even if the trial analysis plan…

Methodology · Statistics 2021-07-06 Alejandro Schuler

Multi-stage (designed) procedures, obtained by splitting the sampling budget suitably across stages, and designing the sampling at a particular stage based on information about the parameter obtained from previous stages, are often…

Methodology · Statistics 2014-01-08 Atul Mallik , Moulinath Banerjee , George Michailidis

We study best-arm identification in stochastic multi-armed bandits under the fixed-confidence setting, focusing on instances with multiple optimal arms. Unlike prior work that addresses the unknown-number-of-optimal-arms case, we consider…

Machine Learning · Computer Science 2026-03-05 Lan V. Truong

Preferably in two- or three-arm randomized clinical trials, a few (2,3) correlated multiple primary endpoints are considered. In addition to the closed testing principle based on different global tests, two max(maxT) tests are compared with…

Applications · Statistics 2021-03-16 Ludwig A. Hothorn , Siegfried Kropf

Recently multi-armed bandit problem arises in many real-life scenarios where arms must be sampled in batches, due to limited time the agent can wait for the feedback. Such applications include biological experimentation and online…

Machine Learning · Statistics 2023-12-22 Shengyu Cao , Simai He , Ruoqing Jiang , Jin Xu , Hongsong Yuan

Clinical trials are notorious for their high failure rates and steep costs, leading to wasted time and resources spend, prolonged development timelines, and delayed patient access to new therapies. A key contributor to these failures is…

Quantum Physics · Physics 2026-01-19 Laia Domingo , Christine Johnson

Longitudinal cluster randomized trials (L-CRTs) are increasingly used to evaluate the cost-effectiveness of healthcare interventions across multiple assessment periods, yet design methods for powering these trials remain underdeveloped.…

Methodology · Statistics 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

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

Any strategy used to distribute a robot ensemble over a set of sequential tasks is subject to inaccuracy due to robot-level uncertainties and environmental influences on the robots' behavior. We approach the problem of inaccuracy during…

Robotics · Computer Science 2022-12-21 Thales C. Silva , Victoria Edwards , M. Ani Hsieh
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