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Efforts to develop biomarker-targeted anti-cancer therapies have progressed rapidly in recent years. Six antibodies acting on programmed death ligand 1 or programmed death 1 pathways were approved in 75 cancer indications between 2015 and…

Methodology · Statistics 2022-10-07 Emily C. Zabor , Alexander M. Kaizer , Nathan A. Pennell , Brian P. Hobbs

In the setting of multi-armed trials, adaptive designs are a popular way to increase estimation efficiency, identify optimal treatments, or maximize rewards to individuals. Recent work has considered the case of estimating the effects of K…

Methodology · Statistics 2026-02-10 Evan T. R. Rosenman , Kristen B. Hunter

Pathologic complete response (pCR) is a common primary endpoint for a phase II trial or even accelerated approval of neoadjuvant cancer therapy. If granted, a two-arm confirmatory trial is often required to demonstrate the efficacy with a…

Methodology · Statistics 2021-05-25 Marta Bofill Roig , Yu Shen , Guadalupe Gómez Melis

Human epidermal growth factor receptor 2 (HER2) is a critical protein in cancer cell growth that signifies the aggressiveness of breast cancer (BC) and helps predict its prognosis. Accurate assessment of immunohistochemically (IHC) stained…

Risk stratification is a key tool in clinical decision-making, yet current approaches often fail to translate sophisticated survival analysis into actionable clinical criteria. We present a novel method for unsupervised machine learning…

In this paper, we aim at solving a class of multiple testing problems under the Bayesian sequential decision framework. Our motivating application comes from binary labeling tasks in crowdsourcing, where the requestor needs to…

Methodology · Statistics 2017-08-29 Xiaoou Li , Yunxiao Chen , Xi Chen , Jingchen Liu , Zhiliang Ying

Adaptive clinical trials rely on interim analyses, flexible stopping, and data-dependent design modifications that complicate statistical guarantees when fixed-horizon test statistics are repeatedly inspected or reused after adaptations.…

Methodology · Statistics 2026-02-09 Alexandra Sokolova , Vadim Sokolov

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

Methodology · Statistics 2022-11-14 Martin Law

Adaptive seamless designs combine confirmatory testing, a domain of phase III trials, with features such as treatment or subgroup selection, typically associated with phase II trials. They promise to increase the efficiency of development…

Methodology · Statistics 2019-01-25 Tim Friede , Nigel Stallard , Nicholas Parsons

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

Self-selected samples are frequently obtained due to different levels of survey participation propensity of the survey individuals. When the survey participation is related to the survey topic of interest, propensity score weighting…

Applications · Statistics 2014-11-24 Sixia Chen , Jae-Kwang Kim

We consider an adaptive experiment for treatment choice and design a minimax and Bayes optimal adaptive experiment with respect to regret. Given binary treatments, the experimenter's goal is to choose the treatment with the highest expected…

Econometrics · Economics 2025-12-10 Masahiro Kato

Group testing is an efficient method for testing a large population to detect infected individuals. In this paper, we consider an efficient adaptive two stage group testing scheme. Using a straightforward analysis, we characterize the…

Methodology · Statistics 2020-08-26 Arjun Kodialam

Breast cancer is the most diagnosed cancer in women, with HER2 status critically guiding treatment decisions. Noninvasive prediction of HER2 status from dynamic contrast-enhanced MRI (DCE-MRI) could streamline diagnostics and reduce…

Quantitative Methods · Quantitative Biology 2025-10-17 Naomi Fridman , Anat Goldstein

This article proposes a novel adaptive design algorithm that can be used to find optimal treatment allocations in N-of-1 clinical trials. This new methodology uses two Laplace approximations to provide a computationally efficient estimate…

Computation · Statistics 2020-07-30 S. G. Jagath Senarathne , Antony M. Overstall , James M. McGree

Covariate adjustment is an approach to improve the precision of trial analyses by adjusting for baseline variables that are prognostic of the primary endpoint. Motivated by the SEARCH Universal HIV Test-and-Treat Trial (2013-2017), we tell…

Methodology · Statistics 2025-12-16 Laura B. Balzer , Mark J. van der Laan , Maya L. Petersen

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

Medication recommendation is a crucial task in healthcare, especially for patients with complex medical conditions. However, existing methods often struggle to effectively balance the reuse of historical medications with the introduction of…

Artificial Intelligence · Computer Science 2025-07-08 Feiyue Wu , Tianxing Wu , Shenqi Jing

We develop methodology for a multistage decision problem with flexible number of stages in which the rewards are survival times that are subject to censoring. We present a novel Q-learning algorithm that is adjusted for censored data and…

Statistics Theory · Mathematics 2012-05-31 Yair Goldberg , Michael R. Kosorok

Technological advancements in the field of mobile devices and wearable sensors have helped overcome obstacles in the delivery of care, making it possible to deliver behavioral treatments anytime and anywhere. Increasingly the delivery of…

Applications · Statistics 2017-11-13 Walter Dempsey , Peng Liao , Santosh Kumar , Susan A. Murphy
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