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Bayesian sample size calculations in clinical trials usually rely on complex Monte Carlo simulations in practice. Obtaining bounds on Bayesian notions of the false-positive rate and power often lack closed-form or approximate numerical…

Methodology · Statistics 2026-03-03 Riko Kelter

In a given randomized experiment, individuals are often volunteers and can differ in important ways from a population of interest. It is thus of interest to focus on the sample at hand. This paper focuses on inference about the sample local…

Methodology · Statistics 2024-09-23 Zhen Zhong , Per Johansson , Junni L. Zhang

Adaptive designs for multi-armed clinical trials have become increasingly popular recently in many areas of medical research because of their potential to shorten development times and to increase patient response. However, developing…

Applications · Statistics 2017-03-16 Adam Smith , Sofia S. Villar

Basket trials can efficiently evaluate a single treatment across multiple diseases with a common shared target. Prior methods for randomized basket trials required baskets to have the same sample and effect sizes. To that end, we developed…

Methodology · Statistics 2024-11-22 Sahil S. Patel , Desmond Zeya Chen , David Castle , Clement Ma

The sample size of a clinical trial relies on information about nuisance parameters such as the outcome variance. When no or only limited information is available, it has been proposed to include an internal pilot study in the design of the…

Applications · Statistics 2017-07-18 Tobias Mütze , Tim Friede

The class of autoregressive (AR) processes is extensively used to model temporal dependence in observed time series. Such models are easily available and routinely fitted using freely available statistical software like R. A potential…

Methodology · Statistics 2020-10-13 Sigrunn H. Sørbye , Pedro G. Nicolau , Håvard Rue

This paper considers real-time control and learning problems for finite-dimensional linear systems under binary-valued and randomly disturbed output observations. This has long been regarded as an open problem because the exact values of…

Systems and Control · Electrical Eng. & Systems 2024-11-12 Lantian Zhang , Lei Guo

Response-adaptive randomization allows the probabilities of allocating patients to treatments in a clinical trial to change based on the previously observed response data, in order to achieve different experimental goals. One concern over…

Methodology · Statistics 2022-04-13 Ekkehard Glimm , David Robertson

The goals in clinical and cohort studies often include evaluation of the association of a time-dependent binary treatment or exposure with a survival outcome. Recently, several impactful studies targeted the association between…

Applications · Statistics 2019-01-24 Daniel Nevo , Tsuyoshi Hamada , Shuji Ogino , Molin Wang

Pragmatic trials increasingly define outcomes using real-world data such as electronic health records, where assessments are collected during routine care rather than at fixed timepoints. Consequently, these uncontrolled assessments may be…

Interval designs are a class of phase I trial designs for which the decision of dose assignment is determined by comparing the observed toxicity rate at the current dose with a prespecified (toxicity tolerance) interval. If the observed…

Methodology · Statistics 2013-09-20 Suyu Liu , Ying Yuan

Long pulse durations necessary in selective inversion recovery (SIR) experiments along with radiation damping (RD) introduce difficulties in quantitative nuclear magnetic resonance measurements, such as those that allow for the…

Medical Physics · Physics 2014-12-01 John S. Ginn , Heather M. Whitney

Large observational datasets, including those derived from electronic health records, are a valuable resource for medical research but are often affected by missingness, measurement error, and misclassification. Two-phase sampling with…

Methodology · Statistics 2026-03-23 Jasper B. Yang , Bryan E. Shepherd , Thomas Lumley , Pamela A. Shaw

We propose a multi-metric flexible Bayesian framework to support efficient interim decision-making in multi-arm multi-stage phase II clinical trials. Multi-arm multi-stage phase II studies increase the efficiency of drug development, but…

Applications · Statistics 2023-12-15 Suzanne M. Dufault , Angela M. Crook , Katie Rolfe , Patrick P. J. Phillips

A sequential multiple assignment randomized trial (SMART) facilitates comparison of multiple adaptive treatment strategies (ATSs) simultaneously. Previous studies have established a framework to test the homogeneity of multiple ATSs by a…

Methodology · Statistics 2022-11-04 Liwen Wu , Junyao Wang , Abdus S. Wahed

Broken adaptive ridge (BAR) is a computationally scalable surrogate to $L_0$-penalized regression, which involves iteratively performing reweighted $L_2$ penalized regressions and enjoys some appealing properties of both $L_0$ and $L_2$…

Methodology · Statistics 2020-11-30 Zhihua Sun , Yi Liu , Kani Chen , Gang Li

Mid-study design modifications are becoming increasingly accepted in confirmatory clinical trials, so long as appropriate methods are applied such that error rates are controlled. It is therefore unfortunate that the important case of…

Applications · Statistics 2014-05-08 Dominic Magirr , Thomas Jaki , Franz Koenig , Martin Posch

In epidemiological or demographic studies, with variable age at onset, a typical quantity of interest is the incidence of a disease (for example the cancer incidence). In these studies, the individuals are usually highly heterogeneous in…

Statistics Theory · Mathematics 2025-05-20 Vivien Goepp , Jean-Christophe Thalabard , Grégory Nuel , Olivier Bouaziz

Multi-armed bandit (MAB) algorithms are efficient approaches to reduce the opportunity cost of online experimentation and are used by companies to find the best product from periodically refreshed product catalogs. However, these algorithms…

Machine Learning · Computer Science 2024-12-19 Mohsen Bayati , Junyu Cao , Wanning Chen

Multi-armed bandits are widely used for sequential experimentation in clinical trials, recommendation systems, and online platforms. While regret minimization and valid inference from adaptively collected data have each been studied…

Methodology · Statistics 2026-04-28 Yu-Shiou Willy Lin , Dae Woong Ham , Iavor Bojinov
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