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It is increasingly common for therapies in oncology to be given in combination. In some cases, patients can benefit from the interaction between two drugs, although often at the risk of higher toxicity. A large number of designs to conduct…

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

The win ratio (WR) statistic is increasingly used to evaluate treatment effects based on prioritized composite endpoints, yet existing Bayesian adaptive designs are not directly applicable because the WR is a summary statistic derived from…

Methodology · Statistics 2026-02-20 Di Zhu , Yong Zang

Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

Methodology · Statistics 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

Multi-modal fusion approaches aim to integrate information from different data sources. Unlike natural datasets, such as in audio-visual applications, where samples consist of "paired" modalities, data in healthcare is often collected…

Image and Video Processing · Electrical Eng. & Systems 2023-03-03 Nasir Hayat , Krzysztof J. Geras , Farah E. Shamout

This paper deals exclusively with crossover designs for the purpose of comparing t test treatments with a control treatment when the number of periods is no larger than t+1. Among other results it specifies sufficient conditions for a…

Statistics Theory · Mathematics 2007-06-13 A. S. Hedayat , Min Yang

Major Depressive Disorder (MDD) is one of the most common causes of disability worldwide. Unfortunately, about one-third of patients do not benefit sufficiently from available treatments and not many new drugs have been developed in this…

A crossover trial is an efficient trial design when there is no carry-over effect. To reduce the impact of the biological carry-over effect, a washout period is often designed. However, the carry-over effect remains an outstanding concern…

Applications · Statistics 2024-03-06 Danni Shi , Ting Ye

PURPOSE Providing rapid answers and early acces to patients to innovative treatments without randomized clinical trial (RCT) is growing, with benefit estimated from single-arm trials. This has become common in oncology, impacting the…

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

Multi-armed bandit problems (MABPs) are a special type of optimal control problem well suited to model resource allocation under uncertainty in a wide variety of contexts. Since the first publication of the optimal solution of the classic…

Methodology · Statistics 2015-07-30 Sofía S. Villar , Jack Bowden , James Wason

This paper is motivated by evaluating the benefits of patients receiving mechanical circulatory support (MCS) devices in end-stage heart failure management inference, in which hypothesis testing for a treatment effect on the risk of…

Applications · Statistics 2026-01-06 Yiyuan Huang , Ling Zhou , Min Zhang , Peter X. K. Song

In this paper, the problem of error control of stepwise multiple testing procedures is considered. For two-sided hypotheses, control of both type 1 and type 3 (or directional) errors is required, and thus mixed directional familywise error…

Statistics Theory · Mathematics 2016-02-10 Wenge Guo , Joseph P. Romano

Allocating patients to treatment arms during a trial based on the observed responses accumulated prior to the decision point, and sequential adaptation of this allocation,, could minimize the expected number of failures or maximize total…

Methodology · Statistics 2022-09-01 Zhongying Xu , Andriy I. Bandos , Tianzhou Ma , Lu Tang , Victor B. Talisa , Chung-Chou H. Chang

A new model for controlled sensing for multihypothesis testing is proposed and studied in the sequential setting. This new model, termed {\em controlled Markovian observation} model, exhibits a more complicated memory structure in the…

Optimization and Control · Mathematics 2014-07-01 Sirin Nitinawarat , Venupogal V. Veeravalli

In many scenarios such as genome-wide association studies where dependences between variables commonly exist, it is often of interest to infer the interaction effects in the model. However, testing pairwise interactions among millions of…

Methodology · Statistics 2022-09-02 Jingyi Duan , Yang Ning , Xi Chen , Yong Chen

We consider a multi-hypothesis testing problem involving a K-armed bandit. Each arm's signal follows a distribution from a vector exponential family. The actual parameters of the arms are unknown to the decision maker. The decision maker…

Information Theory · Computer Science 2022-06-13 Gayathri R Prabhu , Srikrishna Bhashyam , Aditya Gopalan , Rajesh Sundaresan

Platform trials are randomized clinical trials that allow simultaneous comparison of multiple interventions, usually against a common control. Arms to test experimental interventions may enter and leave the platform over time. This implies…

Methodology · Statistics 2024-04-12 Marta Bofill Roig , Ekkehard Glimm , Tobias Mielke , Martin Posch

When an experimenter has the option of running an adaptive trial, is it admissible to ignore this option and run a non-adaptive trial instead? We provide a negative answer to this question in the best-arm identification problem, where the…

Machine Learning · Statistics 2025-06-06 Guido Imbens , Chao Qin , Stefan Wager

A central goal in designing clinical trials is to find the test that maximizes power (or equivalently minimizes required sample size) for finding a false null hypothesis subject to the constraint of type I error. When there is more than one…

Methodology · Statistics 2022-09-21 Ruth Heller , Abba Krieger , Saharon Rosset