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When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

Methodology · Statistics 2020-08-03 Johannes Krisam , Dorothea Weber , Richard F. Schlenk , Meinhard Kieser

One of the main goals of sequential, multiple assignment, randomized trials (SMART) is to find the most efficacious design embedded dynamic treatment regimes. The analysis method known as multiple comparisons with the best (MCB) allows…

Methodology · Statistics 2020-08-07 William J. Artman , Ashkan Ertefaie , Kevin G. Lynch , James R. McKay

As increasingly complex hypothesis-testing scenarios are considered in many scientific fields, analytic derivation of null distributions is often out of reach. To the rescue comes Monte Carlo testing, which may appear deceptively simple: as…

Methodology · Statistics 2015-04-13 Egil Ferkingstad , Lars Holden , Geir Kjetil Sandve

Copula mixed models for trivariate (or bivariate) meta-analysis of diagnostic test accuracy studies accounting (or not) for disease prevalence have been proposed in the biostatistics literature to synthesize information. However, many…

Methodology · Statistics 2018-07-12 Aristidis K. Nikoloulopoulos

We conducted a systematic comparison of statistical methods used for the analysis of time-to-event outcomes under various proportional and nonproportional hazard (NPH) scenarios. Our study used data from recently published oncology trials…

Applications · Statistics 2025-02-12 Xinyu Zhang , Erich J. Greene , Ondrej Blaha , Wei Wei

A Monte Carlo simulation was used to determine which assumptions for ordered categorical data, continuity vs. discrete categories, most frequently identifies the underlying factor structure when a response variable has five ordered…

Applications · Statistics 2020-09-17 R. Noah Padgett , Rebecca J. Tipton

In microbiome and genomic studies, the regression of compositional data has been a crucial tool for identifying microbial taxa or genes that are associated with clinical phenotypes. To account for the variation in sequencing depth, the…

Methodology · Statistics 2021-03-11 Pixu Shi , Yuchen Zhou , Anru R. Zhang

In observational studies of treatment effects, matched samples are created so treated and control groups are similar in terms of observable covariates. Traditionally such matched samples consist of matched pairs. If a pair match fails to…

Methodology · Statistics 2014-10-22 Luke Keele , Sam Pimentel , Frank Yoon

Recently, a method [7] was proposed to generate contrastive explanations for differentiable models such as deep neural networks, where one has complete access to the model. In this work, we propose a method, Model Agnostic Contrastive…

Machine Learning · Computer Science 2019-06-04 Amit Dhurandhar , Tejaswini Pedapati , Avinash Balakrishnan , Pin-Yu Chen , Karthikeyan Shanmugam , Ruchir Puri

Method comparison studies are essential for development in medical and clinical fields. These studies often compare a cheaper, faster, or less invasive measuring method with a widely used one to see if they have sufficient agreement for…

Methodology · Statistics 2019-06-27 Wei Wang , Nan Lin , Jordan D. Oberhaus , Michael S. Avidan

A resurgence of interest in multiple hypothesis testing has occurred in the last decade. Motivated by studies in genomics, microarrays, DNA sequencing, drug screening, clinical trials, bioassays, education and psychology, statisticians have…

Statistics Theory · Mathematics 2007-06-13 Arthur Cohen , Harold B. Sackrowitz

In this paper, we provide the first investigation into adaptive combinatorial experimental design, focusing on the trade-off between regret minimization and statistical power in combinatorial multi-armed bandits (CMAB). While minimizing…

Machine Learning · Computer Science 2026-03-02 Hongrui Xie , Junyu Cao , Kan Xu

Background: Multi-collimator proton minibeam radiation therapy (MC-pMBRT) has recently emerged as a versatile technique for dose shaping, enabling peak-valley dose patterns in organs-at-risk (OAR) while maintaining a uniform dose…

Medical Physics · Physics 2025-02-25 Nimita Shinde , Weijie Zhang , Yuting Lin , Hao Gao

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu

Successful pharmaceutical drug development requires finding correct doses that provide an optimum balance between efficacy and toxicity. Competing responses to dose such as efficacy and toxicity often will increase with dose, and it is…

Applications · Statistics 2024-01-26 A. Lawrence Gould

Count endpoints are common in clinical trials, particularly for recurrent events such as hypoglycemia. When interest centers on comparing overall event rates between treatment groups, negative binomial (NB) regression is widely used because…

Methodology · Statistics 2026-01-27 Jiren Sun , Linda Amoafo , Yongming Qu

The comparison of proportions is considered in the asymptotic generalized linear model with the odds ratio as effect size. When several doses are compared with a control assuming an order restriction, a Williams-type trend test can be used.…

Applications · Statistics 2020-11-30 Ludwig A. Hothorn

Conducting a randomization test is a common method for testing causal null hypotheses in randomized experiments. The popularity of randomization tests is largely because their statistical validity only depends on the randomization design,…

Methodology · Statistics 2025-01-15 Siyu Heng , Pamela A. Shaw

We provide an approach to exploratory data analysis in matched observational studies with a single intervention and multiple endpoints. In such settings, the researcher would like to explore evidence for actual treatment effects among these…

Methodology · Statistics 2025-12-10 Mengqi Lin , Colin Fogarty

Joinpoint regression is used to determine the number of segments needed to adequately explain the relationship between two variables. This methodology can be widely applied to real problems, but we focus on epidemiological data, the main…

Applications · Statistics 2011-12-08 Miguel A. Martinez-Beneito , Gonzalo García-Donato , Diego Salmerón
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