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Researchers often turn to block randomization to increase the precision of their inference or due to practical considerations, such as in multisite trials. However, if the number of treatments under consideration is large it might not be…

Methodology · Statistics 2025-08-26 Taehyeon Koo , Nicole E. Pashley

We examine study designs for extending (generalizing or transporting) causal inferences from a randomized trial to a target population. Specifically, we consider nested trial designs, where randomized individuals are nested within a sample…

Biomarker is a critically important tool in modern clinical diagnosis, prognosis, and classification/prediction. However, there are fiscal and analytical barriers to biomarker research. Selective Genotyping is an approach to increasing…

Methodology · Statistics 2022-08-02 A. Adam Ding , Natalie DelRocco , Samuel Wu

The standard paradigm for confirmatory clinical trials is to compare experimental treatments with a control, for example the standard of care or a placebo. However, it is not always the case that a suitable control exists. Efficient…

Methodology · Statistics 2024-10-29 Thomas Burnett , Thomas Jaki

In a group sequential clinical trial, accumulated data are analysed at numerous time-points in order to allow early decisions about a hypothesis of interest. These designs have historically been recommended for their ethical, administrative…

Computation · Statistics 2017-11-29 Michael Grayling , James Wason , Adrian Mander

Various methodological options exist on evaluating differences in both subgroups and the overall population. Most desirable is the simultaneous study of multiple endpoints in several populations. We investigate a newer method using multiple…

Applications · Statistics 2020-07-23 Charlotte Vogel , Frank Schaarschmidt , Christian Ritz , Franz Koenig , Ludwig A. Hothorn

Multi-arm multi-stage trial designs can bring notable gains in efficiency to the drug development process. However, for normally distributed endpoints, the determination of a design typically depends on the assumption that the patient…

Methodology · Statistics 2017-10-11 Michael Grayling , James Wason , Adrian Mander

Test procedures for multiple hypotheses in a group sequential clinical trial that control the family-wise error rate are considered. Several graphical group sequential tests suggested in the literature, which are special cases of…

Methodology · Statistics 2026-05-13 Liane Kluge , Werner Brannath

Machine learning approaches have been effective in predicting adverse outcomes in different clinical settings. These models are often developed and evaluated on datasets with heterogeneous patient populations. However, good predictive…

Machine Learning · Computer Science 2018-06-11 Harini Suresh , Jen J. Gong , John Guttag

In randomized trials, appropriately adjusting for baseline variables and short-term outcomes can lead to increased precision and reduced sample size. We examine the impact of such adjustment in group sequential designs, i.e., designs with…

Methodology · Statistics 2019-10-28 Tianchen Qian , Michael Rosenblum , Huitong Qiu

In a split conformal framework with $K$ classes, a calibration sample of $n$ labeled examples is observed for inference on the label of a new unlabeled example. We explore the setting where a `batch' of $m$ independent such unlabeled…

Methodology · Statistics 2025-03-19 Ulysse Gazin , Ruth Heller , Etienne Roquain , Aldo Solari

Count data and recurrent events in clinical trials, such as the number of lesions in magnetic resonance imaging in multiple sclerosis, the number of relapses in multiple sclerosis, the number of hospitalizations in heart failure, and the…

Applications · Statistics 2019-03-07 Tobias Mütze , Ekkehard Glimm , Heinz Schmidli , Tim Friede

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

In covariate-adaptive or response-adaptive randomization, the treatment assignment and outcome can be correlated. Under this situation, re-randomization tests are a straightforward and attractive method to provide valid statistical…

Methodology · Statistics 2023-03-14 Yilong Zhang , Yujie Zhao , Yiwen Luo

We present a new optimization method for the group selection problem in linear regression. In this problem, predictors are assumed to have a natural group structure and the goal is to select a small set of groups that best fits the…

Methodology · Statistics 2024-04-23 Anant Mathur , Sarat Moka , Benoit Liquet , Zdravko Botev

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

In cancer research, clustering techniques are widely used for exploratory analyses and dimensionality reduction, playing a critical role in the identification of novel cancer subtypes, often with direct implications for patient management.…

In confirmatory clinical trials, it has been proposed to use a simple iterative graphical approach to construct and perform intersection hypotheses tests with a weighted Bonferroni-type procedure to control type I errors in the strong…

Methodology · Statistics 2022-08-03 Tianyu Zhan , Alan H Hartford , Jian Kang , Walter W Offen

Multi-label learning predicts a subset of labels from a given label set for an unseen instance while considering label correlations. A known challenge with multi-label classification is the long-tailed distribution of labels. Many studies…

Machine Learning · Computer Science 2021-12-06 Vithya Yogarajan , Bernhard Pfahringer , Tony Smith , Jacob Montiel

The Bayes factor, the data-based updating factor from prior to posterior odds, is a principled measure of relative evidence for two competing hypotheses. It is naturally suited to sequential data analysis in settings such as clinical trials…

Methodology · Statistics 2026-01-07 Samuel Pawel , Leonhard Held