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In learning-phase clinical trials in drug development, adaptive designs can be efficient and highly informative when used appropriately. In this article, we extend the multiple comparison procedures with modeling techniques (MCP-Mod)…

Methodology · Statistics 2021-08-02 Shiyang Ma , Michael P. McDermott

Over time, clinical trials have increasingly incorporated complex design and analysis elements such as interim analyses, adaptations, multiple endpoints, and sophisticated multiplicity schemes for multiple endpoints and/or treatment arms…

We study nonasymptotic (finite-sample) confidence intervals for treatment effects in randomized experiments. In the existing literature, the effective sample sizes of nonasymptotic confidence intervals tend to be looser than the…

Bayesian adaptive designs have gained popularity in all phases of clinical trials with numerous new developments in the past few decades. During the COVID-19 pandemic, the need to establish evidence for the effectiveness of vaccines,…

Methodology · Statistics 2022-03-08 Shirin Golchi

Adaptive Conformal Inference (ACI) provides finite-sample coverage guarantees, enhancing the prediction reliability under non-exchangeability. This study demonstrates that these desirable properties of ACI do not require the use of…

Machine Learning · Statistics 2025-07-02 Johan Hallberg Szabadváry , Tuwe Löfström

We consider inference in regression discontinuity designs when the running variable only takes a moderate number of distinct values. In particular, we study the common practice of using confidence intervals (CIs) based on standard errors…

Applications · Statistics 2018-07-26 Michal Kolesár , Christoph Rothe

This paper studies the construction of adaptive confidence intervals under Huber's contamination model when the contamination proportion is unknown. For the robust confidence interval of a Gaussian mean, we show that the optimal length of…

Statistics Theory · Mathematics 2025-06-05 Yuetian Luo , Chao Gao

Response-adaptive clinical trial designs allow targeting a given objective by skewing the allocation of participants to treatments based on observed outcomes. Response-adaptive designs face greater regulatory scrutiny due to potential type…

Methodology · Statistics 2025-03-19 Stef Baas , Peter Jacko , Sofía S. Villar

Minimizing the number of patients exposed to potentially harmful drugs in early onco logical trials is a major concern during planning. Adaptive designs account for the inherent uncertainty about the true effect size by determining the…

Applications · Statistics 2016-05-03 Kevin Kunzmann , Meinhard Kieser

We consider the problem of adaptive inference on a regression function at a point under a multivariate nonparametric regression setting. The regression function belongs to a H\"older class and is assumed to be monotone with respect to some…

Statistics Theory · Mathematics 2020-12-01 Koohyun Kwon , Soonwoo Kwon

When assessing the quality of prediction models in machine learning, confidence intervals (CIs) for the generalization error, which measures predictive performance, are a crucial tool. Luckily, there exist many methods for computing such…

Corrected confidence intervals are developed for the mean of the second component of a bivariate normal process when the first component is being monitored sequentially. This is accomplished by constructing a first approximation to a…

Statistics Theory · Mathematics 2007-06-13 R. C. Weng , D. S. Coad

The analysis of multiple time-to-event outcomes in a randomised controlled clinical trial can be accomplished with exisiting methods. However, depending on the characteristics of the disease under investigation and the circumstances in…

Methodology · Statistics 2026-03-02 Moritz Fabian Danzer , Andreas Faldum , Thorsten Simon , Barbara Hero , Rene Schmidt

Introduction: there is an ongoing debate about directional inference of two-sided hypothesis tests for which some authors argue that rejecting $\theta = \theta_0$ does not allow to conclude that $\theta > \theta_0$ or $\theta < \theta_0$…

Other Statistics · Statistics 2021-03-24 André Gillibert , Jacques Bénichou , Bruno Falissard

Adaptive confidence intervals for regression functions are constructed under shape constraints of monotonicity and convexity. A natural benchmark is established for the minimum expected length of confidence intervals at a given function in…

Statistics Theory · Mathematics 2013-05-27 T. Tony Cai , Mark G. Low , Yin Xia

Adapting the final sample size of a trial to the evidence accruing during the trial is a natural way to address planning uncertainty. Designs with adaptive sample size need to account for their optional stopping to guarantee strict type-I…

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

This paper studies the power properties of confidence intervals (CIs) for a partially-identified parameter of interest with an interval identified set. We assume the researcher has bounds estimators to construct the CIs proposed by Stoye…

Econometrics · Economics 2024-10-23 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

Proximal causal inference (PCI) is a recently proposed framework to identify and estimate the causal effect of an exposure on an outcome in the presence of hidden confounders, using observed proxies. Specifically, PCI relies on two types of…

Methodology · Statistics 2025-07-29 Prabrisha Rakshit , Xu Shi , Eric Tchetgen Tchetgen

The average treatment effect (ATE) is widely used to evaluate the effectiveness of drugs and other medical interventions. In safety-critical applications like medicine, reliable inferences about the ATE typically require valid uncertainty…

Machine Learning · Computer Science 2025-10-13 Maresa Schröder , Justin Hartenstein , Stefan Feuerriegel