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Conditional (European Medicines Agency) or accelerated (U.S. Food and Drug Administration) approval of drugs allow earlier access to promising new treatments that address unmet medical needs. Certain post-marketing requirements must…

统计方法学 · 统计学 2022-10-19 Manja Deforth , Charlotte Micheloud , Kit C Roes , Leonhard Held

The two-trials rule for drug approval requires "at least two adequate and well-controlled studies, each convincing on its own, to establish effectiveness". This is usually implemented by requiring two significant pivotal trials and is the…

统计方法学 · 统计学 2023-11-10 Leonhard Held

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

统计方法学 · 统计学 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

Pearson's chi-square tests are among the most commonly applied statistical tools across a wide range of scientific disciplines, including medicine, engineering, biology, sociology, marketing and business. However, its usage in some areas is…

统计方法学 · 统计学 2025-05-13 Vladimir Gurvich , Mariya Naumova

Recently, the U.S. Food and Drug Administration (FDA) released draft guidance \citep{FDA2026} signaling a paradigm shift that facilitates the use of Bayesian methodology as the primary analysis and decision framework for drug approval. The…

统计方法学 · 统计学 2026-03-23 Peng Yang , Li Wang , Ying Yuan

The question of testing for equality in distribution between two linear models, each consisting of sums of distinct discrete independent random variables with unequal numbers of observations, has emerged from the biological research. In…

统计理论 · 数学 2020-09-01 Giulio Prevedello , Ken R. Duffy

Comparisons of different treatments or production processes are the goals of a significant fraction of applied research. Unsurprisingly, two-sample problems play a main role in Statistics through natural questions such as `Is the the new…

统计方法学 · 统计学 2017-09-05 P. C. Álvarez-Esteban , E. del Barrio , J. A. Cuesta-Albertos , C. Matrán

A bioequivalence study is a type of clinical trial designed to compare the biological equivalence of two different formulations of a drug. Such studies are typically conducted in controlled clinical settings with human subjects, who are…

统计方法学 · 统计学 2023-06-13 Kexuan Li , Susie Sinks , Peng Sun , Lingli Yang

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

Testing the equality of the covariance matrices of two high-dimensional samples is a fundamental inference problem in statistics. Several tests have been proposed but they are either too liberal or too conservative when the required…

统计理论 · 数学 2023-01-04 Jin-Ting Zhang , Jingyi Wang , Tianming Zhu

Statistical depth, which measures the center-outward rank of a given sample with respect to its underlying distribution, has become a popular and powerful tool in nonparametric inference. In this paper, we investigate the use of statistical…

统计方法学 · 统计学 2025-11-25 Chifeng Shen , Yuejiao Fu , Michael Chen , Xiaoping Shi

A non parametric method based on the empirical likelihood is proposed for detecting the change in the coefficients of high-dimensional linear model where the number of model variables may increase as the sample size increases. This amounts…

统计理论 · 数学 2015-06-22 Gabriela Ciuperca , Zahraa Salloum

This short note considers the problem of testing the null hypothesis that the mean values of two multivariate normal variables are proportional. We show that the usual likelihood ratio $\chi^2$-test is valid non-asymptotically. Our proof…

统计理论 · 数学 2021-03-10 Etaash Katiyar , Qingyuan Zhao

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…

统计方法学 · 统计学 2024-10-29 Thomas Burnett , Thomas Jaki

As it is well known, the standard deviation of a weighted average depends only on the individual standard deviations, but not on the dispersion of the values around the mean. This property leads sometimes to the embarrassing situation in…

数据分析、统计与概率 · 物理学 2020-01-22 Giulio D'Agostini

Testing hypothesis of independence between two random elements on a joint alphabet is a fundamental exercise in statistics. Pearson's chi-squared test is an effective test for such a situation when the contingency table is relatively small.…

统计理论 · 数学 2025-03-19 Jialin Zhang , Zhiyi Zhang

Based on the test for equality of quantiles originally introduced by Kosorok (1999), we propose new power formulas for the comparison of one quantile between two treatment groups, as well as for the comparison of a collection of quantiles.…

统计方法学 · 统计学 2026-03-10 Beatriz Farah , Olivier Bouaziz , Aurélien Latouche

The era of big data is coming, and evidence-based medicine is attracting increasing attention to improve decision making in medical practice via integrating evidence from well designed and conducted clinical research. Meta-analysis is a…

统计方法学 · 统计学 2016-10-05 Dehui Luo , Xiang Wan , Jiming Liu , Tiejun Tong

Large-scale simultaneous hypothesis testing appears in many areas such as microarray studies, genome-wide association studies, brain imaging, disease mapping and astronomical surveys. A well-known inference method is to control the false…

统计方法学 · 统计学 2025-07-22 Xiaoqing Niu , Pengfei Li , Yuejiao Fu

Considerable interest has recently been focused on studying multiple phenotypes simultaneously in both epidemiological and genomic studies, either to capture the multidimensionality of complex disorders or to understand shared etiology of…

统计方法学 · 统计学 2015-11-26 Denis Agniel , Katherine P. Liao , Tianxi Cai
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