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Estimating the effect of a change in a particular risk factor and a chronic disease requires information on the risk factor from two time points; the enrolment and the first follow-up. When using observational data to study the effect of…

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…

Causal inference is widely used in various fields, such as biology, psychology and economics, etc. In observational studies, we need to balance the covariates before estimating causal effect. This study extends the one-dimensional entropy…

统计方法学 · 统计学 2022-05-19 Juan Chen , Yingchun Zhou

In clinical settings, we often face the challenge of building prediction models based on small observational data sets. For example, such a data set might be from a medical center in a multi-center study. Differences between centers might…

Preferably in two- or three-arm randomized clinical trials, a few (2,3) correlated multiple primary endpoints are considered. In addition to the closed testing principle based on different global tests, two max(maxT) tests are compared with…

应用统计 · 统计学 2021-03-16 Ludwig A. Hothorn , Siegfried Kropf

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data…

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

统计方法学 · 统计学 2026-03-09 Samuel I. Watson , James Martin

Often both Aggregate Data (AD) studies and Individual Patient Data (IPD) studies are available for specific treatments. Combining these two sources of data could improve the overall meta-analytic estimates of treatment effects. Moreover,…

统计方法学 · 统计学 2021-11-15 Neha Agarwala , Junyong Park , Anindya Roy

Functional principal component analysis has been shown to be invaluable for revealing variation modes of longitudinal outcomes, which serves as important building blocks for forecasting and model building. Decades of research have advanced…

统计方法学 · 统计学 2024-10-07 Peijun Sang , Dehan Kong , Shu Yang

For a trial with primary endpoint overall survival for a molecule with curative potential, statistical methods that rely on the proportional hazards assumption may underestimate the power and the time to final analysis. We show how a cure…

应用统计 · 统计学 2023-04-17 Kaspar Rufibach , Dominik Heinzmann , Annabelle Monnet

We describe how the target trial framework can be used to plan and report analyses that attempt to answer causal questions by combining information from multiple, diverse sources. Such analyses may involve comparisons of treatments…

统计方法学 · 统计学 2026-05-26 Lawson Ung , Miguel A. Hernán , Issa J. Dahabreh

The measurement of the efficiency of an event selection is always an important part of the analysis of experimental data. The statistical techniques which are needed to determine the efficiency and its uncertainty are reviewed. Frequentist…

数据分析、统计与概率 · 物理学 2012-08-28 Diego Casadei

High dimensional case control studies are ubiquitous in the biological sciences, particularly genomics. To maximise power while constraining cost and to minimise type-1 error rates, researchers typically seek to replicate findings in a…

统计方法学 · 统计学 2017-07-11 James Liley

Experiments deliver credible treatment-effect estimates but, because they are costly, are often restricted to specific sites, small populations, or particular mechanisms. A common practice across several fields is therefore to combine…

计量经济学 · 经济学 2025-12-30 Aristotelis Epanomeritakis , Davide Viviano

Composite endpoints are frequently used as primary or secondary analyses in cardiovascular clinical trials to increase clinical relevance and statistical efficiency. Alternatively, the Win Ratio (WR) and other Win Statistics (WS) analyses…

统计方法学 · 统计学 2025-12-23 Yunhan Mou , Scott Hummel , Yuan Huang

In many phase II trials in solid tumours, patients are assessed using endpoints based on the Response Evaluation Criteria in Solid Tumours (RECIST) scale. Often, analyses are based on the response rate. This is the proportion of patients…

统计方法学 · 统计学 2017-03-02 Chien-Ju Lin , James Wason

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

统计计算 · 统计学 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

In this paper, we propose a general method for testing composite hypotheses. Our idea is to use confidence limits to define stopping and decision rules. The requirements of operating characteristic function can be satisfied by adjusting the…

统计理论 · 数学 2012-02-10 Xinjia Chen

In this paper, a Bayesian approach is developed for simultaneously comparing multiple experimental treatments with a common control treatment in an exploratory clinical trial. The sample size is set to ensure that, at the end of the study,…

统计理论 · 数学 2019-11-14 John Whitehead , Faye Cleary , Amanda Turner

This paper develops a unified and computationally efficient method for change-point estimation along the time dimension in a non-stationary spatio-temporal process. By modeling a non-stationary spatio-temporal process as a piecewise…

统计方法学 · 统计学 2023-10-09 Zifeng Zhao , Ting Fung Ma , Wai Leong Ng , Chun Yip Yau