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Pragmatic trials evaluating health care interventions often adopt cluster randomization due to scientific or logistical considerations. Previous reviews have shown that co-primary endpoints are common in pragmatic trials but infrequently…

统计方法学 · 统计学 2022-05-03 Siyun Yang , Mirjam Moerbeek , Monica Taljaard , Fan Li

When developing a clinical prediction model, the sample size of the development dataset is a key consideration. Small sample sizes lead to greater concerns of overfitting, instability, poor performance and lack of fairness. Previous…

Meta-analysis aims to combine effect measures from several studies. For continuous outcomes, the most popular effect measures use simple or standardized differences in sample means. However, a number of applications focus on the absolute…

统计方法学 · 统计学 2023-10-03 Elena Kulinskaya , David C. Hoaglin

The increasing interest in subpopulation analysis has led to the development of various new trial designs and analysis methods in the fields of personalized medicine and targeted therapies. In this paper, subpopulations are defined in terms…

统计方法学 · 统计学 2020-12-01 Roland Gerard Gera , Tim Friede

Meta-analyses frequently include trials that report multiple effect sizes based on a common set of study participants. These effect sizes will generally be correlated. Cluster-robust variance-covariance estimators are a fruitful approach…

统计方法学 · 统计学 2022-03-07 Thilo Welz , Wolfgang Viechtbauer , Markus Pauly

Sample size calculations play a central role in study design because sample size affects study interpretability, costs, hospital resources, and staff time. For most veterinary orthopaedic risk-factor studies, either the sample size…

应用统计 · 统计学 2023-10-10 Richard Evans , Antonio Pozzi

We describe how to calculate standard errors for A/B tests that include clustered data, ratio metrics, and/or covariate adjustment. We may do this for power analysis/sample size calculations prior to running an experiment using historical…

统计方法学 · 统计学 2024-06-12 Tim Hesterberg , Ben Knight

Breast cancer detection is still an open research field, despite a tremendous effort devoted to work in this area. Effect size is a statistical concept that measures the strength of the relationship between two variables on a numeric scale.…

机器学习 · 统计学 2024-11-12 Nicolas Masino , Antonio Quintero-Rincon

The problem tackled in this paper is the determination of sample size for a given level and power in the context of a simple linear regression model. At a technical level, the simple linear regression model is a five-parameter model. It is…

统计方法学 · 统计学 2019-07-25 Tianyuan Guan , M. Khorshed Alam , M. Bhaskara Rao

A common goal in comparative effectiveness research is to estimate treatment effects on pre-specified subpopulations of patients. Though widely used in medical research, causal inference methods for such subgroup analysis remain…

Accurate power and sample size estimation are crucial to the design and analysis of genetic association studies. When analyzing a binary trait via logistic regression, important covariates such as age and sex are typically included in the…

统计方法学 · 统计学 2022-10-05 Ziang Zhang , Lei Sun

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

统计方法学 · 统计学 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

For randomized controlled trials to be conclusive, it is important to set the target sample size accurately at the design stage. Comparing two normal populations, the sample size calculation requires specification of the variance other than…

统计方法学 · 统计学 2026-02-04 Hirotada Maeda , Satoshi Hattori , Tim Friede

Two-stage randomized experiments are becoming an increasingly popular experimental design for causal inference when the outcome of one unit may be affected by the treatment assignments of other units in the same cluster. In this paper, we…

统计方法学 · 统计学 2022-10-21 Zhichao Jiang , Kosuke Imai , Anup Malani

Causal inference analyses often use existing observational data, which in many cases has some clustering of individuals. In this paper we discuss propensity score weighting methods in a multilevel setting where within clusters individuals…

应用统计 · 统计学 2020-12-24 Youjin Lee , Trang Q. Nguyen , Elizabeth A. Stuart

Good (Frequentist) statistical practice requires that statistical tests be performed in order to determine if the phenomenon being observed could plausibly occur by chance if the null hypothesis is false. Good practice also requires that a…

计算机与社会 · 计算机科学 2024-08-06 Michael Guerzhoy

Cluster or group randomized trials (CRTs) are increasingly used for both behavioral and system-level interventions, where entire clusters are randomly assigned to a study condition or intervention. Apart from the assigned cluster-level…

统计方法学 · 统计学 2024-11-19 Shubhadeep Chakraborty , Bo Wang , Ram Tiwari , Samiran Ghosh

We address estimation of intervention effects in experimental designs in which (a) interventions are assigned at the cluster level; (b) clusters are selected to form pairs, matched on observed characteristics; and (c) intervention is…

统计方法学 · 统计学 2014-11-24 Zhenke Wu , Constantine E. Frangakis , Thomas A. Louis , Daniel O. Scharfstein

Clustering is part of unsupervised analysis methods that consist in grouping samples into homogeneous and separate subgroups of observations also called clusters. To interpret the clusters, statistical hypothesis testing is often used to…

统计方法学 · 统计学 2022-10-25 Benjamin Hivert , Denis Agniel , Rodolphe Thiébaut , Boris P Hejblum

The selection of the assumed effect size (AES) critically determines the duration of an experiment, and hence its accuracy and efficiency. Traditionally, experimenters determine AES based on domain knowledge. However, this method becomes…

机器学习 · 计算机科学 2025-04-15 Yu Liu , Runzhe Wan , James McQueen , Doug Hains , Jinxiang Gu , Rui Song