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This paper explores an approach to Bayesian sample size determination in clinical trials. The approach falls into the category of what is often called "proper Bayesian", in that it does not mix frequentist concepts with Bayesian ones. A…

Methodology · Statistics 2012-04-23 Robb J. Muirhead , Adina I. Soaita

Quantitative evidence synthesis methods aim to combine data from multiple medical trials to infer relative effects of different interventions. A challenge arises when trials report continuous outcomes on different measurement scales. To…

Methodology · Statistics 2024-02-29 Annabel L Davies , A E Ades , Julian PT Higgins

Random effects meta-analysis is a widely applied methodology to synthetize research findings of studies in a specific scientific question. Besides estimating the mean effect, an important aim of the meta-analysis is to summarize the…

Applications · Statistics 2026-01-28 Peter Matrai , Tamas Koi , Zoltan Sipos , Nelli Farkas

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

Methodology · Statistics 2022-11-10 Wenqi Shi , Xi Lin

Aims: To propose a general sample size framework for developing or updating a clinical prediction model using any statistical or machine learning method, based on drawing samples from anticipated posterior distributions and targeting…

We propose a general semi-supervised inference framework focused on the estimation of the population mean. As usual in semi-supervised settings, there exists an unlabeled sample of covariate vectors and a labeled sample consisting of…

Methodology · Statistics 2018-08-15 Anru Zhang , Lawrence D. Brown , T. Tony Cai

We consider stochastic optimization problems which use observed data to estimate essential characteristics of the random quantities involved. Sample average approximation (SAA) or empirical (plug-in) estimation are very popular ways to use…

Statistics Theory · Mathematics 2021-03-16 Darinka Dentcheva , Yang Lin

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,…

Statistics Theory · Mathematics 2019-11-14 John Whitehead , Faye Cleary , Amanda Turner

Multivariate meta-analysis can be adapted to a wide range of situations for multiple outcomes and multiple treatment groups when combining studies together. The within-study correlation between effect sizes is often assumed known in…

Methodology · Statistics 2020-03-12 Xiaohuan Xue

Traditional covariate selection methods for causal inference focus on achieving unbiasedness and asymptotic efficiency. In many practical scenarios, researchers must estimate causal effects from observational data with limited sample sizes…

Statistics Theory · Mathematics 2025-06-17 Nadja Rutsch , Sara Magliacane , Stéphanie van der Pas

It is well-known that trimmed sample means are robust against heavy tails and data contamination. This paper analyzes the performance of trimmed means and related methods in two novel contexts. The first one consists of estimating…

Statistics Theory · Mathematics 2025-12-03 Roberto I. Oliveira , Lucas Resende

We consider a Bayesian framework for estimating the sample size of a clinical trial. The new approach, called BESS, is built upon three pillars: Sample size of the trial, Evidence from the observed data, and Confidence of the final decision…

Methodology · Statistics 2026-01-21 Dehua Bi , Yuan Ji

Meta-analysis, because of both logistical convenience and statistical efficiency, is widely popular for synthesizing information on common parameters of interest across multiple studies. We propose developing a generalized meta-analysis…

Methodology · Statistics 2018-11-27 Prosenjit Kundu , Runlong Tang , Nilanjan Chatterjee

We present methods for causally interpretable meta-analyses that combine information from multiple randomized trials to estimate potential (counterfactual) outcome means and average treatment effects in a target population. We consider…

The Birnbaum-Saunders distribution has been widely applied in several areas of science and although several methodologies related to this distribution have been proposed, the problem of determining the optimal sample size for estimating its…

Methodology · Statistics 2020-07-27 Eliardo G. Costa , Manoel Santos-Neto

Subsampling is a computationally efficient and scalable method to draw inference in large data settings based on a subset of the data rather than needing to consider the whole dataset. When employing subsampling techniques, a crucial…

Methodology · Statistics 2025-10-08 Amalan Mahendran , Helen Thompson , James M. McGree

Two-phase sampling designs are frequently employed in epidemiological studies and large-scale health surveys. In such designs, certain variables are exclusively collected within a second-phase random subsample of the initial first-phase…

Methodology · Statistics 2024-03-25 Lingxiao Wang

Imbalanced data is a frequently encountered problem in machine learning. Despite a vast amount of literature on sampling techniques for imbalanced data, there is a limited number of studies that address the issue of the optimal sampling…

Machine Learning · Computer Science 2022-07-12 Firuz Kamalov , Amir F. Atiya , Dina Elreedy

Sample overlap is a common issue in evidence synthesis in the field of medical research, particularly when integrating findings from observational studies utilizing existing databases such as registries. Due to the general inaccessibility…

Methodology · Statistics 2026-02-26 Zhentian Zhang , Tim Friede , Tim Mathes

The determination of the sample size required by a crossover trial typically depends on the specification of one or more variance components. Uncertainty about the value of these parameters at the design stage means that there is often a…

Methodology · Statistics 2018-03-28 Michael Grayling , Adrian Mander , James Wason