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Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

Applications · Statistics 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu

While there exists a large amount of literature on the general challenges of and best practices for trustworthy online A/B testing, there are limited studies on sample size estimation, which plays a crucial role in trustworthy and efficient…

Methodology · Statistics 2023-08-21 Jing Zhou , Jiannan Lu , Anas Shallah

Scientific experiments study interventions that show evidence of an effect size that is meaningfully large, negligibly small, or inconclusively broad. Previously, we proposed contra-analysis as a decision-making process to help determine…

Methodology · Statistics 2023-03-17 Bruce A. Corliss , Yaotian Wang , Heman Shakeri , Philip E. Bourne

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers.…

Background: Pairwise and network meta-analyses using fixed effect and random effects models are commonly applied to synthesise evidence from randomised controlled trials. The models differ in their assumptions and the interpretation of the…

Methodology · Statistics 2017-08-04 Shijie Ren , Jeremy E. Oakley , John W. Stevens

Random-effects meta-analyses are widely used for evidence synthesis in medical research. However, conventional methods based on large-sample approximations often exhibit poor performance in case of very few studies (e.g., 2 to 4), which is…

Methodology · Statistics 2025-11-20 Ao Huang , Christian Röver , Tim Friede

Important questions for impact evaluation require knowledge not only of average effects, but of the distribution of treatment effects. The inability to observe individual counterfactuals makes answering these empirical questions…

Econometrics · Economics 2026-05-25 Bruno Fava

Predictive models ground many state-of-the-art developments in statistical brain image analysis: decoding, MVPA, searchlight, or extraction of biomarkers. The principled approach to establish their validity and usefulness is…

Quantitative Methods · Quantitative Biology 2017-06-26 Gaël Varoquaux

Score tests have the advantage of requiring estimation alone of the model restricted by the null hypothesis, which often is much simpler than models defined under the alternative hypothesis. This is typically so when the alternative…

Econometrics · Economics 2021-07-14 Giuseppe Cavaliere , Zeng-Hua Lu , Anders Rahbek , Yuhong Yang

P values or risk ratios from multiple, independent studies, observational or randomized, can be computationally combined to provide an overall assessment of a research question in meta-analysis. There is a need to examine the reliability of…

Methodology · Statistics 2021-10-28 S. Stanley Young , Warren B. Kindzierski

Current statistical inference problems in areas like astronomy, genomics, and marketing routinely involve the simultaneous testing of thousands -- even millions -- of null hypotheses. For high-dimensional multivariate distributions, these…

Methodology · Statistics 2017-04-25 Weixin Cai , Nima S. Hejazi , Alan E. Hubbard

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

This article proposes different tests for treatment effect heterogeneity when the outcome of interest, typically a duration variable, may be right-censored. The proposed tests study whether a policy 1) has zero distributional (average)…

Methodology · Statistics 2020-02-19 Pedro H. C. Sant'Anna

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

Experimental and observational studies often lead to spurious association between the outcome and independent variables describing the intervention, because of confounding to third-party factors. Even in randomized clinical trials,…

Methodology · Statistics 2023-12-04 Orestis Loukas , Ho Ryun Chung

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power…

We consider the conditional randomization test as a way to account for covariate imbalance in randomized experiments. The test accounts for covariate imbalance by comparing the observed test statistic to the null distribution of the test…

In meta-analyses, publication bias is a well-known, important and challenging issue because the validity of the results from a meta-analysis is threatened if the sample of studies retrieved for review is biased. One popular method to deal…

Methodology · Statistics 2020-07-03 Rui Duan , Jin Piao , Arielle Marks-Anglin , Jiayi Tong , Lifeng Lin , Haitao Chu , Jing Ning , Yong Chen

Ideally, a meta-analysis will summarize data from several unbiased studies. Here we consider the less than ideal situation in which contributing studies may be compromised by measurement error. Measurement error affects every study design,…

At every phase of scientific research, scientists must decide how to allocate limited resources to pursue the research inquiries with the greatest potential. This prioritization dictates which controlled interventions are studied, awarded…

Methodology · Statistics 2022-10-11 Bruce A. Corliss , Yaotian Wang , Heman Shakeri , Philip E. Bourne
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