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Though platform trials have been touted for their flexibility and streamlined use of trial resources, their statistical efficiency is not well understood. We fill this gap by establishing their greater efficiency for comparing the relative…

Methodology · Statistics 2022-01-28 Tzu-Jung Huang , Alex Luedtke , the AMP Investigators Group

We consider high-dimensional regression over subgroups of observations. Our work is motivated by biomedical problems, where disease subtypes, for example, may differ with respect to underlying regression models, but sample sizes at the…

Given the cost and duration of phase III and phase IV clinical trials, the development of statistical methods for go/no-go decisions is vital. In this paper, we introduce a Bayesian methodology to compute the probability of success based on…

Methodology · Statistics 2020-10-27 Ethan M. Alt , Matthew A. Psioda , Joseph G. Ibrahim

Bayesian hierarchical models are a methodology for aggregation and synthesis of data from heterogeneous settings, used widely in statistics and other disciplines. I apply this framework to the evidence from 7 randomized experiments of…

Economics · Quantitative Finance 2016-07-14 Rachael Meager

Suppose we have a binary treatment used to influence an outcome. Given data from an observational or controlled study, we wish to determine whether or not there exists some subset of observed covariates in which the treatment is more…

Methodology · Statistics 2016-03-22 Alexander R. Luedtke , Mark J. van der Laan

In clinical trials, ensuring the quality and validity of data for downstream analysis and results is paramount, thus necessitating thorough data monitoring. This typically involves employing edit checks and manual queries during data…

Other Statistics · Statistics 2026-01-15 Yuxi Zhao , Margaret Gamalo

We introduce a factor analysis model that summarizes the dependencies between observed variable groups, instead of dependencies between individual variables as standard factor analysis does. A group may correspond to one view of the same…

Machine Learning · Statistics 2014-11-19 Seppo Virtanen , Arto Klami , Suleiman A. Khan , Samuel Kaski

Subclassification estimators are one of the methods used to estimate causal effects of interest using the propensity score. This method is more stable compared to other weighting methods, such as inverse probability weighting estimators, in…

Methodology · Statistics 2024-10-22 Shunichiro Orihara , Tomotaka Momozaki

Meta-analysis aims to generalize results from multiple related statistical analyses through a combined analysis. While the natural outcome of a Bayesian study is a posterior distribution, traditional Bayesian meta-analyses proceed by…

Meta-analysis is widely used to integrate results from multiple experiments to obtain generalized insights. Since meta-analysis datasets are often heteroscedastic due to varying subgroups and temporal heterogeneity arising from experiments…

Methodology · Statistics 2026-01-19 Kohsuke Kubota , Shonosuke Sugasawa , Keiichi Ochiai , Takahiro Hoshino

Mendelian randomization is the use of genetic variants to assess the existence of a causal relationship between a risk factor and an outcome of interest. Here, we focus on two-sample summary-data Mendelian randomization analyses with many…

Quantitative Methods · Quantitative Biology 2022-09-16 Apostolos Gkatzionis , Stephen Burgess , Paul J. Newcombe

In this study, we introduce the application of causal disparity analysis to unveil intricate relationships and causal pathways between sensitive attributes and the targeted outcomes within real-world observational data. Our methodology…

Computers and Society · Computer Science 2024-08-08 Farnaz Kohankhaki , Shaina Raza , Oluwanifemi Bamgbose , Deval Pandya , Elham Dolatabadi

Subgroup analysis evaluates treatment effects across multiple sub-populations. When subgroups are defined by latent memberships inferred from imperfect measurements, the analysis typically involves two inter-connected models, a latent class…

Methodology · Statistics 2026-01-05 Yuanhui Luo , Xinzhou Guo , Yuqi Gu

Within-individual variability of health indicators measured over time is becoming commonly used to inform about disease progression. Simple summary statistics (e.g. the standard deviation for each individual) are often used but they are not…

Joint models (JM) for longitudinal and survival data have gained increasing interest and found applications in a wide range of clinical and biomedical settings. These models facilitate the understanding of the relationship between outcomes…

Methodology · Statistics 2023-08-25 Sida Chen , Danilo Alvares , Christopher Jackson , Jessica Barrett

In medical, social, and behavioral research we often encounter datasets with a multilevel structure and multiple correlated dependent variables. These data are frequently collected from a study population that distinguishes several…

Methodology · Statistics 2023-12-18 Xynthia Kavelaars , Joris Mulder , Maurits Kaptein

We propose a framework for testing the homogeneity of conditional average treatment effects (CATEs) across multiple experimental and observational studies. Our approach leverages multiple randomized trials to assess whether treatment…

Econometrics · Economics 2026-02-25 Ana Armendariz , Martin Huber

We investigate the estimation of subgroup treatment effects with observational data. Existing propensity score matching and weighting methods are mostly developed for estimating overall treatment effect. Although the true propensity score…

Methodology · Statistics 2017-07-20 Jing Dong , Junni L Zhang , Fan Li

Effect modification means the magnitude or stability of a treatment effect varies as a function of an observed covariate. Generally, larger and more stable treatment effects are insensitive to larger biases from unmeasured covariates, so a…

Methodology · Statistics 2018-03-12 Kwonsang Lee , Dylan S. Small , Paul R. Rosenbaum

Targeted therapies on the basis of genomic aberrations analysis of the tumor have shown promising results in cancer prognosis and treatment. Regardless of tumor type, trials that match patients to targeted therapies for their particular…

Applications · Statistics 2018-04-18 Yanxun Xu , Peter Mueller , Apostolia M Tsimberidou , Donald Berry