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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

Bivariate meta-analysis provides a useful framework for combining information across related studies and has been utilised to combine evidence from clinical studies to evaluate treatment efficacy on two outcomes. It has also been used to…

Applications · Statistics 2022-05-20 Tasos Papanikos , John R Thompson , Keith R Abrams , Sylwia Bujkiewicz

In this paper, we propose a robust method to estimate the average treatment effects in observational studies when the number of potential confounders is possibly much greater than the sample size. We first use a class of penalized…

Methodology · Statistics 2018-12-21 Yang Ning , Sida Peng , Kosuke Imai

Cumulative probability models (CPMs) are a robust alternative to linear models for continuous outcomes. However, they are not feasible for very large datasets due to elevated running time and memory usage, which depend on the sample size,…

Computation · Statistics 2022-07-15 Chun Li , Guo Chen , Bryan E. Shepherd

Joint models for longitudinal biomarkers and time-to-event data are widely used in longitudinal studies. Many joint modeling approaches have been proposed to deal with different types of longitudinal biomarkers and survival outcomes.…

Methodology · Statistics 2016-09-27 Molei Liu , Jiehuan Sun , Jose D. Herazo-Maya , Naftali Kaminski , Hongyu Zhao

Objective: Provide guidance on sample size considerations for developing predictive models by empirically establishing the adequate sample size, which balances the competing objectives of improving model performance and reducing model…

Applications · Statistics 2024-07-25 Luis H. John , Jan A. Kors , Jenna M. Reps , Patrick B. Ryan , Peter R. Rijnbeek

Time-varying covariates in longitudinal studies frequently evolve through reciprocal feedback, undergo role reversal, and reflect unobserved individual heterogeneity. Standard statistical frameworks often assume fixed covariate roles and…

Methodology · Statistics 2026-02-27 Niloofar Ramezani , Pascal Nitiema , Jeffrey R. Wilson

Experimental comparisons of performance represent an important aspect of research on optimization algorithms. In this work we present a methodology for defining the required sample sizes for designing experiments with desired statistical…

Neural and Evolutionary Computing · Computer Science 2018-10-16 Felipe Campelo , Fernanda Takahashi

Conventional methods for analyzing composite endpoints in clinical trials often only focus on the time to the first occurrence of all events in the composite. Therefore, they have inherent limitations because the individual patients' first…

Methodology · Statistics 2022-11-29 Jialu Wang , Yeh-Fong Chen , Thomas Gwise

We propose a new method for multivariate response regression and covariance estimation when elements of the response vector are of mixed types, for example some continuous and some discrete. Our method is based on a model which assumes the…

Methodology · Statistics 2022-03-04 Karl Oskar Ekvall , Aaron J. Molstad

Response times collected in computerised assessments provide information about the underlying response process and may exhibit within-person variation over the course of a test. We propose a latent variable model for log response times that…

Methodology · Statistics 2026-05-29 Gabriel Wallin , Nivedita Bhaktha

Joint Models for longitudinal and time-to-event data have gained a lot of attention in the last few years as they are a helpful technique to approach common a data structure in clinical studies where longitudinal outcomes are recorded…

In various biomedical studies, analysis often focuses on data magnitudes, particularly when algebraic signs are irrelevant or lost. For repeated measures studies involving magnitude outcomes, incorporating random effects is essential as…

Methodology · Statistics 2025-07-16 Wen Teng , Niall D. Ferguson , Ewan C. Goligher , Anna Heath

The win ratio offers a flexible approach to incorporate the hierarchy of clinical outcomes into the analysis of a composite endpoint, enabling simultaneous consideration of multiple outcome types, unlike traditional time-to-first-event…

Methodology · Statistics 2025-07-22 David Kronthaler , Matthias Schwenkglenks , Felix Beuschlein , Ulrike Held

The primary endpoint in oncology is usually overall survival, where differences between therapies may only be observable after many years. To avoid withholding of a promising therapy, preliminary approval based on a surrogate endpoint is…

Methodology · Statistics 2022-05-25 Samuel Kilian , Johannes Krisam , Meinhard Kieser

In medical and biological research, longitudinal data and survival data types are commonly seen. Traditional statistical models mostly consider to deal with either of the data types, such as linear mixed models for longitudinal data, and…

Methodology · Statistics 2021-07-12 Jizi Shangguan

The era of big data is coming, and evidence-based medicine is attracting increasing attention to improve decision making in medical practice via integrating evidence from well designed and conducted clinical research. Meta-analysis is a…

Methodology · Statistics 2016-10-05 Dehui Luo , Xiang Wan , Jiming Liu , Tiejun Tong

Background: Linear mixed-effects models are central for analyzing longitudinal continuous data, yet many learners meet them as scattered formulas or software output rather than as a coherent workflow. There is a need for a single,…

Methodology · Statistics 2025-11-19 Sunday A. Adetunji

We introduce a multivariate multidimensional mixed-effects regression model in a finite mixture framework. We relax the usual unidimensionality assumption on the random effects multivariate distribution. Thus, we introduce a…

Methodology · Statistics 2014-10-20 Alessandra Marcelletti , Antonello Maruotti , Giovanni Trovato

When evaluating the performance of a model for individualised risk prediction, the sample size needs to be large enough to precisely estimate the performance measures of interest. Current sample size guidance is based on precisely…

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