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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

Motivated by the Acute Respiratory Distress Syndrome Network (ARDSNetwork) ARDS respiratory management (ARMA) trial, we developed a flexible Bayesian machine learning approach to estimate the average causal effect and heterogeneous causal…

应用统计 · 统计学 2024-10-29 Xinyuan Chen , Michael O. Harhay , Guangyu Tong , Fan Li

Due to patient heterogeneity in response to various aspects of any treatment program, biomedical and clinical research is gradually shifting from the traditional "one-size-fits-all" approach to the new paradigm of personalized medicine. An…

统计方法学 · 统计学 2019-11-21 Min Qian , Bibhas Chakraborty , Raju Maiti , Ying Kuen Cheung

There is interest in learning about the causal effects of modern contraceptive use on empowerment outcomes. Data on this question often come from family planning (FP) programs that increase access to FP and facilitate contraceptive use…

统计方法学 · 统计学 2026-03-31 Lucas Godoy Garraza , Ilene Speizer , Leontine Alkema

Longitudinal biomarkers are frequently collected in clinical studies due to their strong association with time-to-event outcomes. While considerable progress has been made in methods for jointly modeling longitudinal and survival data,…

统计方法学 · 统计学 2026-02-18 Yuan Bian , Shelley B. Bull

Bayesian additive regression trees (BART) are popular Bayesian ensemble models used in regression and classification analysis. Under this modeling framework, the regression function is approximated by an ensemble of decision trees,…

统计计算 · 统计学 2025-11-26 Marco Battiston , Yu Luo

We consider the procedure proposed by Bhandari et al. (2009) in the context of two-treatment clinical trials, with the objective of minimizing the applications of the less effective drug to the least number of patients. Our focus is on an…

统计理论 · 数学 2025-06-30 Sampurna Kundu , Jayant Jha , Subir Kumar Bhandari

The application of loss reweighting in modern deep learning presents a nuanced picture. While it fails to alter the terminal learning phase in overparameterized deep neural networks (DNNs) trained on high-dimensional datasets, empirical…

机器学习 · 计算机科学 2026-01-21 Yize Zhao , Christos Thrampoulidis

Sample size reestimation can be a powerful tool to ensure that a clinical trial meets its prespecified power requirements when uncertainty regarding a design parameter exists at the planning stage. However, long term primary endpoints can…

统计方法学 · 统计学 2026-05-14 Aritra Mukherjee , Michael J Grayling , James J M S Wason

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

统计方法学 · 统计学 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

The dynamics of molecules are governed by rare event transitions between long-lived (metastable) states. To explore these transitions efficiently, many enhanced sampling protocols have been introduced that involve using simulations with…

化学物理 · 物理学 2022-09-30 Maaike M. Galama , Hao Wu , Andreas Krämer , Mohsen Sadeghi , Frank Noé

With the robust uptick in the applications of Bayesian external data borrowing, eliciting a prior distribution with the proper amount of information becomes increasingly critical. The prior effective sample size (ESS) is an intuitive and…

统计方法学 · 统计学 2024-04-23 Hongtao Zhang , Keaven M Anderson , Zachary Zimmer , Gregory Golm , Aditi Sapre , Joseph G Ibrahim

The primary analysis of randomized screening trials for cancer typically adheres to the intention-to-screen principle, measuring cancer-specific mortality reductions between screening and control arms. These mortality reductions result from…

统计方法学 · 统计学 2021-07-30 Sudipta Saha , Zhihui Liu , Olli Saarela

In many practical tasks it is needed to estimate an effect of treatment on individual level. For example, in medicine it is essential to determine the patients that would benefit from a certain medicament. In marketing, knowing the persons…

统计方法学 · 统计学 2019-02-28 Aleksey Buzmakov

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

统计方法学 · 统计学 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

We consider the design of a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome. We detail Bayesian inference for the analysis of the trial using a linear mixed-effects model. The treatment is compared to…

统计方法学 · 统计学 2022-08-29 Kevin J Wilson

Win statistics have gained increasing popularity as primary analysis methods for clinical trials with hierarchical endpoints (HEs) as primary endpoints. However, existing sample size and power calculation approaches in trial design still…

统计方法学 · 统计学 2026-05-19 Baoshan Zhang , Huiman X. Barnhart , Yuan Wu , Roland A. Matsouaka

Marginal structural models fit via inverse probability of treatment weighting are commonly used to control for confounding when estimating causal effects from observational data. When planning a study that will be analyzed with marginal…

应用统计 · 统计学 2020-03-16 Bonnie E. Shook-Sa , Michael G. Hudgens

Sequential, multiple assignment randomized trials (SMARTs), which assist in the optimization of adaptive interventions, are growing in popularity in education and behavioral sciences. This is unsurprising, as adaptive interventions reflect…

统计方法学 · 统计学 2023-09-26 Timothy Lycurgus , Amy Kilbourne , Daniel Almirall

Conditional power calculations are frequently used to guide the decision whether or not to stop a trial for futility or to modify planned sample size. These ignore the information in short-term endpoints and baseline covariates, and thereby…

统计方法学 · 统计学 2019-04-11 Kelly Van Lancker , An Vandebosch , Stijn Vansteelandt