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Error correction of sequenced reads remains a difficult task, especially in single-cell sequencing projects with extremely non-uniform coverage. While existing error correction tools designed for standard (multi-cell) sequencing data…

Quantitative Methods · Quantitative Biology 2013-01-31 Sergey I. Nikolenko , Anton I. Korobeynikov , Max A. Alekseyev

Studies using assays to quantify the expression of thousands of genes on tens to thousands of cell samples have been carried out for over 20 years. Such assays are based on microarrays, DNA sequencing or other molecular technologies. All…

Statistics Theory · Mathematics 2025-06-18 Jiming Jiang , Johann A. Gagnon-Bartsch , Terence P. Speed

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

In biomolecular systems (especially all-atom models) with many degrees of freedom such as proteins and nucleic acids, there exist an astronomically large number of local-minimum-energy states. Conventional simulations in the canonical…

Statistical Mechanics · Physics 2010-12-30 Ayori Mitsutake , Yoshiharu Mori , Yuko Okamoto

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

Genetic risk prediction is an important component of individualized medicine, but prediction accuracies remain low for many complex diseases. A fundamental limitation is the sample sizes of the studies on which the prediction algorithms are…

Methodology · Statistics 2017-06-20 Sihai Dave Zhao

The use of high-dimensional data for targeted therapeutic interventions requires new ways to characterize the heterogeneity observed across subgroups of a specific population. In particular, models for partially exchangeable data are needed…

Methodology · Statistics 2020-08-18 Francesco Denti , Federico Camerlenghi , Michele Guindani , Antonietta Mira

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…

Methodology · Statistics 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

In early phase drug development of combination therapy, the primary objective is to preliminarily assess whether there is additive activity from a novel agent when combined with an established monotherapy. Due to potential feasibility…

Methodology · Statistics 2025-02-24 Zhaohua Lu , John Toso , Girma Ayele , Philip He

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods…

Methodology · Statistics 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

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…

Methodology · Statistics 2022-08-29 Kevin J Wilson

Randomized controlled trials (RCTs) are widely regarded as the gold standard for causal inference in biomedical research. For instance, when estimating the average treatment effect on the treated (ATT), a doubly robust estimation procedure…

Methodology · Statistics 2025-09-26 Chi-Shian Dai , Chao Ying , Yang Ning , Jiwei Zhao

Researchers require timely access to real-world longitudinal electronic health records (EHR) to develop, test, validate, and implement machine learning solutions that improve the quality and efficiency of healthcare. In contrast, health…

Machine Learning · Computer Science 2020-12-21 Siddharth Biswal , Soumya Ghosh , Jon Duke , Bradley Malin , Walter Stewart , Jimeng Sun

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

Methodology · Statistics 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

Electrocardiogram (ECG) is a widely used non-invasive diagnostic tool for heart diseases. Many studies have devised ECG analysis models (e.g., classifiers) to assist diagnosis. As an upstream task, researches have built generative models to…

Machine Learning · Computer Science 2023-05-30 Jintai Chen , Kuanlun Liao , Kun Wei , Haochao Ying , Danny Z. Chen , Jian Wu

In this study, I propose a five-step algorithm for synthetic control method for comparative studies. My algorithm builds on the synthetic control model of Abadie et al., 2015 and the later model of Amjad et al., 2018. I apply all three…

General Economics · Economics 2021-10-27 Mani Bayani

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

Methodology · Statistics 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

Chest X-rays (CXR) are essential for diagnosing a variety of conditions, but when used on new populations, model generalizability issues limit their efficacy. Generative AI, particularly denoising diffusion probabilistic models (DDPMs),…

In observational studies with time-to-event outcomes, the g-formula can be used to estimate a treatment effect in the presence of confounding factors. However, the asymptotic distribution of the corresponding stochastic process is…

Statistics Theory · Mathematics 2024-04-26 Jasmin Rühl , Sarah Friedrich
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