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Clinical trials face mounting challenges: fragmented patient populations, slow enrollment, and unsustainable costs, particularly for late phase trials in oncology and rare diseases. While external control arms built from real-world data…

Machine Learning · Computer Science 2025-11-21 Perrine Chassat , Van Tuan Nguyen , Lucas Ducrot , Emilie Lanoy , Agathe Guilloux

In some randomized clinical trials, patients may die before the measurements of their outcomes. Even though randomization generates comparable treatment and control groups, the remaining survivors often differ significantly in background…

Applications · Statistics 2018-03-07 Fan Yang , Peng Ding

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

Survival analysis is a statistical technique used to estimate the time until an event occurs. Although it is applied across a wide range of fields, adjusting for reporting delays under practical constraints remains a significant challenge…

Machine Learning · Statistics 2025-10-27 Yuta Shikuri , Hironori Fujisawa

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…

Balanced representation learning methods have been applied successfully to counterfactual inference from observational data. However, approaches that account for survival outcomes are relatively limited. Survival data are frequently…

Machine Learning · Statistics 2021-03-04 Paidamoyo Chapfuwa , Serge Assaad , Shuxi Zeng , Michael J. Pencina , Lawrence Carin , Ricardo Henao

We overview Bayesian estimation, hypothesis testing, and model-averaging and illustrate how they benefit parametric survival analysis. We contrast the Bayesian framework to the currently dominant frequentist approach and highlight…

Methodology · Statistics 2022-09-13 František Bartoš , Frederik Aust , Julia M. Haaf

We consider event-driven clinical trials, where the analysis is performed once a pre-determined number of clinical events has been reached. For example, these events could be progression in oncology or a stroke in cardiovascular trials. At…

Methodology · Statistics 2021-08-23 Vladimir Anisimov , Stephen Gormley , Rosalind Baverstock , Cynthia Kineza

Motivation: Identification of genomic, molecular and clinical markers prognostic of patient survival is important for developing personalized disease prevention, diagnostic and treatment approaches. Modern omics technologies have made it…

Applications · Statistics 2024-03-05 Zhi Zhao , John Zobolas , Manuela Zucknick , Tero Aittokallio

Survival regression is widely used to model time-to-events data, to explore how covariates may influence the occurrence of events. Modern datasets often encompass a vast number of covariates across many subjects, with only a subset of the…

Methodology · Statistics 2024-09-18 Abhishek Mandal , Abhisek Chakraborty

Typically, electronic health record data are not collected towards a specific research question. Instead, they comprise numerous observations recruited at different ages, whose medical, environmental and oftentimes also genetic data are…

Methodology · Statistics 2024-11-19 Nir Keret , Malka Gorfine

When to initiate treatment on patients is an important problem in many medical studies such as AIDS and cancer. In this article, we formulate the treatment initiation time problem for time-to-event data and propose an optimal individualized…

Methodology · Statistics 2021-09-30 Xin Chen , Rui Song , Jiajia Zhang , Swann Arp Adams , Liuquan Sun , Wenbin Lu

With the rapid development of data collection and aggregation technologies in many scientific disciplines, it is becoming increasingly ubiquitous to conduct large-scale or online regression to analyze real-world data and unveil real-world…

Methodology · Statistics 2021-03-22 Jinfeng Xu , Zhiliang Ying , Na Zhao

Surrogate endpoint (SE) for overall survival in cancer patients is essential to improving the efficiency of oncology drug development. In practice, we may discover a new patient level association with survival, based on one or more clinical…

Applications · Statistics 2022-11-08 Wei Zou

Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

Combined inference for heterogeneous high-dimensional data is critical in modern biology, where clinical and various kinds of molecular data may be available from a single study. Classical genetic association studies regress a single…

Applications · Statistics 2017-03-22 Hélène Ruffieux , Anthony C. Davison , Jörg Hager , Irina Irincheeva

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Determining the sample size of an experiment can be challenging, even more so when incorporating external information via a prior distribution. Such information is increasingly used to reduce the size of the control group in randomized…

Applications · Statistics 2019-07-10 Beat Neuenschwander , Sebastian Weber , Heinz Schmidli , Anthony O'Hagan

Analyses of adverse events (AEs) are an important aspect of the evaluation of experimental therapies. The SAVVY (Survival analysis for AdVerse events with Varying follow-up times) project aims to improve the analyses of AE data in clinical…

We present a new method for analyzing stochastic epidemic models under minimal assumptions. The method, dubbed DSA, is based on a simple yet powerful observation, namely that population-level mean-field trajectories described by a system of…

Populations and Evolution · Quantitative Biology 2022-02-22 Francesco Di Lauro , Wasiur R. KhudaBukhsh , Istvan Z. Kiss , Eben Kenah , Max Jensen , Grzegorz A. Rempala
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