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Mid-study design modifications are becoming increasingly accepted in confirmatory clinical trials, so long as appropriate methods are applied such that error rates are controlled. It is therefore unfortunate that the important case of…

Applications · Statistics 2014-05-08 Dominic Magirr , Thomas Jaki , Franz Koenig , Martin Posch

We develop new methods to integrate experimental and observational data in causal inference. While randomized controlled trials offer strong internal validity, they are often costly and therefore limited in sample size. Observational data,…

Econometrics · Economics 2025-11-04 Xuelin Yang , Licong Lin , Susan Athey , Michael I. Jordan , Guido W. Imbens

Estimating individualized treatment rules is a central task for personalized medicine. [zhao2012estimating] and [zhang2012robust] proposed outcome weighted learning to estimate individualized treatment rules directly through maximizing the…

Methodology · Statistics 2017-10-02 Yifan Cui , Ruoqing Zhu , Michael Kosorok

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as…

Methodology · Statistics 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast

Sequential Monte Carlo (SMC) algorithms represent a suite of robust computational methodologies utilized for state estimation and parameter inference within dynamical systems, particularly in real-time or online environments where data…

This paper shows how data science can contribute to improving empirical research in economics by leveraging on large datasets and extracting information otherwise unsuitable for a traditional econometric approach. As a test-bed for our…

General Economics · Economics 2019-11-05 Marco Guerzoni , Consuelo R. Nava , Massimiliano Nuccio

Five-year cancer survival rates are widely reported and often interpreted to mean that early detection saves lives, that a late fatal diagnosis would have been prevented by earlier detection, and that increasing survival over time proves…

Methodology · Statistics 2026-03-23 Allen B. Downey

Machine learning (ML) holds great potential for accurately forecasting treatment outcomes over time, which could ultimately enable the adoption of more individualized treatment strategies in many practical applications. However, a…

Machine Learning · Statistics 2023-06-08 Toon Vanderschueren , Alicia Curth , Wouter Verbeke , Mihaela van der Schaar

Electronic health records (EHR) are widely used to study clinical decisions, yet unmeasured confounding remains a persistent challenge. Proxy variables offer a potential solution. In EHR data, clinicians already record many such…

Methodology · Statistics 2026-03-23 Haley Colgate Kottler , Amy Cochran

The expected information gain is an important quality criterion of Bayesian experimental designs, which measures how much the information entropy about uncertain quantity of interest $\theta$ is reduced on average by collecting relevant…

Computation · Statistics 2020-06-11 Takashi Goda , Tomohiko Hironaka , Takeru Iwamoto

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

Period-prevalent cohorts are often used for their cost-saving potential in epidemiological studies of survival outcomes. Under this design, prevalent patients allow for evaluations of long-term survival outcomes without the need for long…

Methodology · Statistics 2024-10-28 Nicholas Hartman

The accurate prediction of patient prognosis is a critical challenge in clinical practice. With the availability of various patient information, physicians can optimize medical care by closely monitoring disease progression and therapy…

Applications · Statistics 2023-11-28 He Weiyi

Decision making in modern stochastic systems, including e-commerce platforms, financial markets and healthcare systems, has evolved into a multifaceted process that combines information acquisition and adaptive information sources. This…

Optimization and Control · Mathematics 2026-01-07 Renyuan Xu , Thaleia Zariphopoulou , Luhao Zhang

An assurance calculation is a Bayesian alternative to a power calculation. One may be performed to aid the planning of a clinical trial, specifically setting the sample size or to support decisions about whether or not to perform a study.…

Applications · Statistics 2024-03-29 James Salsbury , Jeremy Oakley , Steven Julious , Lisa Hampson

Effective learning from electronic health records (EHR) data for prediction of clinical outcomes is often challenging because of features recorded at irregular timesteps and loss to follow-up as well as competing events such as death or…

Machine Learning · Computer Science 2022-08-11 Intae Moon , Stefan Groha , Alexander Gusev

The analysis of causal effects when the outcome of interest is possibly truncated by death has a long history in statistics and causal inference. The survivor average causal effect is commonly identified with more assumptions than those…

Methodology · Statistics 2020-03-24 Jaffer M. Zaidi , Eric J. Tchetgen Tchetgen , Tyler J. VanderWeele

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

A prediction interval covers a future observation from a random process in repeated sampling, and is typically constructed by identifying a pivotal quantity that is also an ancillary statistic. Analogously, a tolerance interval covers a…

Methodology · Statistics 2022-01-19 Geoffrey S Johnson

Survival analysis concerns the task of predicting the time until an event occurs. Often used in the medical field, survival analysis deals with incomplete (i.e., censored) data, for instance, from patients who did not experience the event…

Machine Learning · Computer Science 2026-05-29 Thalea Schlender , Peter A. N. Bosman , Tanja Alderliesten