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Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

Methodology · Statistics 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

Bayesian response adaptive clinical trials are currently evaluating experimental therapies for several diseases. Adaptive decisions, such as pre-planned variations of the randomization probabilities, attempt to accelerate the development of…

Methodology · Statistics 2021-05-25 Marta Bonsaglio , Sandra Fortini , Steffen Ventz , Lorenzo Trippa

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

Applications · Statistics 2019-02-04 Timothy NeCamp , Josh Gardner , Christopher Brooks

Digital monitoring studies collect real-time high frequency data via mobile sensors in the subjects' natural environment. This data can be used to model the impact of changes in physiology on recurrent event outcomes such as smoking, drug…

Methodology · Statistics 2022-04-15 Walter Dempsey

Bayesian inference and the use of posterior or posterior predictive probabilities for decision making have become increasingly popular in clinical trials. The current practice in Bayesian clinical trials relies on a hybrid…

Methodology · Statistics 2024-04-30 Shirin Golchi , James Willard

We use the exact finite sample likelihood and statistical decision theory to answer questions of ``why?'' and ``what should you have done?'' using data from randomized experiments and a utility function that prioritizes safety over…

Econometrics · Economics 2024-07-26 Neil Christy , A. E. Kowalski

The integration of mobile health (mHealth) devices into behavioral health research has fundamentally changed the way researchers and interventionalists are able to collect data as well as deploy and evaluate intervention strategies. In…

Methodology · Statistics 2021-12-17 Matthew D. Koslovsky , Emily T. Hebert , Michael S. Businelle , Marina Vannucci

Randomized experiments play a major role in data-driven decision making across many different fields and disciplines. In medicine, for example, randomized controlled trials (RCTs) are the backbone of clinical trial methodology for testing…

Applications · Statistics 2016-08-30 Andrew W. Correia

Nearly a quarter of visits to the Emergency Department are for conditions that could have been managed via outpatient treatment; improvements that allow patients to quickly recognize and receive appropriate treatment are crucial. The…

Quantitative Methods · Quantitative Biology 2021-12-01 Sara M. Clifton , Chaeryon Kang , Jingyi Jessica Li , Qi Long , Nirmish Shah , Daniel M. Abrams

Agent-based simulation with a synthetic population can help us compare different treatment conditions while keeping everything else constant within the same population (i.e., as digital twins). Such population-scale simulations require…

Methodology · Statistics 2024-03-26 Abdulrahman A. Ahmed , M. Amin Rahimian , Mark S. Roberts

In a randomized control trial, the precision of an average treatment effect estimator can be improved either by collecting data on additional individuals, or by collecting additional covariates that predict the outcome variable. We propose…

Methodology · Statistics 2017-09-27 Pedro Carneiro , Sokbae Lee , Daniel Wilhelm

In the design of clinical trials, it is essential to assess the design operating characteristics (e.g., power and the type I error rate). Common practice for the evaluation of operating characteristics in Bayesian clinical trials relies on…

Methodology · Statistics 2026-03-17 Luke Hagar , Shirin Golchi

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

Estimating the effect of a treatment on a given outcome, conditioned on a vector of covariates, is central in many applications. However, learning the impact of a treatment on a continuous temporal response, when the covariates suffer…

Machine Learning · Computer Science 2019-06-11 Guangyi Zhang , Reza Ashrafi , Anne Juuti , Kirsi Pietiläinen , Pekka Marttinen

There are multiple cluster randomised trial designs that vary in when the clusters cross between control and intervention states, when observations are made within clusters, and how many observations are made at that time point. Identifying…

Methodology · Statistics 2023-07-20 Samuel I. Watson , Alan Girling , Karla Hemming

It is a common practice in randomized clinical trials with the standard survival outcome to follow patients until a prespecified number of events have been observed, a type of trial known as the event-driven trial. The event-driven design…

Methodology · Statistics 2026-02-10 Jingwen Zhang , Satoshi Hattori

Minimizing the number of patients exposed to potentially harmful drugs in early onco logical trials is a major concern during planning. Adaptive designs account for the inherent uncertainty about the true effect size by determining the…

Applications · Statistics 2016-05-03 Kevin Kunzmann , Meinhard Kieser

When resources are scarce, an allocation policy is needed to decide who receives a resource. This problem occurs, for instance, when allocating scarce medical resources and is often solved using modern ML methods. This paper introduces…

Machine Learning · Computer Science 2024-02-20 Niclas Boehmer , Yash Nair , Sanket Shah , Lucas Janson , Aparna Taneja , Milind Tambe

We consider the following comparative effectiveness scenario. There are two treatments for a particular medical condition: a randomized experiment has demonstrated mediocre effectiveness for the first treatment, while a non-randomized study…

Methodology · Statistics 2024-08-23 Brian Knaeble , Erich Kummerfeld

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

Methodology · Statistics 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong