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Simulation offers a simple and flexible way to estimate the power of a clinical trial when analytic formulae are not available. The computational burden of using simulation has, however, restricted its application to only the simplest of…

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

Methodology · Statistics 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

We propose a novel adaptive design for clinical trials with time-to-event outcomes and covariates (which may consist of or include biomarkers). Our method is based on the expected entropy of the posterior distribution of a proportional…

Applications · Statistics 2016-03-29 James E. Barrett

We study optimization for data-driven decision-making when we have observations of the uncertain parameters within the optimization model together with concurrent observations of covariates. Given a new covariate observation, the goal is to…

Optimization and Control · Mathematics 2022-07-28 Rohit Kannan , Güzin Bayraksan , James R. Luedtke

Algorithms and technologies are essential tools that pervade all aspects of our daily lives. In the last decades, health care research benefited from new computer-based recruiting methods, the use of federated architectures for data…

Computers and Society · Computer Science 2023-01-26 Chiara Criscuolo , Tommaso Dolci , Mattia Salnitri

Computational models are powerful tools for understanding human cognition and behavior. They let us express our theories clearly and precisely, and offer predictions that can be subtle and often counter-intuitive. However, this same…

This study designs an adaptive experiment for efficiently estimating average treatment effects (ATEs). In each round of our adaptive experiment, an experimenter sequentially samples an experimental unit, assigns a treatment, and observes…

Methodology · Statistics 2024-06-21 Masahiro Kato , Akihiro Oga , Wataru Komatsubara , Ryo Inokuchi

Hierarchical random effect models are used for different purposes in clinical research and other areas. In general, the main focus is on population parameters related to the expected treatment effects or group differences among all units of…

Applications · Statistics 2021-04-07 Maryna Prus , Norbert Benda , Rainer Schwabe

Experimentation is widely utilized for causal inference and data-driven decision-making across disciplines. In an A/B experiment, for example, an online business randomizes two different treatments (e.g., website designs) to their customers…

Methodology · Statistics 2025-01-15 Wenxuan Guo , JungHo Lee , Panos Toulis

Targeted therapies on the basis of genomic aberrations analysis of the tumor have shown promising results in cancer prognosis and treatment. Regardless of tumor type, trials that match patients to targeted therapies for their particular…

Applications · Statistics 2018-04-18 Yanxun Xu , Peter Mueller , Apostolia M Tsimberidou , Donald Berry

In recent years, real-world external controls have grown in popularity as a tool to empower randomized placebo-controlled trials, particularly in rare diseases or cases where balanced randomization is unethical or impractical. However, as…

Methodology · Statistics 2024-11-14 Chenyin Gao , Shu Yang , Mingyang Shan , Wenyu Ye , Ilya Lipkovich , Douglas Faries

The e-value is gaining traction as a robust alternative to p-values and Bayes factors for quantifying statistical evidence. e-values are a promising method for adaptive clinical trials due to their anytime-validity: e-values ensure type I…

Methodology · Statistics 2026-05-28 Stef Baas , Judith ter Schure , Joost van Rosmalen

Benkeser et al. demonstrate how adjustment for baseline covariates in randomized trials can meaningfully improve precision for a variety of outcome types. Their findings build on a long history, starting in 1932 with R.A. Fisher and…

Methodology · Statistics 2026-03-03 Laura B. Balzer , Erica Cai , Lucas Godoy Garraza , Pracheta Amaranath

Scalable and accurate identification of specific clinical outcomes has been enabled by machine-learning applied to electronic medical record (EMR) systems. The development of classification models requires the collection of a complete…

Methodology · Statistics 2020-11-09 W. Katherine Tan , Patrick J. Heagerty

Model identification of battery dynamics is a central problem in energy research; many energy management systems and design processes rely on accurate battery models for efficiency optimization. The standard methodology for battery…

Machine Learning · Computer Science 2023-10-13 Gokhan Budan , Francesca Damiani , Can Kurtulus , N. Kemal Ure

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

Methodology · Statistics 2010-12-01 Peter F. Thall

The question of how individual patient data from cohort studies or historical clinical trials can be leveraged for designing more powerful, or smaller yet equally powerful, clinical trials becomes increasingly important in the era of…

Methodology · Statistics 2022-09-01 Sandra Siegfried , Stephen Senn , Torsten Hothorn

Design of experiments (DOE) is playing an essential role in learning and improving a variety of objects and processes. The article discusses the application of unsupervised machine learning to support the pragmatic designs of complex…

Machine Learning · Computer Science 2021-10-07 Alex Glushkovsky

This article describes a new, efficient way of finding control and state trajectories in optimal control problems by reformulation as a system of differential-algebraic equations (DAEs). The optimal control and state vectors can be obtained…

Systems and Control · Electrical Eng. & Systems 2025-06-13 Prakitr Srisuma , George Barbastathis , Richard D. Braatz

Treatment effect estimation is a fundamental problem in causal inference. We focus on designing efficient randomized controlled trials, to accurately estimate the effect of some treatment on a population of $n$ individuals. In particular,…

Machine Learning · Computer Science 2022-10-14 Raghavendra Addanki , David Arbour , Tung Mai , Cameron Musco , Anup Rao