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Applications of artificial intelligence (AI) in drug development continue to increase at a rapid pace. Regulatory authorities have provided increasingly clear perspectives on the use of AI in regulated applications, including recent draft…

Applications · Statistics 2026-05-25 Aaron M. Smith , Tala Fakhouri , Run Zhuang , Jonathan R. Walsh

Correctly identifying an individual's social context from passively worn sensors holds promise for delivering just-in-time adaptive interventions (JITAIs) to treat social anxiety disorder. In this study, we present results using passively…

Personalized treatments are gaining momentum across all fields of medicine. Precision medicine can be applied to neuromodulatory techniques, where focused brain stimulation treatments such as repetitive transcranial magnetic stimulation…

In this work, we proposed a novel inferential procedure assisted by machine learning based adjustment for randomized control trials. The method was developed under the Rosenbaum's framework of exact tests in randomized experiments with…

Methodology · Statistics 2024-07-23 Han Yu , Alan D. Hutson , Xiaoyi Ma

We propose a prognostic stratum matching framework that addresses the deficiencies of Randomized trial data subgroup analysis and transforms ObservAtional Data to be used as if they were randomized, thus paving the road for precision…

Applications · Statistics 2023-11-06 Dimitris Bertsimas , Angelos G. Koulouras , Georgios Antonios Margonis

The partial effectiveness of drugs is of importance to the pharmaceutical industry. Randomized controlled trials (RCTs) assuming the existence of a subgroup sensitive to the treatment are already used. These designs, however, are available…

Applications · Statistics 2018-12-07 Daria Rukina

Joint maximum likelihood (JML) estimation is one of the earliest approaches to fitting item response theory (IRT) models. This procedure treats both the item and person parameters as unknown but fixed model parameters and estimates them…

Methodology · Statistics 2019-06-17 Yunxiao Chen , Xiaoou Li , Siliang Zhang

Item response theory (IRT) models have been widely used in educational measurement testing. When there are repeated observations available for individuals through time, a dynamic structure for the latent trait of ability needs to be…

Applications · Statistics 2013-04-17 Xiaojing Wang , James O. Berger , Donald S. Burdick

Knowledge of the medical decision process, which can be modeled as medical decision trees (MDTs), is critical to building clinical decision support systems. However, current MDT construction methods rely heavily on time-consuming and…

Computation and Language · Computer Science 2025-10-30 Yuheng Li , Jiechao Gao , Wei Han , Wenwen Ouyang , Wei Zhu , Hui Yi Leong

Sequential multiple assignment randomized trials (SMARTs) are used to construct data-driven optimal intervention strategies for subjects based on their intervention and covariate histories in different branches of health and behavioral…

Methodology · Statistics 2022-04-28 Palash Ghosh , Xiaoxi Yan , Bibhas Chakraborty

In computerized adaptive testing (CAT), items (questions) are selected in real time based on the already observed responses, so that the ability of the examinee can be estimated as accurately as possible. This is typically formulated as a…

Statistics Theory · Mathematics 2015-01-08 Shiyu Wang , Georgios Fellouris , Hua-Hua Chang

Over time, clinical trials have increasingly incorporated complex design and analysis elements such as interim analyses, adaptations, multiple endpoints, and sophisticated multiplicity schemes for multiple endpoints and/or treatment arms…

An optimal dynamic treatment regime (DTR) is a sequence of decision rules aimed at providing the best course of treatments individualized to patients. While conventional DTR estimation uses longitudinal data, such data can also be…

Methodology · Statistics 2025-02-06 Larry Dong , Eleanor Pullenayegum , Rodolphe Thiébaut , Olli Saarela

Adaptive approaches, allowing for more flexible trial design, have been proposed for individually randomized trials to save time or reduce sample size. However, adaptive designs for cluster-randomized trials in which groups of participants…

Methodology · Statistics 2022-01-10 Junwei Shen , Shirin Golchi , Erica E. M. Moodie , David Benrimoh

Conducting experiments in federated, distributed, and heterogeneous testbeds is a challenging task for researchers. Researchers have to take care of the whole experiment life cycle, ensure the reproducibility of each run, and the…

Networking and Internet Architecture · Computer Science 2016-01-18 Matthias Wachs , Nadine Herold , Stephan-A. Posselt , Florian Dold , Georg Carle

This paper presents a machine learning approach to multidimensional item response theory (MIRT), a class of latent factor models that can be used to model and predict student performance from observed assessment data. Inspired by…

Machine Learning · Statistics 2025-01-08 Yoav Bergner , Peter F. Halpin , Jill-Jênn Vie

Mobile health studies can leverage longitudinal sensor data from smartphones to guide the application of personalized medical interventions. In this paper, we propose that adoption of an instrumental variable approach for randomized trials…

Large language models (LLMs) are increasingly deployed as "agents" for decision-making (DM) in interactive and dynamic environments. Yet, since they were not originally designed for DM, recent studies show that LLMs can struggle even in…

Artificial Intelligence · Computer Science 2025-11-07 Chanwoo Park , Ziyang Chen , Asuman Ozdaglar , Kaiqing Zhang

When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

Methodology · Statistics 2020-08-03 Johannes Krisam , Dorothea Weber , Richard F. Schlenk , Meinhard Kieser

Hierarchical composite endpoints, such as those analyzed using the Finkelstein-Schoenfeld (FS) statistic, are increasingly used in clinical trials for their ability to incorporate clinically prioritized outcomes. However, adaptive design…

Methodology · Statistics 2025-04-22 Krishna Padmanabhan , Cyrus Mehta
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