English
Related papers

Related papers: Regulatory Expectations for Bayesian Methods in Dr…

200 papers

Model-assisted interval designs such as the Keyboard design are transparent and easy to implement in phase I oncology trials. However, interim decisions based solely on data from the current dose may overlook informative signals from…

Applications · Statistics 2026-05-26 Jiangyan Zhao , Xian Shi , Jin Xu

Design of experiments has traditionally relied on the frequentist hypothesis testing framework where the optimal size of the experiment is specified as the minimum sample size that guarantees a required level of power. Sample size…

Methodology · Statistics 2025-08-07 Shirin Golchi , Luke Hagar

Optimal design is a critical yet challenging task within many applications. This challenge arises from the need for extensive trial and error, often done through simulations or running field experiments. Fortunately, sequential optimal…

Machine Learning · Computer Science 2025-03-25 Xubo Yue , Raed Al Kontar , Albert S. Berahas , Yang Liu , Blake N. Johnson

Randomized controlled experiments assess new policy impacts on performance metrics to inform launch decisions. Traditional approaches evaluate metrics independently despite correlations, and mixed results (e.g., positive revenue impact,…

Applications · Statistics 2026-01-29 Hoiyi Ng , Guido Imbens

Modern epidemiological analytics increasingly use machine learning models that offer strong prediction but often lack calibrated uncertainty. Bayesian methods provide principled uncertainty quantification, yet are viewed as difficult to…

Machine Learning · Statistics 2025-11-18 Debashis Chatterjee

Clinical decisions are often guided by clinical prediction models or diagnostic tests. Decision curve analysis (DCA) combines classical assessment of predictive performance with the consequences of using these strategies for clinical…

Methodology · Statistics 2023-08-07 Giuliano N. F. Cruz , Keegan Korthauer

Observers and practitioners of artificial intelligence (AI) have proposed an FDA-style licensing regime for the most advanced AI models, or 'frontier' models. In this paper, we explore the applicability of approval regulation -- that is,…

Computers and Society · Computer Science 2024-08-05 Daniel Carpenter , Carson Ezell

In developing products for rare diseases, statistical challenges arise due to the limited number of patients available for participation in drug trials and other clinical research. Bayesian adaptive clinical trial designs offer the…

Applications · Statistics 2019-09-19 Xiao Wu , Yi Xu , Bradley P. Carlin

The design of an experiment can be always be considered at least implicitly Bayesian, with prior knowledge used informally to aid decisions such as the variables to be studied and the choice of a plausible relationship between the…

Methodology · Statistics 2017-01-03 David C. Woods , Antony M. Overstall , Maria Adamou , Timothy W. Waite

With the advancement of precision medicine there is an increasing need for design and analysis methods in clinical trials with the objective of investigating effect heterogeneity and estimating subgroup effects. As this requires precise…

Methodology · Statistics 2026-03-12 Shirin Golchi , Satoshi Morita

Healthcare companies must submit pharmaceutical drugs or medical devices to regulatory bodies before marketing new technology. Regulatory bodies frequently require transparent and interpretable computational modelling to justify a new…

Applications · Statistics 2019-01-15 Simon Olofsson , Marc Peter Deisenroth , Ruth Misener

Inferring causal structures from experimentation is a central task in many domains. For example, in biology, recent advances allow us to obtain single-cell expression data under multiple interventions such as drugs or gene knockouts.…

Machine Learning · Computer Science 2023-02-24 Alexander Hägele , Jonas Rothfuss , Lars Lorch , Vignesh Ram Somnath , Bernhard Schölkopf , Andreas Krause

Reinforcement learning and data-driven autonomous controllers are commonly evaluated using cumulative reward and empirical success frequency under finite simulation trajectories. However, such empirical metrics do not necessarily provide…

Machine Learning · Computer Science 2026-05-28 Fei Jiang , Lei Yang

Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and…

We propose a Bayesian approach for both medical inquiry and disease inference, the two major phases in differential diagnosis. Unlike previous work that simulates data from given probabilities and uses ML algorithms on them, we directly use…

Artificial Intelligence · Computer Science 2021-10-26 Hong Guan , Chitta Baral

The purpose of a phase I dose-finding clinical trial is to investigate the toxicity profiles of various doses for a new drug and identify the maximum tolerated dose. Over the past three decades, various dose-finding designs have been…

Methodology · Statistics 2021-11-25 Yunshan Duan , Shijie Yuan , Yuan Ji , Peter Mueller

Recent advancements in generative machine learning have enabled rapid progress in biological design tools (BDTs) such as protein structure and sequence prediction models. The unprecedented predictive accuracy and novel design capabilities…

Computers and Society · Computer Science 2023-12-01 Richard Moulange , Max Langenkamp , Tessa Alexanian , Samuel Curtis , Morgan Livingston

Research on decision support applications in healthcare, such as those related to diagnosis, prediction, treatment planning, etc., have seen enormously increased interest recently. This development is thanks to the increase in data…

Machine Learning · Computer Science 2021-03-25 Jussi Tohka , Mark van Gils

Causal discovery is crucial for understanding complex systems and informing decisions. While observational data can uncover causal relationships under certain assumptions, it often falls short, making active interventions necessary. Current…

Machine Learning · Computer Science 2024-06-18 Yuxuan Wang , Mingzhou Liu , Xinwei Sun , Wei Wang , Yizhou Wang

Some scientific research questions ask to guide decisions and others do not. By their nature frequentist hypothesis-tests yield a dichotomous test decision as result, rendering them rather inappropriate for latter types of research…

Methodology · Statistics 2021-10-20 Patrick Schwaferts , Thomas Augustin