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In the era of fast-paced precision medicine, observational studies play a major role in properly evaluating new treatments in clinical practice. Yet, unobserved confounding can significantly compromise causal conclusions drawn from…

Machine Learning · Statistics 2026-03-20 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

Methodology · Statistics 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

Classifiers are often tested on relatively small data sets, which should lead to uncertain performance metrics. Nevertheless, these metrics are usually taken at face value. We present an approach to quantify the uncertainty of…

Machine Learning · Statistics 2021-03-05 Niklas Tötsch , Daniel Hoffmann

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…

Precise estimation of predictive uncertainty in deep neural networks is a critical requirement for reliable decision-making in machine learning and statistical modeling, particularly in the context of medical AI. Conformal Prediction (CP)…

Machine Learning · Computer Science 2024-01-05 Hamed Karimi , Reza Samavi

Data following an interval structure are increasingly prevalent in many scientific applications. In medicine, clinical events are often monitored between two clinical visits, making the exact time of the event unknown and generating…

Methodology · Statistics 2025-04-01 Carlos García Meixide , Michael R. Kosorok , Marcos Matabuena

Reliable uncertainty estimation is one of the major challenges for medical classification tasks. While many approaches have been proposed, recently the statistical framework of conformal predictions has gained a lot of attention, due to its…

Machine Learning · Computer Science 2025-06-24 Hendrik Mehrtens , Tabea Bucher , Titus J. Brinker

Monte Carlo methods to evaluate and maximize the likelihood function enable the construction of confidence intervals and hypothesis tests, facilitating scientific investigation using models for which the likelihood function is intractable.…

Methodology · Statistics 2017-02-13 Edward L. Ionides , Carles Breto , Joonha Park , Richard A. Smith , Aaron A. King

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

Methodology · Statistics 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

In the early stages of drug discovery, decisions regarding which experiments to pursue can be influenced by computational models. These decisions are critical due to the time-consuming and expensive nature of the experiments. Therefore, it…

Machine Learning · Computer Science 2024-09-09 Emma Svensson , Hannah Rosa Friesacher , Susanne Winiwarter , Lewis Mervin , Adam Arany , Ola Engkvist

Computer simulations have become an important tool across the biomedical sciences and beyond. For many important problems several different models or hypotheses exist and choosing which one best describes reality or observed data is not…

Quantitative Methods · Quantitative Biology 2010-01-20 Tina Toni , Michael P. H. Stumpf

Predictive biomarkers are playing an essential role in precision medicine. Identifying an optimal cutoff to select patient subsets with greater benefit from treatment is critical and more challenging for predictive biomarkers measured with…

Applications · Statistics 2025-04-29 Chi Zhang , Wei Shi , Spencer Woody , Qing Liu

Machine learning is used in medicine to support physicians in examination, diagnosis, and predicting outcomes. One of the most dynamic area is the usage of patient generated health data from intensive care units. The goal of this paper is…

The annotation of disease severity for medical image datasets often relies on collaborative decisions from multiple human graders. The intra-observer variability derived from individual differences always persists in this process, yet the…

Computer Vision and Pattern Recognition · Computer Science 2022-05-03 Chi Liu , Zongyuan Ge , Mingguang He , Xiaotong Han

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

Non-probabilistic convex model utilizes a convex set to quantify the uncertainty domain of uncertain-but-bounded parameters, which is very effective for structural uncertainty analysis with limited or poor-quality experimental data. To…

Other Statistics · Statistics 2018-01-18 Ni Bingyu , Jiang Chao , Huang Zhiliang

Virtual experiments (VEs), a modern tool in metrology, can be used to help perform an uncertainty evaluation for the measurand. Current guidelines in metrology do not cover the many possibilities to incorporate VEs into an uncertainty…

Applications · Statistics 2024-04-18 Finn Hughes , Manuel Marschall , Gerd Wübbeler , Gertjan Kok , Marcel van Dijk , Clemens Elster

When we use simulation to evaluate the performance of a stochastic system, the simulation often contains input distributions estimated from real-world data; therefore, there is both simulation and input uncertainty in the performance…

Methodology · Statistics 2020-11-10 Wei Xie , Barry L. Nelson , Russell R. Barton

The field of medical diagnosis has undergone a significant transformation with the advent of large language models (LLMs), yet the challenges of interpretability within these models remain largely unaddressed. This study introduces…

Computation and Language · Computer Science 2024-09-17 Junying Chen , Chi Gui , Anningzhe Gao , Ke Ji , Xidong Wang , Xiang Wan , Benyou Wang

In system development, epistemic uncertainty is an ever-present possibility when reasoning about the causal factors during hazard analysis. Such uncertainty is common when complicated systems interact with one another, and it is dangerous…

Software Engineering · Computer Science 2017-10-20 Chris Leong , Tim Kelly , Rob Alexander
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