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Background: When developing a clinical prediction model using time-to-event data, previous research focuses on the sample size to minimise overfitting and precisely estimate the overall risk. However, instability of individual-level risk…

Clinical prediction models enable healthcare professionals to estimate individual outcomes using patient characteristics. Current sample size guidelines for developing or updating models with continuous outcomes aim to minimise overfitting…

Background: Clinical prediction models are increasingly used to inform healthcare decisions, but determining the minimum sample size for their development remains a critical and unresolved challenge. Inadequate sample sizes can lead to…

Machine Learning · Computer Science 2026-03-02 Diana Shamsutdinova , Felix Zimmer , Oyebayo Ridwan Olaniran , Sarah Markham , Daniel Stahl , Gordon Forbes , Ewan Carr

Aims: To propose a general sample size framework for developing or updating a clinical prediction model using any statistical or machine learning method, based on drawing samples from anticipated posterior distributions and targeting…

When prospectively developing a new clinical prediction model (CPM), fixed sample size calculations are typically conducted before data collection based on sensible assumptions. But if the assumptions are inaccurate the actual sample size…

Clinical prediction models are estimated using a sample of limited size from the target population, leading to uncertainty in predictions, even when the model is correctly specified. Generally, not all patient profiles are observed…

Methodology · Statistics 2024-02-09 Doranne Thomassen , Saskia le Cessie , Hans van Houwelingen , Ewout Steyerberg

Research often necessitates of samples, yet obtaining large enough samples is not always possible. When it is, the researcher may use one of two methods for deciding upon the required sample size: rules-of-thumb, quick yet uncertain, and…

Methodology · Statistics 2016-04-08 Jose D. Perezgonzalez

Clinical prediction models estimate an individual's risk of a particular health outcome, conditional on their values of multiple predictors. A developed model is a consequence of the development dataset and the chosen model building…

Methodology · Statistics 2024-07-15 Richard D Riley , Gary S Collins

When evaluating the performance of a model for individualised risk prediction, the sample size needs to be large enough to precisely estimate the performance measures of interest. Current sample size guidance is based on precisely…

Clinical prediction models must be developed using sufficiently large datasets to minimise overfitting and ensure robust predictive performance. Existing sample size calculations assume complete predictor data for all included participants,…

How can we draw trustworthy scientific conclusions? One criterion is that a study can be replicated by independent teams. While replication is critically important, it is arguably insufficient. If a study is biased for some reason and other…

Methodology · Statistics 2025-02-06 Yujin Jeong , Dominik Rothenhäusler

Existing approaches to sample size calculations for developing clinical prediction models have focused on ensuring that the expected value of a chosen performance measure meets a pre-specified target. For example, to limit…

Methodology · Statistics 2025-09-18 Menelaos Pavlou , Rumana Z. Omar , Gareth Ambler

Over the last few decades, prediction models have become a fundamental tool in statistics, chemometrics, and related fields. However, to ensure that such models have high value, the inferences that they generate must be reliable. In this…

Estimation frameworks for statistical inference are preferred to hypothesis testing when quantifying uncertainty and precise estimation are more valuable than binary decisions about statistical significance. Study design for…

Methodology · Statistics 2025-10-29 Luke Hagar , Nathaniel T. Stevens

Objective: Provide guidance on sample size considerations for developing predictive models by empirically establishing the adequate sample size, which balances the competing objectives of improving model performance and reducing model…

Applications · Statistics 2024-07-25 Luis H. John , Jan A. Kors , Jenna M. Reps , Patrick B. Ryan , Peter R. Rijnbeek

Composite binary endpoints are increasingly used as primary endpoints in clinical trials. When designing a trial, it is crucial to determine the appropriate sample size for testing the statistical differences between treatment groups for…

Applications · Statistics 2019-01-15 Marta Bofill Roig , Guadalupe Gómez Melis

Multinomial logistic regression models allow one to predict the risk of a categorical outcome with more than 2 categories. When developing such a model, researchers should ensure the number of participants (n) is appropriate relative to the…

Recent research reveals that machine learning (ML) models are highly sensitive to minor changes in their training procedure, such as the inclusion or exclusion of a single data point, leading to conflicting predictions on individual data…

Machine Learning · Computer Science 2025-07-15 Falaah Arif Khan , Denys Herasymuk , Nazar Protsiv , Julia Stoyanovich

In fairness audits, a standard objective is to detect whether a given algorithm performs substantially differently between subgroups. Properly powering the statistical analysis of such audits is crucial for obtaining informative fairness…

Applications · Statistics 2023-12-11 Harvineet Singh , Fan Xia , Mi-Ok Kim , Romain Pirracchio , Rumi Chunara , Jean Feng

We cover how to determine a sufficiently large sample size for a $K$-armed randomized experiment in order to estimate conditional counterfactual expectations in data-driven subgroups. The sub-groups can be output by any feature space…

Machine Learning · Computer Science 2024-03-08 Gabriel Ruiz
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