Diana Shamsutdinova

Diana Shamsutdinova, Felix Zimmer, Oyebayo Ridwan Olaniran et al.

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 overfitting, poor generalisability, and biased predictions. Existing approaches, such as heuristic rules, closed-form formulas, and simulation-based methods, vary in flexibility and accuracy, particularly for complex data structures and machine learning models. Methods: We review current methodologies for sample size estimation in prediction modelling and introduce a conceptual framework that distinguishes between mean-based and assurance-based criteria. Building on this, we propose a novel simulation-based approach that integrates learning curves, Gaussian Process optimisation, and assurance principles to identify sample sizes that achieve target performance with high probability. This approach is implemented in pmsims, an open-source, model-agnostic R package. Results: Through case studies, we demonstrate that sample size estimates vary substantially across methods, performance metrics, and modelling strategies. Compared to existing tools, pmsims provides flexible, efficient, and interpretable solutions that accommodate diverse models and user-defined metrics while explicitly accounting for variability in model performance. Conclusions: Our framework and software advance sample size methodology for clinical prediction modelling by combining flexibility with computational efficiency. Future work should extend these methods to hierarchical and multimodal data, incorporate fairness and stability metrics, and address challenges such as missing data and complex dependency structures.

Jaya Chaturvedi, Diana Shamsutdinova, Felix Zimmer et al.

Sample size calculation is an essential step in most data-based disciplines. Large enough samples ensure representativeness of the population and determine the precision of estimates. This is true for most quantitative studies, including those that employ machine learning methods, such as natural language processing, where free-text is used to generate predictions and classify instances of text. Within the healthcare domain, the lack of sufficient corpora of previously collected data can be a limiting factor when determining sample sizes for new studies. This paper tries to address the issue by making recommendations on sample sizes for text classification tasks in the healthcare domain. Models trained on the MIMIC-III database of critical care records from Beth Israel Deaconess Medical Center were used to classify documents as having or not having Unspecified Essential Hypertension, the most common diagnosis code in the database. Simulations were performed using various classifiers on different sample sizes and class proportions. This was repeated for a comparatively less common diagnosis code within the database of diabetes mellitus without mention of complication. Smaller sample sizes resulted in better results when using a K-nearest neighbours classifier, whereas larger sample sizes provided better results with support vector machines and BERT models. Overall, a sample size larger than 1000 was sufficient to provide decent performance metrics. The simulations conducted within this study provide guidelines that can be used as recommendations for selecting appropriate sample sizes and class proportions, and for predicting expected performance, when building classifiers for textual healthcare data. The methodology used here can be modified for sample size estimates calculations with other datasets.

Jaya Chaturvedi, Saniya Deshpande, Chenkai Ma et al.

Introduction: Clinical text classification using natural language processing (NLP) models requires adequate training data to achieve optimal performance. For that, 200-500 documents are typically annotated. The number is constrained by time and costs and lacks justification of the sample size requirements and their relationship to text vocabulary properties. Methods: Using the publicly available MIMIC-III dataset containing hospital discharge notes with ICD-9 diagnoses as labels, we employed pre-trained BERT embeddings followed by Random Forest classifiers to identify 10 randomly selected diagnoses, varying training corpus sizes from 100 to 10,000 documents, and analyzed vocabulary properties by identifying strong and noisy predictive words through Lasso logistic regression on bag-of-words embeddings. Results: Learning curves varied significantly across the 10 classification tasks despite identical preprocessing and algorithms, with 600 documents sufficient to achieve 95% of the performance attainable with 10,000 documents for all tasks. Vocabulary analysis revealed that more strong predictors and fewer noisy predictors were associated with steeper learning curves, where every 100 additional noisy words decreased accuracy by approximately 0.02 while 100 additional strong predictors increased maximum accuracy by approximately 0.04.