SearcharxivSearch

arXiv subjects

Ewan Carr

Publications and source records attributed to Ewan Carr.

5 recordsLinked to original sources

Adaptive Gaussian Process Search for Simulation-Based Sample Size Estimation in Clinical Prediction Models: Validation of the pmsims R Package

Background: Determining an adequate sample size is essential for developing reliable and generalisable clinical prediction models, yet practical guidance on selecting appropriate methods remains limited. Existing analytical and simulation-based approaches often rely on restrictive assumptions and focus on mean-based criteria. We present and validate pmsims, an R package that uses Gaussian process surrogate modelling to provide a flexible and computationally efficient simulation-based framework for sample size determination across diverse prediction settings. Methods: We conducted a comprehensive simulation study with two aims. First, we compared three search engines implemented in pmsims: a Gaussian process-based adaptive method, a deterministic bisection method, and a hybrid approach, across binary, continuous, and survival outcomes. Second, we benchmarked the best-performing pmsims engine against existing analytical (pmsampsize) and simulation-based (samplesizedev) methods, evaluating recommended sample sizes, computational time, and achieved performance on large independent validation datasets. Results: The Gaussian process-based method consistently produced the most stable sample size estimates, particularly in low-signal, high-dimensional settings. In benchmarking, pmsims achieved performance close to prespecified targets across all outcome types, matching simulation-based approaches and outperforming analytical methods in more challenging scenarios. Conclusions: pmsims provides an efficient and flexible framework for principled sample size planning in clinical prediction modelling, requiring fewer model evaluations than non-adaptive simulation approaches.

stat.CO

Sample Size Calculations for Developing Clinical Prediction Models: Overview and pmsims R package

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.

cs.LG

Multilingual Lexical Feature Analysis of Spoken Language for Predicting Major Depression Symptom Severity

Background: Remotely captured spoken language could provide objective, regular indicators of depression symptom severity. However, research to date has largely used non-clinical, cross-sectional written language and complex machine learning (ML) approaches with limited interpretability. Methods: We used linear mixed-effect models to identify interpretable lexical features associated with symptom severity in data from the RADAR-MDD study that comprised 5,846 smartphone recordings and Patient Health Questionnaire (PHQ-8) scores from 467 participants in the UK, Netherlands and Spain. We then developed ML models and systematically assessed via nested cross-validation whether interpretable lexical features or high-dimensional vector embeddings improved the accuracy of PHQ-8 prediction over sociodemographic and confounding features. Results: Depression symptom severity was associated with five lexical features, including reductions in word count measures, use of first-person plural pronouns and positive word frequency. Associations were stable across countries, except for positive word frequency. Lexical features and vector embeddings did improve prediction accuracy beyond baseline models. Limitations: Our cohort was skewed in age (median = 53, IQR 35 to 62) and majority female (n=357), potentially affecting the generalizability of our results. A lack of natural language processing tools for non-English languages restricted our feature choices. Conclusion: Further research is required to realise the value of spoken lexical markers in clinical research and practice including larger and more diverse samples, elicitation protocol development and analytical methods that account for within- and between-individual variations.

cs.CL

A methodological framework and exemplar protocol for the collection and analysis of repeated speech samples

Speech and language biomarkers have the potential to be regular, objective assessments of symptom severity in several health conditions, both in-clinic and remotely using mobile devices. However, the complex nature of speech and often subtle changes associated with health mean that findings are highly dependent on methodological and cohort choices. These are often not reported adequately in studies investigating speech-based health assessment, hindering the progress of methodological speech research. Our objectives were to) facilitate replicable speech research by presenting an adaptable speech collection and analytical method and design checklist for other researchers to adapt for their own experiments and develop an exemplar protocol that reduces and controls for confounding factors in repeated recordings of speech, including device choice, speech elicitation task and non-pathological variability. The presented protocol comprises the elicitation of read speech, held vowels and a picture description collected with a freestanding condenser microphone, 3 smartphones and a headset. We extracted a set of 14 exemplar speech features. We collected healthy speech from 28 individuals 3 times in 1 day, repeated at the same times 8-11 weeks later, and from 25 individuals on 3 days in 1 week at fixed times. Participant characteristics collected included sex, age, native language status and voice use habits. Before each recording, we collected information on recent voice use, food and drink intake, and emotional state. The extracted features are presented providing a resource of normative values. Speech data collection, processing, analysis and reporting towards clinical research and practice varies widely. Greater harmonisation of study protocols and consistent reporting are urgently required to translate speech processing into clinical research and practice.

cs.SD

Towards robust paralinguistic assessment for real-world mobile health (mHealth) monitoring: an initial study of reverberation effects on speech

Speech is promising as an objective, convenient tool to monitor health remotely over time using mobile devices. Numerous paralinguistic features have been demonstrated to contain salient information related to an individual's health. However, mobile device specification and acoustic environments vary widely, risking the reliability of the extracted features. In an initial step towards quantifying these effects, we report the variability of 13 exemplar paralinguistic features commonly reported in the speech-health literature and extracted from the speech of 42 healthy volunteers recorded consecutively in rooms with low and high reverberation with one budget and two higher-end smartphones and a condenser microphone. Our results show reverberation has a clear effect on several features, in particular voice quality markers. They point to new research directions investigating how best to record and process in-the-wild speech for reliable longitudinal health state assessment.

cs.SD