Sample size determination via learning-type curves

Fuente: arXiv
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Auteurs principaux: Dayimu, Alimu, Simidjievski, Nikola, Demiris, Nikolaos, Abraham, Jean
Format: Preprint
Publié: 2023
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author Dayimu, Alimu
Simidjievski, Nikola
Demiris, Nikolaos
Abraham, Jean
author_facet Dayimu, Alimu
Simidjievski, Nikola
Demiris, Nikolaos
Abraham, Jean
contents This paper is concerned with sample size determination methodology for prediction models. We propose combining the individual calculations via a learning-type curve. We suggest two distinct ways of doing so, a deterministic skeleton of a learning curve and a Gaussian process centred upon its deterministic counterpart. We employ several learning algorithms for modelling the primary endpoint and distinct measures for trial efficacy. We find that the performance may vary with the sample size, but borrowing information across sample size universally improves the performance of such calculations. The Gaussian process-based learning curve appears more robust and statistically efficient, while computational efficiency is comparable. We suggest that anchoring against historical evidence when extrapolating sample sizes should be adopted when such data are available. The methods are illustrated on binary and survival endpoints.
format Preprint
id arxiv_https___arxiv_org_abs_2303_09575
institution arXiv
publishDate 2023
record_format arxiv
spellingShingle Sample size determination via learning-type curves
Dayimu, Alimu
Simidjievski, Nikola
Demiris, Nikolaos
Abraham, Jean
Methodology
Applications
This paper is concerned with sample size determination methodology for prediction models. We propose combining the individual calculations via a learning-type curve. We suggest two distinct ways of doing so, a deterministic skeleton of a learning curve and a Gaussian process centred upon its deterministic counterpart. We employ several learning algorithms for modelling the primary endpoint and distinct measures for trial efficacy. We find that the performance may vary with the sample size, but borrowing information across sample size universally improves the performance of such calculations. The Gaussian process-based learning curve appears more robust and statistically efficient, while computational efficiency is comparable. We suggest that anchoring against historical evidence when extrapolating sample sizes should be adopted when such data are available. The methods are illustrated on binary and survival endpoints.
title Sample size determination via learning-type curves
topic Methodology
Applications
url https://arxiv.org/abs/2303.09575