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Increasing efficiency of preclinical research by group sequential designs
Despite the potential benefits of sequential designs, studies evaluating treatments or experimental manipulations in preclinical experimental biomedicine almost exclusively use classical block designs. Our aim with this article is to bring the existing methodology of group sequential designs to the...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5345756/ https://www.ncbi.nlm.nih.gov/pubmed/28282371 http://dx.doi.org/10.1371/journal.pbio.2001307 |
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author | Neumann, Konrad Grittner, Ulrike Piper, Sophie K. Rex, Andre Florez-Vargas, Oscar Karystianis, George Schneider, Alice Wellwood, Ian Siegerink, Bob Ioannidis, John P. A. Kimmelman, Jonathan Dirnagl, Ulrich |
author_facet | Neumann, Konrad Grittner, Ulrike Piper, Sophie K. Rex, Andre Florez-Vargas, Oscar Karystianis, George Schneider, Alice Wellwood, Ian Siegerink, Bob Ioannidis, John P. A. Kimmelman, Jonathan Dirnagl, Ulrich |
author_sort | Neumann, Konrad |
collection | PubMed |
description | Despite the potential benefits of sequential designs, studies evaluating treatments or experimental manipulations in preclinical experimental biomedicine almost exclusively use classical block designs. Our aim with this article is to bring the existing methodology of group sequential designs to the attention of researchers in the preclinical field and to clearly illustrate its potential utility. Group sequential designs can offer higher efficiency than traditional methods and are increasingly used in clinical trials. Using simulation of data, we demonstrate that group sequential designs have the potential to improve the efficiency of experimental studies, even when sample sizes are very small, as is currently prevalent in preclinical experimental biomedicine. When simulating data with a large effect size of d = 1 and a sample size of n = 18 per group, sequential frequentist analysis consumes in the long run only around 80% of the planned number of experimental units. In larger trials (n = 36 per group), additional stopping rules for futility lead to the saving of resources of up to 30% compared to block designs. We argue that these savings should be invested to increase sample sizes and hence power, since the currently underpowered experiments in preclinical biomedicine are a major threat to the value and predictiveness in this research domain. |
format | Online Article Text |
id | pubmed-5345756 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2017 |
publisher | Public Library of Science |
record_format | MEDLINE/PubMed |
spelling | pubmed-53457562017-03-30 Increasing efficiency of preclinical research by group sequential designs Neumann, Konrad Grittner, Ulrike Piper, Sophie K. Rex, Andre Florez-Vargas, Oscar Karystianis, George Schneider, Alice Wellwood, Ian Siegerink, Bob Ioannidis, John P. A. Kimmelman, Jonathan Dirnagl, Ulrich PLoS Biol Perspective Despite the potential benefits of sequential designs, studies evaluating treatments or experimental manipulations in preclinical experimental biomedicine almost exclusively use classical block designs. Our aim with this article is to bring the existing methodology of group sequential designs to the attention of researchers in the preclinical field and to clearly illustrate its potential utility. Group sequential designs can offer higher efficiency than traditional methods and are increasingly used in clinical trials. Using simulation of data, we demonstrate that group sequential designs have the potential to improve the efficiency of experimental studies, even when sample sizes are very small, as is currently prevalent in preclinical experimental biomedicine. When simulating data with a large effect size of d = 1 and a sample size of n = 18 per group, sequential frequentist analysis consumes in the long run only around 80% of the planned number of experimental units. In larger trials (n = 36 per group), additional stopping rules for futility lead to the saving of resources of up to 30% compared to block designs. We argue that these savings should be invested to increase sample sizes and hence power, since the currently underpowered experiments in preclinical biomedicine are a major threat to the value and predictiveness in this research domain. Public Library of Science 2017-03-10 /pmc/articles/PMC5345756/ /pubmed/28282371 http://dx.doi.org/10.1371/journal.pbio.2001307 Text en © 2017 Neumann et al http://creativecommons.org/licenses/by/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. |
spellingShingle | Perspective Neumann, Konrad Grittner, Ulrike Piper, Sophie K. Rex, Andre Florez-Vargas, Oscar Karystianis, George Schneider, Alice Wellwood, Ian Siegerink, Bob Ioannidis, John P. A. Kimmelman, Jonathan Dirnagl, Ulrich Increasing efficiency of preclinical research by group sequential designs |
title | Increasing efficiency of preclinical research by group sequential designs |
title_full | Increasing efficiency of preclinical research by group sequential designs |
title_fullStr | Increasing efficiency of preclinical research by group sequential designs |
title_full_unstemmed | Increasing efficiency of preclinical research by group sequential designs |
title_short | Increasing efficiency of preclinical research by group sequential designs |
title_sort | increasing efficiency of preclinical research by group sequential designs |
topic | Perspective |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5345756/ https://www.ncbi.nlm.nih.gov/pubmed/28282371 http://dx.doi.org/10.1371/journal.pbio.2001307 |
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