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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...

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Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
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.
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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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