Increasing efficiency of preclinical research by group sequential designs

Journal article


Neumann, Konrad, Grittner, Ulrike, Piper, Sophie, Rex, Andre, Florez-Vargas, Oscar, Karystianis, George, Schneider, Alice, Wellwood, Ian, Siegerink, Bob, Ioannidis, John P. A., Kimmelman, Jonathan and Dirnagl, Ulrich. (2017). Increasing efficiency of preclinical research by group sequential designs. PLoS Biology. 15(3), p. Article e2001307. https://doi.org/10.1371/journal.pbio.2001307
AuthorsNeumann, Konrad, Grittner, Ulrike, Piper, Sophie, Rex, Andre, Florez-Vargas, Oscar, Karystianis, George, Schneider, Alice, Wellwood, Ian, Siegerink, Bob, Ioannidis, John P. A., Kimmelman, Jonathan and Dirnagl, Ulrich
Abstract

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.

Year2017
JournalPLoS Biology
Journal citation15 (3), p. Article e2001307
PublisherPublic Library of Science
ISSN1544-9173
Digital Object Identifier (DOI)https://doi.org/10.1371/journal.pbio.2001307
PubMed ID28282371
Scopus EID2-s2.0-85016981067
PubMed Central IDPMC5345756
Open accessPublished as ‘gold’ (paid) open access
Page range1-9
FunderGerman Federal Ministry of Education and Research (BMBF)
Publisher's version
License
File Access Level
Open
Output statusPublished
Publication dates
Online10 Mar 2017
Publication process dates
Deposited02 Dec 2022
Grant ID01EO1301
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