Group sequential designs produce reliable results in preclinical trials

Researchers from Charité-Universitätsmedizin Berlin and the Berlin Institute of Health (BIH) have developed a more flexible study design to improve the efficiency of preclinical research, publishing their findings in PLOS Biology.

In order for new treatments to be developed, preclinical research must be reliable and reproducible. Unfortunately, preclinical results often fail in upholding results and standards in real world practice due to low quality control, lack of randomization and inappropriate study design.

Led by Ulrich Dirnagl, head of experimental neurology at Charité and founding director of the Center for Transforming Biomedical Research at the BIH, the team used group sequential designs to improve the efficiency of preclinical trials in the move toward real-world use. While regularly used in clinical research, group sequential design studies are rare in preclinical research. The flexibility can provide researchers the ability to design studies with lager samples sizes and more concrete evidence. The stopping criteria allow for the studies to end when the treatment fails to produce reliable results.

"Our computer models show that, without affecting the validity of the study, group sequential designs lead to resource savings of 30% when compared to the block designs commonly used in preclinical studies," said Ulrike Grittner, one of the study's first authors. "Higher standards of quality in preclinical research make it easier to translate research findings into clinical research. This means that promising new treatments can be spotted sooner, and can be made available to patients more quickly."