10.6084/M9.FIGSHARE.C.6288617.V1
Natascha Ingrid Drude
Natascha Ingrid
Drude
0000-0002-7153-2894
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Lorena Martinez-Gamboa
Lorena
Martinez-Gamboa
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Meggie Danziger
Meggie
Danziger
0000-0001-9224-1722
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Anja Collazo
Anja
Collazo
0000-0001-5277-0005
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Silke Kniffert
Silke
Kniffert
0000-0001-5084-3960
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Janine Wiebach
Janine
Wiebach
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Charité - University Medicine Berlin
Gustav Nilsonne
Gustav
Nilsonne
0000-0001-5273-0150
Stockholm University
Karolinska Institute
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Frank Konietschke
Frank
Konietschke
Charité - University Medicine Berlin
Sophie K. Piper
Sophie K.
Piper
0000-0002-0147-8992
Charité - University Medicine Berlin
Samuel Pawel
Samuel
Pawel
0000-0003-2779-320X
University of Zurich
Charlotte Micheloud
Charlotte
Micheloud
0000-0002-4995-4505
University of Zurich
Leonhard Held
Leonhard
Held
0000-0002-8686-5325
University of Zurich
Florian Frommlet
Florian
Frommlet
0000-0003-4104-1768
Medical University of Vienna
Daniel Segelcke
Daniel
Segelcke
University Hospital Münster
Esther M. Pogatzki-Zahn
Esther M.
Pogatzki-Zahn
0000-0003-0981-3940
University Hospital Münster
Bernhard Voelkl
Bernhard
Voelkl
0000-0001-5454-2508
University of Bern
Tim Friede
Tim
Friede
Universitätsmedizin Göttingen
Edgar Brunner
Edgar
Brunner
0000-0001-7119-8622
Universitätsmedizin Göttingen
Astrid Dempfle
Astrid
Dempfle
0000-0002-2618-3920
University Hospital Schleswig-Holstein
Bernhard Haller
Bernhard
Haller
0000-0002-9723-393X
Technical University of Munich
Marie Juliane Jung
Marie Juliane
Jung
0000-0002-1503-6191
Leibniz Institute on Aging – Fritz Lipmann Institute
Lars Björn Riecken
Lars Björn
Riecken
0000-0002-1992-4591
Leibniz Institute on Aging – Fritz Lipmann Institute
Hans-Georg Kuhn
Hans-Georg
Kuhn
0000-0003-4247-5613
Charité - University Medicine Berlin
University of Gothenburg
Matthias Tenbusch
Matthias
Tenbusch
0000-0003-3951-9056
University of Erlangen-Nuremberg
Lina Maria Serna Higuita
Lina Maria Serna
Higuita
0000-0001-5182-8295
University of Tübingen
Edmond J. Remarque
Edmond J.
Remarque
Biomedical Primate Research Centre
Servan Luciano Grüninger-Egli
Servan Luciano
Grüninger-Egli
0000-0003-2900-1376
University of Zurich
Katrin Manske
Katrin
Manske
LMU Klinikum
Sebastian Kobold
Sebastian
Kobold
0000-0002-5612-4673
LMU Klinikum
German Cancer Research Center
Marion Rivalan
Marion
Rivalan
0000-0002-1642-7522
Charité - University Medicine Berlin
Lisa Wedekind
Lisa
Wedekind
Jena University Hospital
Juliane C. Wilcke
Juliane C.
Wilcke
Ludwig-Maximilians-Universität München
Anne-Laure Boulesteix
Anne-Laure
Boulesteix
0000-0002-2729-0947
Ludwig-Maximilians-Universität München
Marcus W. Meinhardt
Marcus W.
Meinhardt
0000-0002-5103-0731
Heidelberg University
Rainer Spanagel
Rainer
Spanagel
0000-0003-2151-4521
Heidelberg University
Simone Hettmer
Simone
Hettmer
0000-0003-1709-4448
University Medical Center Freiburg
Irene von Lüttichau
Irene
von Lüttichau
Technical University of Munich
Carla Regina
Carla
Regina
München Klinik Schwabing
Ulrich Dirnagl
Ulrich
Dirnagl
0000-0003-0755-6119
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Ulf Toelch
Ulf
Toelch
0000-0002-8731-3530
Berlin Institute of Health at Charité - Universitätsmedizin Berlin
Planning preclinical confirmatory multicenter trials to strengthen translation from basic to clinical research – a multi-stakeholder workshop report
Abstract Clinical translation from bench to bedside often remains challenging even despite promising preclinical evidence. Among many drivers like biological complexity or poorly understood disease pathology, preclinical evidence often lacks desired robustness. Reasons include low sample sizes, selective reporting, publication bias, and consequently inflated effect sizes. In this context, there is growing consensus that confirmatory multicenter studies -by weeding out false positives- represent an important step in strengthening and generating preclinical evidence before moving on to clinical research. However, there is little guidance on what such a preclinical confirmatory study entails and when it should be conducted in the research trajectory. To close this gap, we organized a workshop to bring together statisticians, clinicians, preclinical scientists, and meta-researcher to discuss and develop recommendations that are solution-oriented and feasible for practitioners. Herein, we summarize and review current approaches and outline strategies that provide decision-critical guidance on when to start and subsequently how to plan a confirmatory study. We define a set of minimum criteria and strategies to strengthen validity before engaging in a confirmatory preclinical trial, including sample size considerations that take the inherent uncertainty of initial (exploratory) studies into account. Beyond this specific guidance, we highlight knowledge gaps that require further research and discuss the role of confirmatory studies in translational biomedical research. In conclusion, this workshop report highlights the need for close interaction and open and honest debate between statisticians, preclinical scientists, meta-researchers (that conduct research on research), and clinicians already at an early stage of a given preclinical research trajectory.
Microbiology
Neuroscience
Biotechnology
Cancer
Science Policy
111714 Mental Health
110309 Infectious Diseases
figshare
2022
2022-11-08
2022-11-08
Collection
10.1186/s41231-022-00130-8
10.6084/m9.figshare.c.6288617
CC BY 4.0