Multi-Laboratory Testing of Early Preclinical Discoveries Identifies Promising Treatments

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Abstract
A fundamental challenge in drug development is the frequent failure of early laboratory research to translate into clinical benefit. One promising solution is to confirm findings from exploratory single-laboratory studies across multiple laboratories before clinical testing. We investigated this approach following the conduct of preclinical multi-laboratory studies across different fields of medicine. For this, we evaluated effect sizes, experimental rigor, and a set of criteria to identify determinants of confirmation success. When tested under increased rigor, only a fraction of multi-laboratory studies confirmed the initial results. The underlying effect size reduction was associated with outcome-relevant experimental differences between exploratory and confirmatory stages. In summary, multi-laboratory studies proved highly informative and served as an effective filter for promising treatments. One Sentence Summary: Confirmatory testing of exploratory findings in rigorous multi-laboratory settings reveals which hold up and what limits their translation.
Funding Information
  • Bundesministerium für Forschung, Technologie und Raumfahrt (01KC1901A)
  • Bundesministerium für Forschung, Technologie und Raumfahrt (01KC2306)
  • Bundesministerium für Forschung, Technologie und Raumfahrt (01KC2003A)
  • Bundesministerium für Forschung, Technologie und Raumfahrt (01KC2004A)
  • Bundesministerium für Forschung, Technologie und Raumfahrt (01KC2005)
  • Bundesministerium für Forschung, Technologie und Raumfahrt (01KC2006A)
  • Deutsche Forschungsgemeinschaft (BO3139/7)
  • Deutsche Forschungsgemeinschaft (BO3139/7-2)

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