OnCo
ideasIdea

Multi-centre randomised animal trials before committing to a human trial

A drug that works in one laboratory's mice often fails elsewhere. Running the key animal study across several independent laboratories first would catch this.

The confirmatory preclinical trial concept borrows randomisation, blinding, power calculation and multi-site replication from clinical research. Multi-laboratory preclinical trials have been run in stroke (the MULTI-PART approach) and in amyotrophic lateral sclerosis, and reversed several accepted findings. Oncology, where phase 2 attrition is highest, has no equivalent standing infrastructure.

Hypothesis
Candidates that pass a randomised, blinded, three-laboratory confirmatory preclinical trial have a materially higher phase 2 success rate than candidates advanced on single-laboratory data.
Rationale
The main source of translational failure is unreliable effect estimates rather than species differences alone; replication is the cheapest available fix and costs a fraction of a failed phase 2.
What would test it
Fund a preclinical trials network to run confirmatory studies for ten agents heading into phase 2 and track subsequent clinical outcomes against matched controls.
Maturity
preclinical evidence
Who has to act
philanthropy
Cost to try
Medium ($1M to $50M)
Years to first evidence
6
Bottlenecks it attacks

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