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

Source: https://onco.cc/ideas/idea-bio1-multicentre-mouse-trials/  
OnCo record `idea-bio1-multicentre-mouse-trials` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A drug that works in one laboratory's mice often fails elsewhere. Running the confirmatory animal study as a randomised, blinded, multi-laboratory trial, as stroke and amyotrophic lateral sclerosis research has done, would catch this before a human trial; oncology has no such standing infrastructure.

## Summary

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.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- 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.
- Proposed test: 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
- Actor: philanthropy

## Sources

- Bottleneck evidence (Lab models that fail to predict what happens in patients): Wong, Siah & Lo, Estimation of clinical trial success rates (Biostatistics 2019): https://doi.org/10.1093/biostatistics/kxx069

## Connected records

- technologies: [Patient-derived xenografts](https://onco.cc/technologies/pdx-models/)
- institutions: [Cancer Research UK](https://onco.cc/institutions/cruk/), [National Cancer Institute (NIH)](https://onco.cc/institutions/nci/)
- bottlenecks: [Lab models that fail to predict what happens in patients](https://onco.cc/bottlenecks/b-preclinical-models/), [Preclinical results do not reproduce](https://onco.cc/bottlenecks/b-reproducibility/), [The valley of death between lab and product](https://onco.cc/bottlenecks/b-translational-valley/)
- key papers: [Estimation of clinical trial success rates and related parameters](https://onco.cc/key-papers/paper-wong-biostatistics/)

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