OnCo
ideasIdea

Independent replication of the key experiment before first-in-human academic trials

Before a new cancer drug from a university is given to people, a separate laboratory should have repeated the main experiment showing it works.

Industry routinely replicates academic findings internally before investing, and reports that a majority fail. Academic and small-biotech investigational new drug applications rest on the originating lab's data. Requiring, or funding through translational programmes, an independent replication of the pivotal efficacy experiment (in the same model, blinded, pre-registered) before first-in-human dosing would catch non-reproducible programmes before patients are exposed.

Hypothesis
At least a quarter of pivotal efficacy experiments will fail independent replication, and programmes whose experiments replicated will show higher early clinical activity than historical academic-origin programmes.
Rationale
Amgen and Bayer reported that only 11 to 25% of landmark preclinical findings reproduced in their hands; nothing in the regulatory pathway checks this for academic sponsors.
What would test it
Fund replication for twenty academic programmes approaching investigational new drug submission through an existing translational accelerator; report replication rate and subsequent clinical outcomes.
Maturity
speculative
Who has to act
regulator
Cost to try
Medium ($1M to $50M)
Years to first evidence
3
Bottlenecks it attacks

Connected

6top

Pages like this

not linked directly; found by shared links

ideas

4
Funders set aside a fixed share of budget for independent replicationMulti-laboratory preclinical trials as the standard for go/no-go decisionsPaid independent statistical review for preclinical papers that inform trialsTop journals require an independent lab to reproduce key findings before publication

bottlenecks

2
Preclinical results do not reproduceThe valley of death between lab and product