ideasIdea
A standing platform trial that assigns treatment by how the tumour escaped
Instead of a new trial for each resistance mechanism, one continuous trial could sort patients into arms based on the reason their last treatment failed.
A perpetual master protocol with a common progression-biopsy and plasma workup, a shared control arm and arms opened and closed as mechanisms and matched agents emerge. This shares infrastructure across sponsors, gives patients a reason to have the biopsy, and produces mechanism-specific efficacy estimates that no single-sponsor trial can. Precedents include Lung-MAP and I-SPY in other settings.
Hypothesis
A resistance platform enrols faster and produces mechanism-specific efficacy answers at lower cost per arm than separate single-mechanism trials, with a majority of enrolled patients receiving a matched agent.
Rationale
Resistance mechanisms are individually rare but collectively common, which is exactly the situation platform designs handle best; the workup requirement is the same for all arms.
What would test it
Launch with three arms in one disease area, and compare cost per randomised patient, time to first readout and screen-failure rate with matched standalone trials.
Maturity
speculative
Who has to act
research
Cost to try
Large (over $50M)
Years to first evidence
6
Bottlenecks it attacks
- Acquired resistance to every therapy · Nearly every targeted therapy stops working within months to a few years as the tumour adapts.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
- Trials enrol too few, too slowly · Fewer than one in ten adults with cancer joins a trial. Trials close for lack of patients, not lack of ideas.