OnCo
ideasIdea

Stop excluding brain metastases from cancer trials

One in five people with advanced cancer has brain spread, yet most trials refuse them. Requiring brain cohorts would give those patients evidence instead of guesswork.

Brain metastasis exclusion criteria persist by habit, and regulators and professional bodies have issued guidance encouraging inclusion of patients with stable treated brain metastases. Making a CNS cohort with CNS-specific response endpoints a default expectation for registrational trials in CNS-tropic cancers, unless justified otherwise, would generate labelled intracranial data rather than post-approval retrospectives.

Hypothesis
A default-inclusion policy raises the proportion of new oncology approvals with intracranial activity data in the label from a minority to over half within five years, without slowing accrual.
Rationale
Inclusion of patients with treated brain metastases has been feasible where sponsors have tried it, and the resulting intracranial data changed practice in HER2-positive breast cancer and ALK-positive lung cancer. The barrier is convention and risk aversion, not science.
What would test it
Audit exclusion criteria in the last 200 registrational oncology protocols, publish the rate, then have one regulator or cooperative group adopt default CNS cohorts for CNS-tropic diseases and measure accrual, safety and labelling outcomes.
Maturity
speculative
Who has to act
regulator
Cost to try
Small (under $1M)
Years to first evidence
4
Bottlenecks it attacks

Connected

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