A standard for tumour-agnostic approvals: minimum histologies and hierarchical modelling
Some drugs are approved for any cancer with a particular mutation. Clear rules on how many cancer types must be tested, and how to combine results across them, would make these approvals more consistent and faster.
Regulators publish a standard for tissue-agnostic evidence: a minimum number of histologies with a minimum number of evaluable patients each, a Bayesian hierarchical model for borrowing across histologies with pre-specified heterogeneity thresholds, and rules for excluding histologies that show no activity. Sponsors design basket trials to the standard, and approvals list the histologies supporting them.
- 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.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.
Pages like this
not linked directly; found by shared links- IdeaAdopt tumour-agnostic cancer drug labels across regions by reliance, not re-review
Shares NTRK, RET, Tumour-agnostic (tissue-agnostic) approval, Basket, umbrella, and platform trials.
- IdeaReciprocal recognition of tumour-agnostic and rare-indication approvals across regulators
Shares NTRK, RET, Tumour-agnostic (tissue-agnostic) approval, BRAF.
- IdeaAgree in advance how to borrow evidence between similar rare cancers
Shares Basket, umbrella, and platform trials, Regulatory divergence between regions, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
- IdeaOne global rare cancer network with n-of-1 and Bayesian trial frameworks
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- IdeaStandards for external control arms built from federated real-world data
Shares Basket, umbrella, and platform trials, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
- IdeaOne standing umbrella trial for all rare cancers in a country
Shares Tumour-agnostic (tissue-agnostic) approval, Basket, umbrella, and platform trials, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
- IdeaThe pathology lab triggers a trial referral the day a rare cancer is diagnosed
Shares Gene fusion, Microsatellite instability (MSI-H) / mismatch repair deficiency (dMMR), Rare and paediatric cancers without markets.
- ProductLarotrectinib
Shares NTRK, Gene fusion, Tumour-agnostic (tissue-agnostic) approval.