Adopt tumour-agnostic cancer drug labels across regions by reliance, not re-review
Some drugs work on a genetic change whatever the cancer. When one regulator approves such a label, others should adopt it rather than demanding trials per cancer type.
Tumour-agnostic approvals (NTRK fusions, MSI-high, BRAF V600E, RET) rest on basket trials that some regulators have accepted and others have restricted to specific tumour types, producing labels that differ by region for the same evidence. Patients with rare tumours in restrictive regions are excluded. The proposal is a standing agreement that tumour-agnostic indications granted by one stringent regulator on basket-trial evidence are adopted by partners within 90 days, with a shared post-marketing registry to fill evidence gaps in rare histologies.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.
- 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.
Pages like this
not linked directly; found by shared links- IdeaA standard for tumour-agnostic approvals: minimum histologies and hierarchical modelling
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.
- IdeaAutomatic reciprocity of orphan and rare-paediatric designations between regulators
Shares Regulatory divergence between regions, Rare and paediatric cancers without markets.
- IdeaOne global rare cancer network with n-of-1 and Bayesian trial frameworks
Shares Basket, umbrella, and platform trials, Regulatory divergence between regions, Rare and paediatric cancers without markets.
- CollectionPancreatic Cancer Action Network (PanCAN)
Shares Tumour-agnostic (tissue-agnostic) approval, Basket, umbrella, and platform trials.
- IdeaOne global paediatric cancer development plan instead of separate FDA and EMA plans
Shares Regulatory divergence between regions, Rare and paediatric cancers without markets.
- IdeaStandards for external control arms built from federated real-world data
Shares Basket, umbrella, and platform trials, Rare and paediatric cancers without markets.