One global rare cancer network with n-of-1 and Bayesian trial frameworks
Rare cancers are collectively common but each is too rare for normal trials. Link every rare cancer patient worldwide into one network with registries and trial designs built for small numbers.
Rare cancers make up roughly a quarter of cancer diagnoses and have worse survival; each is too infrequent for conventional trials and expertise is scattered. Networks such as EURACAN and the International Rare Cancers Initiative show partial solutions. The proposal is a global federated rare cancer network: a single patient-facing registration and referral pathway, standardised molecular workup, a shared registry with outcomes, and pre-agreed trial frameworks (Bayesian borrowing across related histologies, n-of-1 crossover designs, external control arms from the registry) accepted by regulators for approval decisions.
- 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.
- 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.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.