Randomise inside the registry that already follows every patient
Rare cancer patients are already tracked in registries. Offering randomisation inside the registry makes trials far cheaper and lets almost anyone take part.
Trials within cohorts, or registry-based randomised trials, use existing registry infrastructure for identification, consent, allocation and outcome capture, and have been used successfully in cardiology at a fraction of conventional trial cost. Rare cancers are ideal candidates because registries already exist, populations are dispersed, and conventional site-based trials cannot reach enough patients.
- 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.
- Weak real-world evidence and registries · We do not reliably know what happens to patients after approval, so we cannot tell which drugs deliver in practice.
Pages like this
not linked directly; found by shared links- IdeaEmulate the trial in real-world data first to decide which trials to run
Shares AACR Project GENIE, SEER (Surveillance, Epidemiology, and End Results), Weak real-world evidence and registries, Real-world evidence.
- IdeaStandards for external control arms built from federated real-world data
Shares Basket, umbrella, and platform trials, Weak real-world evidence and registries, Real-world evidence, 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, Neuroendocrine tumours, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
- IdeaOne standing umbrella trial for all rare cancers in a country
Shares ClinicalTrials.gov, Basket, umbrella, and platform trials, Neuroendocrine tumours, Rare and paediatric cancers without markets.
- IdeaValidate real-world progression endpoints so pragmatic trials can use them
Shares AACR Project GENIE, Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaLink every national cancer registry to tumour genomics
Shares AACR Project GENIE, SEER (Surveillance, Epidemiology, and End Results), Weak real-world evidence and registries, Rare and paediatric cancers without markets.
- IdeaPay for residual disease tests only inside a trial or registry
Shares SEER (Surveillance, Epidemiology, and End Results), ClinicalTrials.gov, Weak real-world evidence and registries, Real-world evidence.
- IdeaA public rulebook for when an external or synthetic control arm is acceptable
Shares Weak real-world evidence and registries, Real-world evidence, Rare and paediatric cancers without markets, Trial design, endpoints and cost.