After approval, a pragmatic trial in the patients the pivotal trial excluded
Drugs are approved on trials of fit, younger patients and then given to everyone. A required follow-on trial in older, sicker and more diverse patients would show whether the benefit holds in real life.
As a condition of approval, sponsors fund a pragmatic randomised trial with broad eligibility run in community settings, using routinely collected endpoints (OS, hospitalisation, treatment discontinuation) and minimal extra visits, modelled on the FDA's Project Pragmatica and the Pragmatica-Lung trial. Results feed label updates on dosing and benefit in the broader population.
- 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.
- Older and multimorbid patients are excluded and undertreated · Most people with cancer are over 65 but most trial patients are younger and fitter. We guess how to treat the majority.
Pages like this
not linked directly; found by shared links- IdeaRequire post-approval evidence in patients over 75 and update labels accordingly
Shares Older and multimorbid patients are excluded and undertreated, Weak real-world evidence and registries, Real-world evidence.
- IdeaMandatory real-world reporting for patients excluded from pivotal trials
Shares Older and multimorbid patients are excluded and undertreated, Weak real-world evidence and registries, Real-world evidence.
- IdeaStandards for external control arms built from federated real-world data
Shares Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaRegistry-based randomised trials for oncology comparative effectiveness
Shares Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaA public rulebook for when an external or synthetic control arm is acceptable
Shares Weak real-world evidence and registries, Real-world evidence, Trial design, endpoints and cost.
- IdeaLinked prescribing and outcome data to find drug interactions with cancer therapy
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaWearable activity data as a validated real-world endpoint
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaNo mCODE, no payment: tie oncology reimbursement to a minimal structured record
Shares Weak real-world evidence and registries, Real-world evidence.