Every patient on an accelerated-approval drug enrolled in a registry until confirmation
Drugs approved early on promising but unproven results would have every treated patient followed in a registry, so we know within two years whether the promise held.
Accelerated approvals rest on surrogate endpoints; confirmatory trials take a median of several years and some are never completed. The proposal makes registry enrolment with automatic outcome capture (via mCODE and registry linkage) a condition of prescribing under accelerated approval, giving regulators an interim real-world signal on survival and toxicity while the confirmatory trial runs. The FDA's 2023 authority to require confirmatory trials be underway at approval is the policy hook; the registry is the complementary data hook.
- 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.
- 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- IdeaGood practice standards and inspection for real-world data sources
Shares Weak real-world evidence and registries, Real-world evidence, Regulatory divergence between regions.
- IdeaOne international registry, not one per country, for conditional approvals
Shares Weak real-world evidence and registries, Real-world evidence, Regulatory divergence between regions.
- IdeaRequire post-approval evidence in patients over 75 and update labels accordingly
Shares Weak real-world evidence and registries, Real-world evidence, Regulatory divergence between regions.
- IdeaProvisional prices for surrogate-endpoint approvals, reset when survival data arrive
Shares Accelerated approval, Overall survival (OS), Regulatory divergence between regions.
- IdeaCheap long-term survival follow-up by linking trial participants to registries
Shares Accelerated approval, Overall survival (OS), Weak real-world evidence and registries.
- IdeaA public rulebook for when an external or synthetic control arm is acceptable
Shares Accelerated approval, Weak real-world evidence and registries, Real-world evidence.
- IdeaSponsors deposit the confirmatory trial budget in escrow at accelerated approval
Shares Accelerated approval, Regulatory divergence between regions.
- IdeaLinked prescribing and outcome data to find drug interactions with cancer therapy
Shares Weak real-world evidence and registries, Real-world evidence.