Confirmatory trial
The randomised trial a company must run after an accelerated approval to prove the drug really helps patients live longer or better. If it fails or never finishes, the approval is supposed to be withdrawn.
Accelerated approvals rest on surrogate endpoints (response rate, pCR, MRD) from single-arm or early trials; the confirmatory trial tests overall survival, progression-free survival or another clinical benefit against a control. The 2022 FDORA law lets the FDA require the confirmatory trial to be under way at approval and streamlines withdrawal when it fails. Notable failures and withdrawals include PI3K inhibitors in lymphoma, several checkpoint-inhibitor indications ('dangling approvals' reviewed by ODAC in 2021), and melphalan flufenamide. Regulators increasingly scrutinise whether the confirmatory population matches the accelerated one (STARGLO complete response letter).
Pages like this
not linked directly; found by shared links- TermODAC (Oncologic Drugs Advisory Committee)
Shares Complete response letter (CRL), Indication withdrawal.
- TermRegulators beyond the FDA (EMA/CHMP, MHRA, PMDA, NMPA, TGA)
Shares Complete response letter (CRL), Conditional marketing authorisation (EU).
- TermBLA / NDA / MAA (marketing applications) and PDUFA dates
Shares Complete response letter (CRL), Pivotal (registrational) trial.
- TermSingle-arm trial
Shares Pivotal (registrational) trial, Accelerated approval.
- TermPrimary, secondary and co-primary endpoints
- TermLabel, indication and label expansion
Shares Full (traditional, regular) approval, Accelerated approval.