{"entity":{"id":"confirmatory-trial","kind":"term","name":"Confirmatory trial","aka":["confirmatory","confirmatory study","confirmatory phase 3","confirmatory evidence","post-marketing requirement","postmarketing requirement","post-approval trial","verify clinical benefit","conversion to full approval","dangling approval","dangling accelerated approval","confirmatory studies","post-marketing requirements"],"tldr":"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 If it fails or never finishes, the approval is supposed to be withdrawn.","summary":"Accelerated approvals rest on surrogate endpoints (response rate, pCR, MRD) from single-arm or early trials; the confirmatory trial tests overall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).","asOf":"2026-09-09","wikipedia":"https://en.wikipedia.org/wiki/Accelerated_approval_(FDA)","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Accelerated_approval_(FDA)"}],"tags":[],"related":["accelerated-approval","full-approval","approval-withdrawal","surrogate-endpoint","complete-response-letter","surrogate-validation","trial-lifecycle","trial-failure-modes"],"cancers":[],"sections":["drug-discovery"],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["announce","atlantis","keynote-522"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"category":"Trials"},"route":"/terms/confirmatory-trial/","neighbours":{"term":[{"id":"accelerated-approval","kind":"term","name":"Accelerated approval","route":"/terms/accelerated-approval/"},{"id":"complete-response-letter","kind":"term","name":"Complete response letter (CRL)","route":"/terms/complete-response-letter/"},{"id":"conditional-approval","kind":"term","name":"Conditional marketing authorisation (EU)","route":"/terms/conditional-approval/"},{"id":"fdora-2022","kind":"term","name":"FDORA 2022 accelerated approval reforms","route":"/terms/fdora-2022/"},{"id":"full-approval","kind":"term","name":"Full (traditional, regular) approval","route":"/terms/full-approval/"},{"id":"approval-withdrawal","kind":"term","name":"Indication withdrawal","route":"/terms/approval-withdrawal/"},{"id":"japan-conditional-early-approval","kind":"term","name":"Japan conditional early approval and time-limited approval","route":"/terms/japan-conditional-early-approval/"},{"id":"trial-phases","kind":"term","name":"Phase 1, 2 and 3 trials","route":"/terms/trial-phases/"},{"id":"pivotal-trial","kind":"term","name":"Pivotal (registrational) trial","route":"/terms/pivotal-trial/"},{"id":"surrogate-endpoint","kind":"term","name":"Surrogate endpoint","route":"/terms/surrogate-endpoint/"},{"id":"surrogate-validation","kind":"term","name":"Surrogate endpoint validation: which stand-ins have earned trust","route":"/terms/surrogate-validation/"},{"id":"trial-lifecycle","kind":"term","name":"Trial lifecycle: from protocol to label","route":"/terms/trial-lifecycle/"},{"id":"trial-failure-modes","kind":"term","name":"Why trials fail: underpowered, wrong endpoint, control arm drift, subgroup fishing, crossover","route":"/terms/trial-failure-modes/"}],"section":[{"id":"drug-discovery","kind":"section","name":"Drug Discovery Platforms","route":"/fronts/drug-discovery/"}],"trial":[{"id":"announce","kind":"trial","name":"ANNOUNCE","route":"/trials/announce/"},{"id":"atlantis","kind":"trial","name":"ATLANTIS","route":"/trials/atlantis/"},{"id":"keynote-522","kind":"trial","name":"KEYNOTE-522","route":"/trials/keynote-522/"}]}}