termsTerm

Pivotal (registrational) trial

aka pivotal, pivotal trial, pivotal study, pivotal phase 3, registrational, registrational trial, registrational study, registration trial, registration-enabling, registration-directed, approval-enabling, pivotal cohort, registrational cohort, supported approval, basis of approval, approval was based on

The trial (usually a randomised phase 3, sometimes a large phase 2) whose results are submitted to regulators as the main evidence for approval. When a company calls a trial 'registrational' it is signalling that this is the one that decides.

Regulators traditionally required two adequate and well-controlled trials, but in oncology one pivotal trial with confirmatory supporting evidence is the norm. Pivotal trials define the approved population, dose and comparator, so their design is negotiated with the FDA in advance (end-of-phase-2 meetings, special protocol assessment). Phase 2 single-arm cohorts serve as pivotal for accelerated approval in rare or refractory diseases. A 'registration-directed' or 'registration-enabling' phase 2 signals a company's intent to file on it. Pivotal trials are the units this site's evidence and trial pages are built around.

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Term
Complete response letter (CRL)
Shares Confirmatory trial, BLA / NDA / MAA (marketing applications) and PDUFA dates.
Term
Surrogate endpoint
Shares Confirmatory trial, Primary, secondary and co-primary endpoints.
Term
Objective response rate (ORR)
Shares Single-arm trial, Primary, secondary and co-primary endpoints.

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terms

BLA / NDA / MAA (marketing applications) and PDUFA dates Confirmatory trial Primary, secondary and co-primary endpoints Single-arm trial