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The primary endpoint is the single measurement a trial is designed and sized to test, declared in advance; if it is met, the trial is 'positive'. Secondary endpoints are additional measures that can support but not rescue a trial that missed its primary.
Common oncology primary endpoints are overall survival, progression-free survival (often by blinded central review), event-free or disease-free survival, objective response rate, and pathological complete response; dual or co-primary endpoints (PFS and OS) split the type I error between them with hierarchical testing. A trial that misses its primary but shows a survival trend is 'technically negative' (BILCAP, JAVELIN Ovarian) and regulators rarely approve on secondary endpoints alone. Endpoint choice shapes approval speed (surrogates are faster) and interpretation, and changing the primary endpoint mid-trial, or reporting a secondary as if it were primary, are recognised warning signs.
Shares Data maturity (immature vs mature survival data), Statistical significance (P values, alpha, multiplicity), Futility analysis (stopped for futility), Group sequential design, stopping rules and alpha spending.
Shares Pre-specified vs post-hoc analysis, Statistical significance (P values, alpha, multiplicity), Group sequential design, stopping rules and alpha spending, Endpoint.
Shares Data maturity (immature vs mature survival data), Interim analysis, readout and data cut-off, Progression-free survival (PFS), Overall survival (OS).
Shares Futility analysis (stopped for futility), Group sequential design, stopping rules and alpha spending, Trial protocol and statistical analysis plan, Interim analysis, readout and data cut-off.
Shares Pre-specified vs post-hoc analysis, Statistical significance (P values, alpha, multiplicity), Futility analysis (stopped for futility), Trial registration and results reporting (ClinicalTrials.gov, EU CTR).
Shares Futility analysis (stopped for futility), Group sequential design, stopping rules and alpha spending, Interim analysis, readout and data cut-off.
Shares Endpoint, Surrogate endpoint, Event-free / disease-free survival (EFS, DFS, iDFS, RFS), Objective response rate (ORR).
Shares Group sequential design, stopping rules and alpha spending, Surrogate endpoint, Progression-free survival (PFS), Overall survival (OS).