title: Surrogate endpoints in randomized cardiovascular clinical trials.
abstract: Surrogate endpoints predict the occurrence and timing of a clinical endpoint of interest (CEI). Substitution of a surrogate endpoint for a CEI can dramatically reduce the time and cost necessary to complete a Phase III clinical trial. However, assurance that use of a surrogate endpoint will result in a correct conclusion regarding treatment effect on a CEI requires prior rigorous validation of the surrogate. Surrogate endpoints can also be of substantial use in Phase I and II studies to assess whether the intended therapeutic pathway is operative, thus providing assurance regarding the reasonableness of proceeding to a Phase III trial. This paper discusses the uses and validation of surrogate endpoints.
authors.affiliation: The National Heart, Lung, and Blood Institute, Bethesda, MD 20892, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Domanski
authors.forename: Stuart
authors.initials: S
authors.lastname: Pocock
authors.forename: Corine
authors.initials: C
authors.lastname: Bernaud
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Borer
authors.forename: Nancy
authors.initials: N
authors.lastname: Geller
authors.forename: James
authors.initials: J
authors.lastname: Revkin
authors.forename: Faiez
authors.initials: F
authors.lastname: Zannad
mesh_terms: D015415:Biomarkers; D002318:Cardiovascular Diseases / Q000188:drug therapy*; D023381:Endpoint Determination / Q000379:methods*; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D015203:Reproducibility of Results
chemical_list: D015415:Biomarkers