The role of the U.S. Food and Drug Administration review process: clinical trial endpoints in oncology.
The U.S. Food and Drug Administration grants marketing approval for drug products based on a comprehensive review of safety and efficacy data. The clinical trial endpoints that have been used to support approval in the oncology setting have evolved over the last 30 years commensurate with an improved understanding of the natural history of cancers and the mechanisms of action of drugs. Overall survival is the gold standard for a registration trial designed to gain marketing approval; however, additional endpoints have been used in the approval of oncology drugs. Advantages of specific endpoints are discussed, including the accuracy of an endpoint's measurement and its relation to clinical benefit. Surrogate endpoints may be acceptable for "accelerated" approval, with a sponsor commitment to provide evidence of clinical benefit in a subsequent trial.
McKee
Amy E
AE
U.S. Food and Drug Administration, Silver Spring, Maryland 20993, USA. amy.mckee@fda.hhs.gov
Farrell
Ann T
AT
Pazdur
Richard
R
Woodcock
Janet
J
D002986:Clinical Trials as Topic; D017277:Drug Approval / Q000331:legislation & jurisprudence / Q000458:organization & administration*; D033161:Government Regulation; D006801:Humans; D017387:Investigational New Drug Application; D007880:Legislation, Drug / Q000458:organization & administration; D009369:Neoplasms / Q000628:therapy*; D011358:Product Surveillance, Postmarketing; D016019:Survival Analysis; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration