title: End points and United States Food and Drug Administration approval of oncology drugs.
abstract: OBJECTIVE
  To summarize the end points used by the United States Food and Drug Administration (FDA) to approve new cancer drug applications over the last 13 years.
  
  
  METHODS
  The FDA granted marketing approval to 71 oncology drug applications between January 1, 1990, and November 1, 2002. The end points used as the approval basis for each application are presented, and the rationale for each end point is discussed.
  
  
  RESULTS
  The FDA grants either regular marketing approval or accelerated marketing approval for oncology drug applications. Regular approval is based on end points that demonstrate that the drug provides a longer life, a better life, or a favorable effect on an established surrogate for a longer life or a better life. Accelerated approval (AA) is based on a surrogate end point that is less well established but that is reasonably likely to predict a longer or a better life. Tumor response was the approval basis in 26 of 57 regular approvals, supported by relief of tumor-specific symptoms in nine of these 26 regular approvals. Relief of tumor-specific symptoms provided critical support for approval in 13 of 57 regular approvals. Approval was based on tumor response in 12 of 14 AAs.
  
  
  CONCLUSIONS
  End points other than survival were the approval basis for 68% (39 of 57) of oncology drug marketing applications granted regular approval and for all 14 applications granted accelerated approval from January 1, 1990, to November 1, 2002.
authors.affiliation: Division of Oncology Drug Products (HFD-150), Center for Drug Evaluation and Research, United States Food and Drug Administration, Rockville, MD 20857, USA. Johnsonj@cder.fda.gov
authors.forename: John R
authors.initials: JR
authors.lastname: Johnson
authors.forename: Grant
authors.initials: G
authors.lastname: Williams
authors.forename: Richard
authors.initials: R
authors.lastname: Pazdur
mesh_terms: D000970:Antineoplastic Agents / Q000592:standards*; D017277:Drug Approval / Q000379:methods*; D023381:Endpoint Determination; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D000970:Antineoplastic Agents