title: From research to approved treatment: overcoming the obstacles.
abstract: The translation of promising early clinical results into approved therapy for cancer patients is often very difficult. To gain Food and Drug Administration approval, a new agent must be shown to be safe and effective, but the accepted endpoints for demonstrating efficacy, such as improved patient survival, are frequently inappropriate to clinical trials in oncological research. An examination of recent bases for granting FDA approval suggests that changes in research strategies that would allow, eg, the inclusion of improved quality of life and quantified and possibly descriptive data from phase II trials as demonstrations of an agent's efficacy would facilitate the licensing procedure. Additionally, the approaches used by insurance companies to control costs increasingly result in denial of coverage for patients in clinical trials. This problem may stem in part from a general lack of understanding of the nature of investigative therapy and the importance of clinical trials in delivering state-of-the-art care to cancer patients. It is up to all oncologists to promote a better understanding of the procedures and benefits of clinical research, particularly in regard to reimbursement policies.
authors.affiliation: Division of Cancer Treatment, National Cancer Institute, Bethesda, MD 20892.
authors.forename: R E
authors.initials: RE
authors.lastname: Wittes
mesh_terms: D006801:Humans; D009369:Neoplasms / Q000188:drug therapy*; D012106:Research; D014481:United States; D014486:United States Food and Drug Administration