title: A drug's life: the pathway to drug approval.
abstract: In the United States, drugs and medical devices are regulated by the US Food and Drug Administration (FDA). A drug must undergo rigorous testing prior to marketing to and medical use by the general public. The FDA grants marketing approval for drug products based on a comprehensive review of safety and efficacy data. This review article explains the history behind the establishment of the FDA and examines the historical legislation and approval processes for drugs, specifically in the fields of medical oncology and hematology. The agents imatinib (Gleevec, Novartis) and decitabine (Dacogen, Eisai) are used to illustrate both the current FDA regulatory process-specifically the orphan drug designation and accelerated approval process-and why decitabine failed to gain an indication for acute myeloid leukemia. The purpose and construct of the Oncologic Drugs Advisory Committee are also discussed, along with examples of 2 renal cell cancer drugs-axitinib (Inlyta, Pfizer) and tivozanib-that used progression-free survival as an endpoint. Regulatory approval of oncology drugs is the cornerstone of the development of new treatment agents and modalities, which lead to improvements in the standard of cancer care. The future landscape of drug development and regulatory approval will be influenced by the new breakthrough therapy designation, and choice of drug will be guided by genomic insights.
authors: Fellow in hematology and medical oncology at the Cleveland Clinic Taussig Cancer Institute in Cleveland, Ohio.
authors: Michael K
authors: MK
authors: Keng
authors: Clinical specialist in the Department of Pharmacy at the Cleveland Clinic in Cleveland, Ohio.
authors: Candice M
authors: CM
authors: Wenzell
authors: Professor of medicine and the director of the Leukemia Program at the Cleveland Clinic Taussig Cancer Institute in Cleveland, Ohio.
authors: Mikkael A
authors: MA
authors: Sekeres
mesh_terms: D017277:Drug Approval / Q000331:legislation & jurisprudence / Q000379:methods / Q000458:organization & administration / Q000706:statistics & numerical data; D006801:Humans; D014481:United States; D014486:United States Food and Drug Administration