title: Regulatory approvals of pediatric oncology drugs: previous experience and new initiatives.
abstract: OBJECTIVE
  To review the Food and Drug Administration (FDA) experience with approvals of new drugs for pediatric oncology and to discuss new regulatory initiatives directed at pediatric oncology.
  
  
  METHODS
  A retrospective review of FDA archival documents and the published literature.
  
  
  RESULTS
  More than 100 drugs have been approved by the Division of Oncology Drug Products of the FDA for the treatment of malignancies. Only 15 have pediatric use information in their labeling, which is less than 50% of the drugs commonly used in the treatment of pediatric malignancies. In the past 20 years, there have been six submissions to the FDA for pediatric oncology indications. To illustrate principles of the approval process, each submission is discussed.
  
  
  CONCLUSIONS
  Potential reasons for a lack of New Drug Application submissions for pediatric oncology include the small pediatric oncology market compared with the adult oncology market and perceived barriers to performing studies in children. Reasons for failure to approve pediatric indications include small numbers of patients, lack of appropriate controls, and failure to demonstrate patient benefit. Approval criteria include the use of controlled trials, prospective data collection, and disease-appropriate end points. Regulatory initiatives to promote pediatric therapeutic development and product labeling are discussed.
authors.affiliation: Division of Oncology Drug Products, Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, MD 20852, USA. hirschfelds@cder.fda.gov
authors.forename: Steven
authors.initials: S
authors.lastname: Hirschfeld
authors.forename: Peter T C
authors.initials: PT
authors.lastname: Ho
authors.forename: Malcolm
authors.initials: M
authors.lastname: Smith
authors.forename: Richard
authors.initials: R
authors.lastname: Pazdur
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001374:Azacitidine / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D017277:Drug Approval; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy; D008727:Methotrexate / Q000627:therapeutic use; D009447:Neuroblastoma / Q000188:drug therapy; D012516:Osteosarcoma / Q000188:drug therapy; D010372:Pediatrics / Q000592:standards; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000188:drug therapy; D013713:Teniposide / Q000627:therapeutic use; D014481:United States; D014486:United States Food and Drug Administration; D014751:Vindesine / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D013713:Teniposide; D001374:Azacitidine; D014751:Vindesine; D008727:Methotrexate