title: Channeling postmarketing patient data into pharmaceutical regulatory systems.
abstract: A limited amount of data is typically available to support product license applications. That is further complicated by the need to make some medicines available to patients at key times, using expedited drug approval pathways. In addition, increasing immunogenicity concerns have been paralleled by a myriad of biotherapeutics entering development and/or receiving regulatory approval. Postmarketing patient safety is intrinsically dependent on the correct balance of economics, regulatory oversight and legal and enforcement issues. Here, we discuss the changing landscape of pharmacovigilance, with special emphasis on postmarketing commitments and requirements, megadata analysis, regulatory responsibilities and research opportunities. Challenges and possibilities are illustrated with therapeutic drugs approved for treatment of autoimmune diseases, diabetes, cancer, rare diseases and the resurgence of gene therapy.
authors.affiliation: CA Consultants, PO Box 3237, Princeton, NJ 08543, USA. Electronic address: mbarbosa@cacobio.com.
authors.forename: Maria D F S
authors.initials: MD
authors.lastname: Barbosa
authors.affiliation: City of Hope National Cancer Center, Department of Biostatistics, 1500 E. Duarte Road, Duarte, CA 91010, USA.
authors.forename: David D
authors.initials: DD
authors.lastname: Smith
mesh_terms: D000818:Animals; D000906:Antibodies / Q000276:immunology; D001688:Biological Products / Q000276:immunology / Q000627:therapeutic use; D015316:Genetic Therapy; D006801:Humans; D007880:Legislation, Drug; D017891:Pharmacoepidemiology; D011358:Product Surveillance, Postmarketing; D035583:Rare Diseases / Q000628:therapy
chemical_list: D000906:Antibodies; D001688:Biological Products