CellBased Medicinal Products for Global （细胞对全球医药产品）.pdf

Cell-Based Medicinal Products for Global Market: FDA Perspectives CAT-ESGCT Workshop ESGCT Annual Meeting, Brighton, UK October 27, 2011 Steven S. Oh, Ph.D Office of Cellular, Tissue and Gene Therapies Center for Biologics Evaluation and Research US Food and Drug Administration Outline  U.S. regulatory framework  FDA and Office of Cellular, Tissue and Gene Therapies (OCTGT)  Regulatory approaches to cell and gene therapy products  Regulatory approaches to biologic-device combination products  Product development and clinical studies; GMP expectations  FDA experience with cell and gene therapy products  FDA-EMA interactions  FDA/OCTGT resources 2 Human Medical Products Regulated in the U.S  Drugs - Definition: 21 USC 201(g)  Biologics - Definition: 42 USC 351(i)  Medical Devices - Definition: 21 USC 201(h)  Combination Products - Definition: 21 CFR 3.2 (e)(1) 3 U.S. Regulatory Framework: 3-Tiered System  Statutes (Laws): Passed by Congress and signed by the President  Food, Drug Cosmetic Act (FDC Act)  Public Health Service Act (PHS Act)  Regulations (details of the law): Written by FDA and approved by the Executive Branch  21 CFR (Code of Federal Regulations)  Guidance (FDA’s interpretation of the Regulations): Written and approved within FDA  Advice non-binding on FDA or sponsor 4 U.S. Paradigm for Medical Product Regulation  Centralized authority for oversight  FDA oversees the entire lifecycle of a medical pro