FDA Update. Scott A. Brubaker, CTBS Director, Division of Human Tissues Office of Tissues and Advanced Therapies CBER/FDA
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1 FDA Update Scott A. Brubaker, CTBS Director, Division of Human Tissues Office of Tissues and Advanced Therapies CBER/FDA 13 th Annual FDA and the Changing Paradigm for HCT/P Regulation February 13, 2017 Alexandria, Virginia
2 Outline OTAT Update Part 15 Hearing Public Workshops Rule Making Guidance Documents (new logo) 2
3 FDA Organization CBER (Center for Biologics Evaluation and Research) vaccines, blood products, allergenic products, human tissue/tissue products for transplantation, cells, gene therapy, some devices (related to blood and HCT/Ps) CDER (Center for Drug Evaluation and Research) drugs, some biological products CDRH (Center for Devices and Radiological Health) most devices (including for treatment, implants, diagnostics) 3
4 Effective October 16, 2016 Name change: Office of Tissues and Advanced Therapies (OTAT) All of Office of Cellular, Tissue, and Gene Therapies (OCTGT) is now part of OTAT Transfer of some products from Office of Blood Research and Review (OBRR) to OTAT Two Divisions in OBRR have transferred to OTAT Division of Hematology Clinical Review Division of Hematology Research and Review Products for transfusion remain in OBRR
5 OTAT Products Stem cell and stem cell-derived products Hematopoietic, mesenchymal, cord blood, embryonic, ipscs Somatic cell therapies Pancreatic islets, chondrocytes, myoblasts, keratinocytes, hepatocytes Therapeutic vaccines and other antigen-specific active immunotherapies Cancer vaccines and immunotherapies, such as dendritic cells, lymphocyte-based therapies, cancer cell-based therapies, peptides, proteins Non-infectious disease therapeutic vaccines, such as peptides, proteins, small molecules 5
6 OTAT Products, Cont d. Gene therapies Genetically modified cells Plasmids, viral vectors, bacterial vectors Purified and recombinant proteins for hematology (e.g., coagulation factors, thrombin, botulism antitoxin, diphtheria anti-toxin, fibrin sealants) Antivenins Some devices and combination products Devices with a cellular component Selection devices for the manufacture or delivery of cells 6
7 Office of Tissues and Advanced Therapies (OTAT) 7
8 Part 15 Public Hearing Request for Comments - Draft Guidances Relating to the Regulation of Human Cells, Tissues, or Cellular or Tissue- Based Products ( & ) Convened two-day event with over 90 presenters representing industry, patient advocacy, academia, and individuals; 400 attended in person or by webcast Obtained stakeholder input on draft guidance regarding regulation of human cell and tissue-based products: minimal manipulation; homologous use; same surgical procedure exception; and HCT/Ps from adipose tissue FDA is currently considering comments 8
9 CBER Public Workshops Scientific Evidence in the Development of Human Cells, Tissues, and Cellular and Tissue-Based Products Subject to Premarket Approval (9-8-16) Discussed scientific considerations and challenges in development of human cells, tissues, and cellular and tissue-based products (HCT/Ps) subject to premarket approval, including stem cell-based products 9
10 CBER Public Workshops, Cont d. Identification and Characterization of the Infectious Disease Risks of Human Cells, Tissues, and Cellular and Tissue-based Products ( & ) The purpose of the public workshop was to have a scientific discussion of the current methods available for identifying and characterizing infectious disease risks associated with human cells, tissues, and cellular and tissue-based products (HCT/Ps). 10
11 RULE MAKING
12 Final Rules Revisions to Exceptions Applicable to Certain Human Cells, Tissues, and Cellular and Tissue-Based Products; Final Rule, June 22, 2016 Requirements for Foreign and Domestic Establishment Registration and Listing for Human Drugs, Including Drugs That Are Regulated Under a Biologics License Application, and Animal Drugs; Final Rule, August 31,
13 GUIDANCE DOCUMENTS
14 Guidance Documents Donor Screening Recommendations to Reduce the Risk of Transmission of Zika Virus by Human Cells, Tissues, and Cellular and Tissue-Based Products; Guidance for Industry, March 2016 Investigating and Reporting Adverse Reactions Related to Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Regulated Solely under Section 361 of the Public Health Service Act and 21 CFR Part 1271, March 2016 Use of Nucleic Acid Tests to Reduce the Risk of Transmission of Hepatitis B Virus from Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products; Guidance for Industry, August
15 Guidance Documents, Cont d. Use of Nucleic Acid Tests to Reduce the Risk of Transmission of West Nile Virus from Living Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Guidance for Industry, September 2016 Revised Recommendations for Determining Eligibility of Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products Who Have Received Human-Derived Clotting Factor Concentrates; Guidance for Industry, November
16 OTAT Contact Information Scott A. Brubaker, CTBS Regulatory Questions Contact the Regulatory Management Staff at: OTAT Learn Webinar Series 16
17 Public Access to CBER CBER website or Consumer Affairs Branch (CAB) Manufacturers Assistance and Technical Training Branch (MATTB) Follow us on Twitter 17
18
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