1. OCTGT Guidance Document Update Rachael Anatol, Ph.D. Associate Director for Policy-New Legislation February 26, 2014

2. Outline Good Guidance Practices Guidance basics Guidances issued by OCTGT since 2009 OCTGT 2014 Guidance Agenda

3. Good Guidance Practices (GGPs) FDA’s policies and procedures for developing, issuing, and using guidance documents Require FDA to publish yearly guidance agenda Call for public participation

4. Guidance Document Basics Documents prepared for FDA, applicants/sponsors, and the public that describe FDA’s interpretation of or policy on a regulatory issue First means of communication to a broad audience Not legally binding

5. Guidance Document Topics Design, production, labeling, promotion, manufacturing, and testing of regulated products Processing, content, and evaluation or approval of submissions Inspection and enforcement policies

6. OCTGT Draft Guidance Documents Published Since Last Update Use of Nucleic Acid Tests to Reduce the Risk of Transmission of West Nile Virus from Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) ; 2013 Draft Guidance for Industry: Use of Donor Screening Tests to Test Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) for Infection with Treponema pallidum (Syphilis) ; 2013 Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; 2013 Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System; 2013 Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System; 2013

7. OCTGT Final Guidance Documents Published Since Last Update Preclinical Assessment of Investigational Cellular and Gene Therapy Products; 2013 Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage; 2011 Clinical Considerations for Therapeutic Cancer Vaccines; 2011 Potency Tests for Cellular and Gene Therapy Products; 2011 Current Good Tissue Practice (CGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); 2011 Class II Special Controls Guidance Document: Cord Blood Processing System and Storage Container; 2011 Cellular Therapy for Cardiac Disease ; 2010 Considerations for Allogeneic Pancreatic Islet Cell Products ; 2009

8. OCTGT Guidances on 2014 CBER Guidance Agenda Draft Guidance for Industry: Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products Draft Guidance for Industry: Minimal Manipulation of Human Cells, Tissues, and Cellular and Tissue Based Products Final Guidance for Industry: Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System Final Guidance for Industry: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System

9. OCTGT Contact Information Rachael Anatol: Rachael.Strong@fda.hhs.govRachael.Strong@fda.hhs.gov Regulatory Questions: Contact the Regulatory Management Staff in OCTGT at CBEROCTGTRMS@fda.hhs.gov or Lori.Tull@fda.hhs.gov or by calling (301) 827-6536 CBEROCTGTRMS@fda.hhs.gov Lori.Tull@fda.hhs.gov OCTGT Learn Webinar Series: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ ucm232821.htm http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ ucm232821.htm 9

10. Public Access to CBER CBER website: http://www.fda.gov/BiologicsBloodVaccines/default.htmhttp://www.fda.gov/BiologicsBloodVaccines/default.htm. Cellular and gene therapy guidances: http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/defa ult.htm Tissue guidances: http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/default.htm All biologics guidance and CBER 2014 guidance agenda: http://www.fda.gov/biologicsbloodVaccines/GuidanceComplianceRegulatoryInformation/guidances/default.htm Consumer Affairs Branch (CAB) Email: ocod@fda.hhs.gov ocod@fda.hhs.gov Phone: 301-827-3821 Manufacturers Assistance & Technical Training Branch (MATTB) Email: industry.biologics@fda.gov industry.biologics@fda.gov Phone: 301-827-4081 Follow us on Twitter https://www.twitter.com/fdacber