OCTGT Guidance Document Update

Rachael Anatol, Ph.D.Associate Director for Policy-New

LegislationFebruary 26, 2014

Outline

• Good Guidance Practices

• Guidance basics

• Guidances issued by OCTGT since 2009

• OCTGT 2014 Guidance Agenda

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

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

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

OCTGT Draft Guidance Documents Published Since Last Update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• Draft Guidance for Industry: Use of Donor Screening Tests to Test Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) for Infection with Treponema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

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

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

OCTGT Contact Information

• Rachael Anatol: Rachael.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

• OCTGT Learn Webinar Series: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ucm232821.htm

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Public Access to CBERCBER website:http://www.fda.gov/BiologicsBloodVaccines/default.htm.

Cellular and gene therapy guidances:http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/

default.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

Phone: 301-827-3821

Manufacturers Assistance & Technical Training Branch (MATTB)Email: industry.biologics@fda.gov

Phone: 301-827-4081

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