octgt guidance document update rachael anatol, ph.d. associate director for policy-new legislation...

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OCTGT Draft Guidance Documents Published Since Last Update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• Draft Guidance for Industry: Use of Donor Screening Tests to Test Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) for Infection with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

9

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

Follow us on Twitter https://www.twitter.com/fdacber