FDA Regulatory Information

Guidance

• Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Draft Guidance for Industry, September 2021
• FDA Guidance on Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic: Guidance for Industry, Investigators, and Institutional Review Boards, August 2021
• Investigational COVID-19 Convalescent Plasma; Guidance for Industry, February 2021
• Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Guidance for Industry, January 2020
• Human Gene Therapy for Retinal Disorders; Guidance for Industry, January 2020
• Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry, January 2020
• Human Gene Therapy for Rare Diseases; Guidance for Industry, January 2020
• Human Gene Therapy for Hemophilia; Guidance for Industry, January 2020
• Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry, January 2020
• Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, February 2019
• Evaluation of Devices Used with Regenerative Medicine Advanced Therapies, February 2019
• Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products, March 2019
• Guidance for Industry: Standards Development and the Use of Standards in Regulatory Submissions Reviewed in the Center for Biologics Evaluation and Research, March 2019
• Guidance for Industry: Donor Screening Recommendations to Reduce the Risk of Transmission of Zika Virus by Human Cells, Tissues, and Cellular and Tissue-Based Products, May 2018
• Guidance for Industry: Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception, November 2017
• Guidance for Industry: Deviation Reporting for Human Cells, Tissues, and Cellular and Tissue-Based Products Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR Part 1271, September 2017
• Guidance for Industry: 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), September 2016, Corrected May 2017
• Guidance for Industry: 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, August 2016
• Guidance for Industry: Use of Donor Screening Tests to Test Donors of Human Cells, Tissues and Cellular and Tissue-Based Products for Infection with Treponema pallidum (Syphilis), September 2015
• Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products, June 2015
• Guidance for Industry and FDA Staff: 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, March 2014
• 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, March 2014
• Guidance for Industry and Investigators: Safety Reporting Requirements for INDs and BA/BE Studies, December 2012
• Guidance for Industry and Investigators: Safety Reporting Requirements for INDs and BA/BE Studies - Small Entity Compliance Guide, December 2012
• Guidance for Industry: Current Good Tissue Practice (CGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps), December 2011
• Guidance for Industry and FDA Staff: Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications, October 20, 2009
• Guidance for Industry: Certain Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Recovered from Donors Who Were Tested for Communicable Diseases Using Pooled Specimens or Diagnostic Tests, April 2008
• Guidance for Industry: Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps), August 2007

Reporting

• Instructions for Biological Product Deviation Reporting Using the eBPDR System
• Procedures for Handling Adverse Reaction Reports Related to "361" Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps), March 26, 2008

FDA Cellular Therapy Liaison Meetings (CTLM)

Since 2004, ISCT has been the host organization leading over 20 invited stakeholder organizations in an annual face-to-face meeting with the FDA. These closed meetings enable the cell and gene therapy community, and industry to inform the FDA of specific concerns, challenges and recent developments to advance the regulatory field.

View meeting summaries

Contact

• FDA Biological Product Deviation Contacts
• Manufacturers Assistance and Technical Training Branch in the Center for Biologics Evaluation and Research