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Investigational Cellular Therapies

Overview

The FDA requires clinical studies for investigational cellular therapies regulated as biological products to be conducted under an Investigational New Drug (IND) application. An IND allows the investigational product to be shipped across state lines with the intent of collecting safety and efficacy data in humans. For Phase 1 clinical studies, the FDA's review of IND applications primarily focuses on safety data. For Phase 2 and 3 studies, the FDA reviews safety and effectiveness data of an investigational cellular therapy, and also the rights of human subjects enrolled in the study. A listing of the content requirements for an IND application is below. 

Content Requirement for an IND Application 

 
  • Cover sheet (Form FDA 1571)
  • Table of contents
  • Introductory statement and general investigational plan
  • Investigator's brochure
  • Protocol(s)
  • Chemistry, manufacturing, and control information
  • Pharmacology and toxicology information
  • Previous human experience with the investigational drug
  • Additional information (e.g., dependence and abuse potential, radioactive products)
  • Relevant information as requested by the FDA
  • Certification of Compliance with Requirements of ClinicalTrials.gov Data Bank (Form FDA 3674)
  • Statement of Investigator (Form FDA 1572)

An IND application goes into effect 30 days after it is received by the FDA, unless otherwise notified. Once the IND has gone into effect (also referred to as an active IND) and the clinical study protocol has been approved by an Institutional Review Board or ethics committee, the sponsor may proceed with the proposed clinical study; however, the sponsor still is responsible for a variety of IND-related activities such as safety reporting. Sponsors are responsible for promptly reviewing relevant safety information during the course of clinical development. This includes foreign or domestic safety information; information derived from any clinical or epidemiological investigations, animal or in vitro studies; reports in the scientific literature, and unpublished scientific papers; reports from foreign regulatory authorities; and reports of foreign commercial marketing experience for products that are not marketed in the United States. 

Sponsors also must submit a brief progress report of the ongoing and recently completed preclinical and clinical investigations under the IND to the FDA annually (commonly referred to as an Annual Report), within 60 days of the anniversary date that the IND went into effect. An IND Annual Report should include the information listed in 21 CFR 312.33. During clinical development of cellular therapies, sponsors also may need to submit a protocol amendment to the IND application to ensure that the clinical investigations are conducted according to protocols included in the application. Additionally, sponsors may need to report pertinent data as an information amendment when it is beyond the scope of a protocol amendment, IND safety report or annual report.

Effective Oct. 20, 2011, minimally manipulated hematopoietic progenitor cells (HPC) sourced from placental/umbilical cord blood for unrelated allogeneic use require a BLA prior to being marketed, or must be distributed under an IND. The FDA has published a guidance document titled "Guidance for Industry and FDA Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications," March 2014, addressing the regulatory requirements for IND applications for HPC, Cord Blood products.