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Compliance In Focus
Posted by John Lehmann on Mon, Dec 3, 2012

New FDA Guidance: Regarding Cell & Gene Therapy

Not too long ago we posted a blog on cell therapy trials. This is an emerging field in clinicalNew FDA Guidance Cell Gene Therapy research and one we are very excited about. While the FDA is still in the process of building a foundation of the cell therapy industry, there exists a need for this type of medical development to meet the needs of patients. Before any of these life-saving innovations can reach the public, they must meet the requirements for being proven safe and effective in clinical research- and have success in clinical trials. Looks like FDA is taking steps in this direction.

FDA has announced the availability of a new draft guidance regarding this new frontier of clinical research. The draft guidance document is officially titled: “Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products.” The guidance covers the following product areas:

  • Cellular Therapy
  • Gene Therapy
  • Therapeutic Vaccination
  • Xenotransplantation

According to the news-release on Federal Register, “The draft guidance document provides sponsors and individuals that design and implement preclinical studies with recommendations on the substance and scope of preclinical information needed to support clinical trials for investigational products regulated by the Center for Biologics Research and Evaluation (CBER), Office of Cellular, Tissue, and Gene Therapies (OCTGT).”

 The draft guidance offers insight and recommendations including:

  • Preclinical Study Considerations
    • Preclinical program objects
    • Recommendations for general preclinical program design
    • Proof-of-concept studies
  • Recommendations for Investigational Cell Therapy Products
    • Overall Study design
    • Safety
    • Biological responsiveness
  • Recommendations for Investigational Gene Therapy Products
    • Animal species/model(s)
    • Overall Safety considerations
    • Transgene considerations
  • And much more!

FDA further explained that “because the composition of the products, as well as their mechanism of action, vary so greatly from chemical and biological drugs, standardized toxicology testing and other preclinical testing methods are often "not appropriate" for evaluating the products.”

What are your thoughts in the new draft guidance? Do you think this is a step in the right direction for FDA to clarify these unique product types? Comment below.

Photo Credit: http://www.flickr.com/photos/cihr_irsc/

Register for Imaging in Clinical Trials Webinar December 2012

Topics: FDA Guidance, Cell Therapy, Gene Therapy, Clinical Research


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