Regulatory Focus™ > News Articles > FDA Looks to Harmonize Standards for Cellular, Regenerative Therapies

FDA Looks to Harmonize Standards for Cellular, Regenerative Therapies

Posted 19 July 2013 | By Alexander Gaffney, RAC

The US Food and Drug Administration (FDA) has announced it will soon hold a workshop with the intent of discussing the development of "synergized" standards for cellular therapies and regenerative medicine products, saying current standards run the risk of becoming disharmonized.


Regenerative medicine is, as its name implies, the emerging scientific field associated with the idea that cells, tissue, organs and even entire body parts can be regenerated, restored or regrown in order to provide therapeutic effects to a patient. For example, a patient with a failing liver would stand to benefit by having a replacement one regrown using his own stem cells, while a patient who has experienced major skin burns would benefit from the regrowth of her skin cells.

By contrast, cellular therapies involve introducing modified cells into the body in the hopes that they can treat a disease, particularly hereditary ones (as opposed to viral or bacterial conditions).

Both are extraordinarily complicated, and involve unique regulatory considerations. For example, in a recent 3 July 2013 draft guidance document, FDA explained that cellular and gene therapy products carry risks that are often unique to the individual. Because it is generally impossible to run assays on the products (which generally form a first line of defense against safety issues in chemical drugs), FDA advises that the therapy be used as a last-resort option, and that trials introduce the therapy to just a few patients at a time.

Other considerations include the short shelf life of the biological materials, the long-term effects of the implanted tissues and cells, and transportation challenges that can disrupt recommended therapy and clinical trials.

Meeting Ambitions

The workshop, announced on 19 July 2013 in a Federal Register notice, is being hosted by FDA's Center for Biologics Evaluation and Research (CBER), which said it hopes to attract the attention of a broad range of stakeholders.

That attention, it added, will be focused on an analysis of the past, current and future efforts of standards development with respect to cellular therapies and regenerative medicine products, and how stakeholders can become more involved in that process.

While ongoing efforts are extensive, FDA said that "relatively little" is being done to coordinate those efforts, potentially leading to conflicting or disharmonized regulatory standards.

FDA added that it hopes to provide an opportunity to industry to discuss "Areas of high interest for current or future standards development […] and to explore ways to minimize redundancy and maximize collaboration."

The meeting will be held on 7 October 2013 at FDA's White Oak campus in Silver Spring, MD and will also be webcast through its website.

Federal Register Meeting Notice

Tags: Meeting, workshop

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