FDA steps up oversight of cell therapies
- The Food and Drug Administration is stepping up its enforcement of the existing rules covering stem cell therapies in order to protect patients from unproven or potentially dangerous treatments.
- Under "a comprehensive policy framework" that will be put into place next quarter, individual providers and companies will be able to gain approval for products and procedures through a lower-cost and more streamlined process.
- A new working group will pursue unscrupulous clinics through legally enforceable means in order to protect public health.
The profile of cell therapies and regenerative medicine is growing. These approaches have potential to lead to tissue repair and major improvements in health in areas such as cancer, Parkinson's disease and diabetes, and even have the potential to be curative. This opens up a field that is tremendously exciting, but genuine research and development can easily be hampered by bad actors.
Patients desperately seeking solutions for potentially fatal diseases can get to a point that they will clutch at any solution offered, even if the hope is unlikely or untested. FDA Commissioner Scott Gottlieb's statement shows an awareness of the potential for "unscrupulous actors" in the field, and their impact both on vulnerable people and on the reputation of sound researchers and companies.
"These dishonest actors exploit the sincere reports of the significant clinical potential of properly developed products as a way of deceiving patients and preying on the optimism of patients facing bad illnesses. This puts the entire field at risk. Products that are reliably and carefully developed will be harder to advance if bad actors are able to make hollow claims and market unsafe science. To make sure the agency is separating the promise from the unscrupulous hype, we are stepping up our enforcement activity in this area," said Gottlieb.
This week alone, the FDA posted a warning letter to the Stem Cell Clinic of Sunrise, Florida, for marketing stem cell products without FDA approval and for significant deviations from current good manufacturing practice requirements. It also seized five vials of live vaccinia virus – reserved for use by people at high risk of smallpox – belonging to StemImmune, in order to prevent it being used at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California.
Gottlieb is also working to improve access to genuine therapies, stating that he is " committed to doing all we can to help bring to patients more quickly innovative, scientifically proven regenerative cell therapies."
To support this, the FDA is developing a framework that aims to accelerate the development of genuine approaches, to be launched this fall. This will make it clearer whether products and protocols fall under the FDA's authority, and if so, how they can be evaluated for safety and effectiveness in an efficient manner.
"We will be developing a novel approach to FDA approval that we believe will allow very small product developers to gain all the benefits of FDA approval through a process that is minimally burdensome and less costly. We’re mindful of the significant promise offered by regenerative medicine, the cost of innovation in this industry, the small companies engaging in these enterprises, and the difficulty of doing FDA registration trials in this field. Our framework will take measure of all of these challenges," said Gottlieb.
The FDA is also working on a regenerative medicine advanced therapy (RMAT) designation, which will give regenerative cell therapies, including certain gene therapies that permanently alter tissue, access to expedited programs.
- Food and Drug Administration Press release
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