In a press release issued on April 3rd, FDA reiterated its warning to stem cell clinics (and implicitly to other HCT/P facilities that are benefiting from enforcement discretion), asserting that the agency has stepped up oversight over such facilities, and will be ramping up even more after the 36 months of enforcement discretion end in November 2020:
Over the past year, we have sent 45 manufacturers and health care providers regulatory correspondence, including warning letters, and we have two court cases pending. We’re committed to taking appropriate steps to address those that jeopardize the health of the people we are sworn to protect.
Today, we’re continuing these efforts. The agency issued a warning letter to Cord for Life, Inc., located in Altamonte Springs, Florida, for manufacturing unapproved umbilical cord blood products in violation of current good manufacturing practice (CGMP) requirements, including failing to validate processes to prevent bacterial contamination, raising potential significant safety concerns that put patients at risk. In addition, we issued 20 letters today to separate manufacturers and health care providers across the country who may be offering unapproved stem cell products, reiterating the FDA’s compliance and enforcement policy.
In addition, the agency expressed frustration that some in the stem cell industry continue to assert that autologous stem cell procedures (i.e., procedures that take cells or tissue from a person’s body and re-administer them to the same person, usually after some manipulation, and for a different intended use) are not subject to FDA regulation.
There’s a false premise being asserted by some in the field that a product derived from a person’s own body and then manipulated and reinserted for another use different from the one it played in its original location is not subject to FDA regulation just because it originated from the person it was given back to. But stem cell products can create unique and serious risks depending on how they’re manipulated once they’re taken from the body and how they are used once they’re reinserted in the body.
Interestingly, while the agency had issued a similar press release in December 2018 emphasizing that they were “discouraged” by the overall lack of manufacturers wanting to interact with the agency during the 36 months of enforcement discretion, in the current press release FDA states that the industry has made “modest progress” in coming into compliance, though much more work needs to be done.
Finally, as enforcement discretion comes to an end, in addition to the stick of possible regulatory and enforcement action, the agency also held out the carrot of possible relief for firms that undertake well-designed investigational studies with the intent of collecting information to more clearly identify the safety and benefits of their products.
As we come up on the end of this period during which the FDA intends to exercise enforcement discretion, we may take additional steps to delineate an efficient development path for promising products that pose lower risk to patients and that are being developed by sponsors who’ve engaged the regulatory process in a responsible manner by filing INDs. These would be cases where the sponsors have undertaken or are in the process of undertaking well-designed investigational studies with the intent of collecting information to more clearly identify the safety and benefits of their products… In addition, during the next year, the agency will explore whether there are additional ways that it can assist legitimate developers of stem cell products to come into compliance with its regulations. [Emphasis added]
What might these additional steps to delineate an efficient development path be?
In what ways might the agency assist legitimate developers to come into compliance?
We have some thoughts. Stay tuned…