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In an interview with the Pink Sheet, Marks said the guidances on regenerative medicines make clear what the agency will be enforcing.

Cracking the whip against bad actors in the regenerative medicines arena, such as noncompliant stem cell clinics, has always been a priority for the Center for Biologics Evaluation and Research (CBER). But the center plans on taking its enforcement actions a step further in 2018, CBER Director Peter Marks says.


"Enforcement actions on stem cell clinics that were putting out products that could put patients at risk that were unapproved stem cell products was a priority," Marks said in an exclusive interview with the Pink Sheet. "I would say that this year, that priority is probably notched up somewhat.


"Unfortunately, we are aware of products out there in that space, and we will have to take appropriate actions to deal with those products through all of the regulatory tools and authorities that are given to us," Marks added. "Because unfortunately, the agency continues to receive on an ongoing basis information from patients who have been harmed by stem cell therapies, and it is a real concern, especially because we are not talking about information that has been given to us by people who have been harmed by approved stem cell therapies."


Along with the introduction of the Regenerative Medicine Advanced Therapy (RMAT) designation in 2017, FDA released a slew of guidances in November related to regenerative medicines. Two of these were final guidances clarifying the agency's interpretation of the criteria for determining whether cell- and tissue-based products are subject to premarket review.


Specifically, one of the guidances clarifies when cell and tissue-based products qualify for an exception from the requirements of 21 CFR Part 1271 if they are removed from and implanted into the same individual within the same surgical procedure and remain in their original form. The other guidance clarifies how FDA interprets the regulatory definitions of “minimal manipulation” and “homologous use.”


The agency has said that it will apply 36 months of enforcement discretion to give manufacturers a chance to come into compliance, although it will not do so for products that potentially pose a significant safety concern.


"What I can say, is that now that the guidances are out, it is very clear to people what we are going to be enforcing," Marks said. "And I think what you'll see over the course of the coming months will illustrate that with that clarity in there, we expect the field to come into compliance, and if they don't come into compliance, we will take appropriate enforcement action against any product that is deemed to present a potential risk to public health."


Marks, however, also noted that CBER will still have to prioritize which actors it goes after, in spite of the ramped up enforcement efforts.


"There always has to be [prioritization]," the CBER director said. "We admittedly don’t have the bandwidth to go after everything."


Marks has not shied away from his desire to go after bad actors in the regenerative medicine arena. At the Food and Drug Law Institute’s (FDLI) 2017 annual conference, Marks said the agency is aware of some "shady practices" going on in the cell therapy world. (Also see "Regenerative Medicines Provisions Of Cures Act A Top Priority For CBER" - Pink Sheet, 9 Jun, 2017.)


FDA exhibited signs of a higher level of enforcement in August with a series of enforcement actions against stem cell centers, which included the agency seizing five vials of a live vaccinia virus vaccine that was being administered to patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, Calif. (Also see "Crackdown On Bad Regenerative Medicine Could Benefit Gene Therapy" - Pink Sheet, 28 Aug, 2017.)


Mark Schwartz, director at the law firm Hyman, Phempa & McNamara and former deputy director of the Office of Compliance and Biologics Quality (OCBQ) in CBER, previously noted that the center rarely conducts seizures of stem cells, as well as products more broadly. (Also see "US FDA's Drug Seizures Seen As Most Significant Aspect Of Stem Cell Crackdown" - Pink Sheet, 6 Sep, 2017.)


The agency also released two additional draft guidances in November detailing development and review processes to spur the flow of legitimate regenerative medicine products to market.

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