The ISSCR commends the U.S. Food and Drug Administration (FDA) for reaffirming its regulatory authority over stem cell biologics and clarifying the criteria that determine which cell-based products fall under its oversight. These guidance documents will provide greater transparency for patients, medical professionals, and law enforcement authorities, about the legal status of stem cell treatments in the U.S.
The finalized guidance documents, released 16 November, clarify exceptions to regulation that clinics selling unproven stem cell interventions have exploited in an attempt to elude regulatory authority. They provide explicit guidance on key criteria that determine whether a stem cell product falls under FDA jurisdiction. The ISSCR agrees with the FDA that unscrupulous clinics are selling to unsuspecting patients unproven stem cell therapies, whose safety and effectiveness have not been established in clinical trials. These unproven stem cell therapies often have little basis in science or medicine and prey upon the hopes of desperate patients.
“This new FDA effort closes significant regulatory loopholes that have been exploited by businesses promoting unproven treatments that have not been proven safe nor effective for patient use,” said ISSCR president Hans Clevers. “It puts clinics on notice that the agency is paying attention and asserting its authority,” he said. Clevers expressed appreciation for the FDA’s determination to move immediately on clinics employing the riskiest unproven uses of stem cells.
Clevers also noted that the number of clinics providing unproven stem cell interventions has grown rapidly in recent years, including in the U.S., and a sustained commitment to enforce FDA regulation against all clinics that market cell therapies will be required. Additional resources are needed to ensure that the FDA is able to enforce these new rules effectively for the protection of patients.
The ISSCR also supports efforts such as legislation recently signed into law in California to inform patients when stem cell interventions have not yet been approved by the FDA. While stem cell research has enormous potential to yield new therapies for other diseases, currently the only known effective therapies apply to a small subset of diseases, mostly involving the blood and immune systems.
The finalized guidance documents are published on the FDA website: FDA Announces Comprehensive Regenerative Medicine Policy Framework.