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Innovative Regenerative Medicine Therapies – Patient Safety Comes First

two image collage, including photo of a research scientist in the lab looking into microscope and an electron micrograph of three stem cells
A research scientist looking into a microscope, and an electron micrograph of stem cells, color-enhanced for visual clarity.

By: Peter Marks, M.D., Director, Center for Biologics Evaluation and Research

The U.S. Food and Drug Administration continues to facilitate the development and availability of innovative medical products, such as regenerative medicine therapies, that have the potential to treat or even cure diseases or conditions for which few effective treatment options exist. For example, the agency has recently licensed (approved) its first product that received Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring our ongoing commitment to work with sponsors and manufacturers to bring these products to market.

Unapproved products marketed as regenerative medicine therapies may cause serious harm to patients. Cellular therapies, including stem-cell products, are often marketed by clinics as being safe and effective for the treatment of a wide range of diseases or conditions, even though they haven’t been adequately or appropriately studied in clinical trials. 

CBER Director, Peter Marks, M.D., Ph.D.
Peter Marks, M.D., Ph.D.

In 2017, the FDA issued guidance on the regulatory framework for regenerative medicine therapies and announced its intent to exercise enforcement discretion with respect to the FDA’s investigational new drug (IND) and premarket approval requirements for certain regenerative medicine products. This policy gave manufacturers three-and-a-half-years to determine the appropriate regulatory pathway for their products, and if an application is needed, ample time to prepare and submit the appropriate application to the FDA. 

Now that we have reached the end of the compliance and enforcement discretion policy period, we are once again reminding manufacturers, clinics, and health care practitioners and providers that the compliance and enforcement discretion policy for certain human cells, tissues, and cellular and tissue-based products (HCT/Ps), including regenerative medicine therapies, ended on May 31, 2021. If manufacturers continue to illegally market unapproved HCT/Ps, they do so at their own risk and may be subject to an enforcement action.

The FDA continues to receive consumer complaints and has warned consumers about unapproved regenerative medicine products and the unfounded claims made in advertisements and direct-to-patient marketing. Despite the FDA’s warnings that an IND may be required for these products, many entities still ignore such warnings and offer these unapproved and unproven products, with some consumers subsequently experiencing serious adverse effects.

The compliance and enforcement discretion policy was never intended to excuse the violations of manufacturers or health care providers who are offering unapproved regenerative medicine products that have the potential to put patients at significant risk. The policy did not apply to products that have been associated with reported safety concerns or have the potential to cause significant safety concerns to patients.

Indeed, while the policy was in place, the FDA took swift and aggressive action in the face of serious violations of the law, including some involving patient harm. Since November 2017, the FDA has pursued two enforcement actions for injunction against manufacturers of such violative HCT/Ps.  

The FDA prevailed in one of those cases, United States v. US Stem Cell Inc. et al., in June 2019, before the United States District Court for the Southern District of Florida. Earlier this week, the United States Court of Appeals for the Eleventh Circuit affirmed the lower court’s judgment. The US Stem Cell decision is a victory for public health and an endorsement of the FDA’s work to stop stem cell clinics that place patients at risk by marketing products that violate the law. 

The other case for injunction, United States v. Cell Surgical Network et al., is currently being litigated in the United States District Court for the Central District of California. A third enforcement action pursued by the FDA was resolved in March 2018. That case involved the seizure of vials of Vaccinia Virus Vaccine, Live, used to create an unapproved and dangerous stem cell product (a combination of excess amounts of live virus and stromal vascular fraction – a stem cell mixture derived from body fat).

The FDA also has taken numerous actions since the compliance and enforcement policy was announced. During this period, the agency issued 14 Warning Letters and 24 Untitled Letters involving violative HCT/Ps regulated under Section 351 of the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act and applicable FDA regulations. Additionally, since December 2018, the FDA has issued 400 letters to manufacturers and health care providers who may be offering violative stem cell or related products since December 2018.

The FDA reminds all stakeholders that the agency’s acceptance of an establishment registration and HCT/P listing does not constitute a determination that an establishment is in compliance with applicable rules and regulations or that the HCT/P is licensed or approved by the FDA. It is inappropriate and misleading to advertise establishment registration and product listing in any manner that may imply product approval or compliance with the law. 

If manufacturers, clinics, and health care providers offering regenerative medicine products to patients did not contact the FDA about the need for an IND during the period the Tissue Reference Group Rapid Inquiry Program was offered, there remain three options that have been available for many years and these options continue to be available. We want to remind stakeholders that a product that requires but lacks premarket approval may not be lawfully marketed or offered for sale, including when a sponsor has an IND or is pursuing an IND or BLA for its HCT/P.

The FDA is committed to helping advance the development of clinical trials for regenerative medicine products with the shared goal of safe and effective products for patients. We look forward to working with those who share this goal.

For more information: Important Patient and Consumer Information About Regenerative Medicine Therapies

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