By: Peter Marks, MD, Ph.D., Director, Center for Biologics Assessment and Research

The United States Food and Drug Administration plays a vital role in facilitating the development and availability of innovative medical products. Products such as cell-derived therapies, including stem cell products, offer the potential to treat or even cure diseases or conditions for which there are few effective treatment options.

The FDA Regenerative Medicine Policy Framework of November 2017 was developed to facilitate and support innovation in regenerative medicine therapies. As part of this framework, we encourage sponsors to take advantage of any ongoing accelerated programs that may be available to them, including Advanced Therapy in Regenerative Medicine, Breakthrough Therapies, and Expedited Designations, to support product development and approval.

Director of CBER, Peter Marks, MD, Ph.D.
Peter Marks, MD, Ph.D.

The framework also outlines the agency’s intention to exercise enforcement discretion with respect to the FDA’s pre-market and investigational new drug approval (IND) requirements for certain regenerative medicine products until. in November 2020, which was then extended to May 2021. This compliance and enforcement discretion gives manufacturers time to determine if certain requirements apply to their products and, if a request is made. necessary, to prepare and submit the appropriate application to the FDA.

We now reaffirm the timeline for the end of the discretionary compliance and enforcement policy for certain human cell, tissue and human cells and tissues (HCT / P) products, including regenerative medicine therapies. The period during which the FDA intends to exercise its discretion with respect to the IND and pre-market approval requirements for certain HCT / Ps ends on May 31, 2021 and will not be extended.

Since November 2017, the FDA has been working with product developers to help them determine whether they should submit an IND or market application and, if so, how they should submit their application to the FDA. The FDA has developed programs that provide opportunities for engagement between HCT / P manufacturers and the agency, including the Tissue Reference Group (TRG) Rapid Investigation Program (TRIP). TRIP has helped HCT / P manufacturers, including stakeholders who market HCT / Ps to physicians or patients, obtain a rapid, preliminary, informal, and non-binding assessment from the FDA regarding how HCT / Ps specific are regulated. TRIP was a temporary program of the TRG. TRIP started in June 2019 and has been extended twice. It recently ended on March 31, 2021.

Despite the FDA’s best efforts to involve the industry, there is still widespread commercialization of these unapproved products for the treatment or cure of a wide range of illnesses or medical conditions. Many of these unapproved products appear to be HCT / Ps which are regulated as drugs, devices and / or biologics subject to pre-market approval requirements. The extent of the marketing of these unapproved products is evidenced by their inappropriate advertising in various media and the number of consumer complaints about them submitted to the FDA.

These regenerative medicine products are not without risk and are often marketed by clinics as safe and effective for the treatment of a wide range of diseases or conditions, although they have not been sufficiently studied in the clinical tests. We have said it before and want to reiterate it here: Manufacturers, clinics or health care practitioners do not have the right to endanger patients through products that violate the law, including those that do not. having no IND in force or an approved biologics license. We will continue to take action on illegally traded products. Our oversight of cellular and related products has included taking compliance action, including numerous warning and untitled letters, and pursuing enforcement action for serious violations of the law.

As of December 2019, the agency has sent more than 350 letters to manufacturers, clinics and healthcare providers, noting that it has come to our attention that they may offer unapproved regenerative medicine products and reiterating the FDA compliance and enforcement policy.

We encourage the public and patients considering treatment with regenerative medicine products to work with their healthcare providers to learn about the treatment available. Ask questions and understand the potential risks of treating with unapproved products. It is essential to seek treatment only using legally marketed products or, for unapproved products, to enroll in clinical trials under FDA oversight. The public can visit the FDA website to find out if a particular regenerative medicine product is approved.

The FDA remains committed to helping advance the development of safe and effective regenerative medicine products, including stem cell products, for the benefit of those in need. We look forward to working with those who share this goal.