Time's up for questionable cell and tissue products, says Marks

Regulatory NewsRegulatory News | 22 April 2021 |  By 

Peter Marks, MD, PhD

Purveyors of unapproved cellular-derived treatments are about to find out what happens when the US Food and Drug Administration ends its period of enforcement discretion for these regenerative medicine products.
A three-year clock for enforcement discretion began when FDA announced its regenerative medicine policy framework in November 2017; the COVID-19 pandemic prompted a 6-month extension, announced the agency last summer. Time is now up, and no more extensions are forthcoming, according to Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research (CBER). The new enforcement era will begin in just a few weeks, on 1 June. (RELATED: Six more months tacked onto regenerative medicine enforcement date, Regulatory Focus 20 July 2020)
“Despite all of the FDA’s efforts to engage industry, there continues to be broad marketing of these unapproved products for the treatment or cure of a wide range of diseases or medical conditions,” said Marks in a Tuesday FDA Voices piece. Many such unapproved products, he said, have premarket approval requirements since they are regulated as drugs, devices, or biologics.
“The wide extent of the marketing of such unapproved products is evidenced by their inappropriate advertisement in various media and by the number of consumer complaints about them submitted to the FDA,” Marks went on to say, noting that many of the treatments are “not without risk,” though the clinics offering them represent these treatments as both safe and effective.
CBER has sent more than 350 warning letters and notices since December 2019; the letters have gone to manufacturers as well as clinics and individual health care providers who “may be offering unapproved regenerative medicine products,” said Marks.
A representative letter from January of this year shows the breadth of the claims made by some of the clinics that market regenerative medicine products. In a 25 January warning letter, CBER told California-based firm The Body Building, Inc., that a biologics license would be needed before it could contemplate continuation of its marketing “primitive undifferentiated stem cells” to “immediately dissipate any pain in the body.” (RELATED: Stem cell and allergy clinics receive CBER untitled letters, Regulatory Focus 29 January 2021)
That letter also noted that The Body Building was marketing unlicensed and unapproved exosome products, and that the company was making claims that its products could treat Parkinson’s disease, heart disease, autoimmune diseases, diabetes and autism, among other maladies.
In other warning letters during the pandemic, FDA has noted that its risk-based enforcement approach means that therapies purporting to prevent or treat COVID-19 are more likely to draw CBER’s attention, as are those therapies that involve intravenous or intrathecal administration.
A final guidance last updated in July 2020 provides examples, guidelines and a flow chart for how to apply the provisions of 21 CFR 1271.15(b) and 1271.10(a) to human cell, tissue, and cellular and tissue-based products (HCT/Ps). The guidance points developers and marketers of HCT/Ps to which criteria, including the “minimal manipulation” test, must be met in order for a product to be exempt from regulation as a drug, device or biological product.
The Alliance for Regenerative Medicine (ARM), a trade association representing regenerative medicine firms, applauded Marks’ words and the end of the discretionary enforcement era.  “ARM shares the FDA’s concerns about the marketing of unapproved biologic drugs and we support strong oversight and enforcement against bad actors,” Stephen Majors, ARM’s director of public affairs, told Focus. “Strong regulatory oversight is imperative for protecting public health while continuing to foster scientific innovation and the development of safe, effective regenerative medicines.”
Majors also pointed Focus to ARM’s August 2020 comment letter on the Prescription Drug User Fee Act reauthorization (PDUFA VII). Noting the explosive growth in stem cell clinics over the last decade, ARM asked for increased FDA resources “to establish and implement strong, swift, and consistent legal actions against these questionable clinics and prosecute those that are in violation of the law.”
FDA Voices


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