The field of regenerative medicine is ever expanding and evolving. As more viable options become available to patients, it’s important to stay abreast of regulation surrounding many of these applications.
In late 2019, the Food and Drug Administration (FDA) began informing the public of multiple reports of serious adverse events experienced by patients who were “treated” with non-FDA approved products marketed as containing exosomes. As a general matter, exosomes used to treat diseases and conditions in humans are regulated as drugs and biological products under the Public Health Service Act and the Federal Food Drug and Cosmetic Act and are subject to premarket review and approval requirements. At the time of the 2019 warning, there were no FDA-approved exosome products and the FDA since then has not put out any new guidance.
In a decision expected to cause waves through the rapidly-expanding regenerative medicine industry, a U.S. District Court Judge ruled on June 3rd that the U.S. Food and Drug Administration (FDA) is entitled to an injunction in a lawsuit filed against U.S. Stem Cell Clinic, LLC (US Stem Cell) based in Sunrise, Florida. In her decision, U.S. District Court Judge Ursula Ungaro agreed that the FDA has the authority to regulate the popular stem cell procedure known as stromal vascular fraction (SVF) – administering processed stem cells derived from adipose tissue (i.e. fat tissue) – and that US Stem Cell is not exempt from regulation.
To recap, in May 2018, the U.S. Department of Justice (DOJ) filed complaints against US Stem Cell and a California stem cell clinic seeking permanent injunctions to prevent the marketing and administration of the SVF procedures without FDA approval. Prior to the filing of these actions, both companies received warning letters from the FDA. The letters also addressed the results of inspections and the need to resolve significant deviations from manufacturing practice requirements.
Jumping into the stem cell industry can be an exciting venture. However, with this emerging industry comes a mine field of legal pitfalls and potential problems. The keys to a successful business not only include selecting a strong product and building strong relationships with clients but being able to navigate the regulatory framework that accompanies this type of product.
FDA regulations require establishments that perform one or more steps in the manufacturing process of HCT/Ps (i.e. Human Cells, Tissues, and Cellular and Tissue-Based Products) to register and submit a list of products with the agency. If so, you have five days to register after beginning operations. When I mention “manufacturing” to clients they usually interject with “I only want to distribute.” Good point. However, the FDA defines “manufacture” as any or all steps in the recovery, processing, storage, labeling, packaging, or distribution of any human cell or tissue. These registrations must be updated annually and in the event of a change of ownership, within 30 days of the change.
The regenerative medicine arena consists of a wide range of innovative products. Congress, acknowledging the importance of this field, has established a new program via the 21st Century Cures Act to help spur development and provide for accelerated approval for regenerative medicine products similar to the FDA’s fast track and breakthrough therapy designations. This new approval is the Regenerative Medicine Advanced Therapy (RMAT) Designation.
The RMAT Designation includes all the benefits of the FDA’s other accelerated designations including early agency engagement and priority review; however, unlike the other designations, the RMAT Designation does not require evidence that the product offers substantial improvement over other therapies. For a drug to be eligible for the RMAT Designation, it must meet the following:
Through two public channels this month, the FDA further solidified its stance on the innovative field of regenerative medicine. First, in an article published in the New England Journal of Medicine (NEJM), Dr. Scott Gottlieb, FDA Commissioner, and Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER), co-wrote a new paper entitled “Balancing Safety and Innovation for Cell-Based Regenerative Medicine.” On the same day of this publication, the FDA hosted a “Grand Rounds” webcast with Dr. Steven Bauer, Chief of the Cellular and Tissues Therapy Branch within CBER. Taken together, these actions suggest a continued effort by the FDA to take a strong position against predatory clinics touting unapproved therapies while extending an open invitation to industry developers for expedited treatment to encourage innovation.
The U.S. Food and Drug Administration (FDA) has issued new guidance for regenerative medicine manufacturers and healthcare providers. At the core of the guidance are two central interpretations: 1) the FDA’s current interpretation of the minimal manipulation and homologous use criteria set forth in 21 CFR Part 1271; and 2) the FDA’s current view on the same surgical procedure exception under 21 CFR 1271.15(b). Additionally, the FDA issued a notice to all interested stakeholders that the FDA intends to initiate increased discretionary enforcement over the next 36 months for human cell and tissue-based products. Given these developments, healthcare companies and providers impacted by this guidance are strongly encouraged to ensure compliance with the FDA’s new interpretations.
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