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Below we discuss the importance this has for regenerative medicine companies and outline the potential risks of non-compliance. These risks go well beyond FDA enforcement and include the possibility of FTC action, liability under the False Claims Act, product liability claims, and exposure to private lawsuits under the Lanham Act and similar state laws.
In 2017, the FDA released a new framework for regenerative medicine, which consists of four guidelines that we discussed earlier here. In this context, the FDA provided a period of three years for the risk-based enforcement of certain human cells, tissues, or cell- or tissue-based products (HCT / Ps), which ended in November 2020. This “grace period” initially began. The planned term of three years should give certain manufacturers time to assess whether they need to submit a new drug application (IND) or a marketing application to the FDA or whether they meet the four regulatory criteria for further marketing their products Products solely under meet the authority of Section 361 of the Public Health Service Act (PHSA), which does not require pre-market testing and approval. In July 2020, citing the challenges of the COVID-19 pandemic, the FDA extended this grace period by a further six months to May 31, 2021, as we have summarized here.
The FDA’s growing frustration despite efforts to help the industry comply with regulations
In June 2019, the FDA implemented a temporary program called the Tissue Reference Group Rapid Inquiry Program (TRIP) to help HCT / P manufacturers and stakeholders marketing HCT / Ps to get “a quick, preliminary, informal and receive a non-binding assessment of how certain HCT / Ps are regulated ”. We have summarized the possibilities this program offers here. TRIP was originally supposed to last until December 31, 2019 and has been extended to March 31, 2021.
The end of TRIP at this point signaled that the FDA would not extend the enforcement period for HCT / P sponsors again. This signaling was consistent with other public messages from the FDA as well as private communications, indicating the likely increased enforcement of the FDA in the regenerative medicine arena. In the June 2020 JAMA article, Drs. Marks and Hahn cited “an increasing number of adverse events” after unapproved regenerative medicine therapies were used to induce the FDA to prevent harm. They strongly admonished, “Companies that sell products that are in violation of the regulations do so under the mistaken claim that they are exempt from these FDA regulations.”
Since 2019, the FDA has issued more than 350 warning and untitled letters to manufacturers, clinics and healthcare providers in connection with non-approved products for regenerative medicine. Indeed, a number of actions by recent authorities – including FDA quotes, warning letters, product seizures, and even criminal charges – have shown the government’s growing seriousness and commitment to stem cell therapy surveillance that we have summarized here.
The FDA is not extending the discretionary period for enforcement of HCT / P
In an April 21 FDA blog post “Voices”, Dr. Marks that the agency will not again extend the “grace period” for HCT / P companies to meet their regenerative medicine policy framework. In that article, Marks emphasized that the FDA has and will continue to take action regarding illegally marketed HCT / Ps. Marks noted that since December 2019, the FDA has “sent more than 350 letters to manufacturers, clinics, and healthcare providers advising them that they may offer unapproved regenerative medicine products and comply with FDA and the rules repeat enforcement policy. “
So, before May 31stWhen the FDA’s enforcement period ends and the agency takes action against companies operating in an area that does not comply with regenerative medicine guidelines, it is imperative that both HCT / P manufacturers and stakeholders marketing HCT / Ps – These include merchants, billing companies and consultants, health economics consultants, clinics, and healthcare providers. Make sure they meet FDA standards.
Companies that continue to market regenerative medicine products (including tissues) under PHSA Sections 361 and 21 CFR Part 1271 must seriously assess whether their products will still meet the 361 HCT / HCT criteria after the enforcement deadline has passed. Ps can meet. These companies should also consider all marketing risks if these products are deemed to be non-qualified. These risks go beyond FDA enforcement and include the potential for:
False Claims Act (FCA) liability is based on FDA and CMS regulations and marketing practices
Private Qui Tam promotions to try to enforce FCA requirements
Private claims for false advertising and unfair competition under the Lanham Act
False Advertising, Unfair Commercial Practices, and Unfair Competition Claims
Claims for product liability under common national law
Medicare and Medicaid contractor audits
Companies currently marketing HCT / Ps that do not meet the criteria of PHSA Section 361 face limited options if they want to lawfully market these products in the future. Of course, in this situation, the FDA would like companies to file an IND, initiate controlled clinical trials, and ultimately submit a marketing proposal. For many companies in this situation, however, the prospect of opening an IND and initiating registration-quality clinical trials is enormous.
A number of important questions arise in connection with companies seeking FDA approval for their HCT / Ps. For example:
In this situation, will the FDA require companies to conduct preclinical animal studies before initiating human clinical trials?
Will companies be able to convince the FDA to accept data from previous experience with their products to aid direct entry into later clinical trials (rather than having to start conventional phase 1 trials)?
If companies have previously conducted uncontrolled open label studies on their products, is the FDA ready to accept data from these studies in support of a marketing proposal?
If a company lawfully markets an HCT / P in other countries, could the company provide patient data from those countries to the FDA as real evidence in support of regulatory approval?
In this situation, can companies qualify for any of the FDA’s expedited programs for serious medical conditions such as Fast Track, Breakthrough Therapy, Expedited Approval, or Advanced Regenerative Medicine Therapy?
It remains to be seen how flexible the FDA will be in working with companies that use good faith efforts to obtain regulatory approvals for their HCT / Ps.
Patient Access Concerns
The likelihood of upcoming enforcement actions is a concern about patient access to products that have been relied on for many years as companies may decide to withdraw their products instead of seeking FDA approval. The question remains whether companies that decide to withdraw their products should try other precautions for patients in need, such as: B. by conducting clinical trials or making their products available in accordance with the FDA’s expanded access regulations (sometimes referred to as “compassionate use”). ).