The ability to facilitate the regeneration of parts of the human body is “no longer the stuff of science fiction” according to FDA Commissioner Scott Gottlieb.[i] According to Commissioner Gottlieb, the cell based therapies and their use in regenerative medicine is one of the most promising fields of science already producing “improbable advances.” At the current early stages of development, deceptive claims from unscrupulous actors risks “jeopardizing the legitimacy and advancement of the entire field.” In order to curb such deception while simultaneously providing a clear and efficient path for product developers, the FDA has recently published a suite of four new guidance documents related to their regenerative medicine policy framework.
These four guidance documents represent a broad policy framework by the FDA building upon the framework put into effect in 2005. In its never ending battle to ensure the safety and efficacy of medical products while simultaneously supporting innovation, the FDA’s new approach will undoubtedly entail additional requirements on those innovators in the regenerative medicine field. It is important to review the guidance documents to ensure compliance with the FDA, but also to take advantage of the various opportunities for approval for those medicines and products that meet certain criteria. For this purpose, below is a brief summary of what the guidance documents include.
As guidance documents, the information provided does not create legally enforceable responsibilities, but rather they provide a glimpse into how the FDA is currently thinking and how the FDA will likely apply the existing laws and regulations that govern regenerative medicine products. Two of the documents are final guidance documents now in effect with the remaining two documents in draft form allowing the public to provide their comments.
Final guidance documents
The first guidance document titled “Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception”[ii] provides clarity on when cell and tissue-based products would be excepted from the current regulations if they are removed from and implanted into the same individual during the same surgical procedure and remain in their original form.
The second guidance document titled “Regulatory Considerations for Human, Cell, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use”[iii] clarifies how the FDA interprets the regulatory definitions for “minimal manipulation” and “homologous use.” This guidance is particularly helpful in that these concepts establish the legal threshold for when a particular product is subject to the FDA’s premarket approval requirements. In light of the how this new interpretation will effect manufacturers, healthcare providers, the FDA plans to exercise ‘enforcement discretion’ to those products that do not pose a potential significant safety concern for 36 months.
Draft guidance documents
The first draft guidance document titled “Evaluation of Devices Used with Regenerative Medicine Advanced Therapies”[iv] attempts to clarify how the FDA will evaluate devices referred to as “regenerative medicine advanced therapies” or “RMATs” in accordance with the regenerative medicine provisions in the 21st Century Cures Act. These devices include medical devices used in the recovery, isolation, or delivery of RMATs. It is the goal of the FDA to simplify and streamline the application of the regulatory requirements of RMATs.
The second draft guidance document titled “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions”[v] describes, unsurprisingly, various expedited programs to sponsors of RMATs. In addition, the guidance also describes the new RMAT designation process to be used.
The two draft guidance documents were posted to the Federal Register on November 17, 2017, with a comment period ending February 15, 2018. There is ample time for interested parties to assist the FDA in finalizing the draft guidance documents by submitting a comment by the February 15, 2018, deadline.
These guidance documents may provide new opportunities or new requirements for those operating in the field of regenerative medicines. They should be reviewed to ensure compliance with existing laws and regulations while also looking to take advantage of the programs to accelerate FDA approval.
[i] “Statement from FDA Commissioner Scott Gottlieb, M.D. on FDA’s comprehensive new policy approach to facilitating the development of innovative regenerative medicine products to improve human health” posted November 16, 2017, available at https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585342.htm; see also, “FDA announces comprehensive regenerative medicine policy framework” posted November 16, 2017, available at https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585345.htm.