In November of 2017, FDA published its enforcement discretion policy regarding HCT/Ps that don’t meet all four criteria under 21 CFR 1271.10(a):
To give manufacturers time to determine if they need to submit an IND or marketing application in light of this guidance and, if such an application is needed, to prepare the IND or marketing application, for the first 36 months following issuance of this guidance FDA generally intends to exercise enforcement discretion with respect to the IND and the premarket approval requirements for HCT/Ps that do not meet one or more of the 21 CFR 1271.10(a) criteria, provided that use of the HCT/P does not raise reported safety concerns or potential significant safety concerns.
FDA has believed for some time that numerous HCT/P manufacturers don’t meet all four criteria for regulation solely under 21 CFR Part 1271, and they have been hoping that the publication of the 2017 Guidance on Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue Based Products: Minimal Manipulation and Homologous Use, together with the 36 months of enforcement discretion (with 22 months remaining), will entice many of these firms to meet with FDA, either to discuss whether some type of clearance or approval for their product is required or, alternatively, to discuss the data requirements for said clearance or approval. However, thus far it does not appear as though many firms have taken FDA up on their offer.
In a press release issued on December 20th, 2018, FDA stated:
Even though a few sponsors have come to us, we are discouraged by the overall lack of manufacturers wanting to interact with the agency in this enforcement discretion period… [T]here’s a clear line between appropriate development of these products and practices that sidestep important regulatory controls needed to protect patients. [Emphasis added]
FDA also indicated in this press release that once the 36 months of enforcement discretion lapses the agency will be “increasing oversight” related to cell-based regenerative medicine products. It is somewhat unclear what FDA means by “increasing oversight” though it is likely to include a combination of Warning Letters to stem cell companies and injunctions and seizures involving those firms that are deemed to pose a more significant public health risk.
To that end, the agency fired a warning shot across the bow of these regenerative medicine companies in late December by also issuing “To Whom It May Concern” letters, stating that the recipient of the letter appears to offer stem cell products “to treat a variety of diseases or conditions.” The letter goes on to remind the recipient of the agency’s November 2017 “comprehensive regenerative medicine policy framework…” and that the enforcement discretion period “…provides manufacturers time to comply with the IND and premarket approval requirements and engage with FDA to determine whether they need to submit an IND or marketing authorization application, and if so, to submit their application to FDA.”
One can expect FDA to continue to ramp up the pressure on stem cell companies as the enforcement discretion period winds down. We’ll keep you posted on all developments.