At the tail end of December, the FDA released draft guidelines on Cell and Tissue-Based Products, titled “Minimal Manipulation of Human Cells, Tissues, and Cellular and Tissue-Based Products.” The guidelines came on the heels of another set of guidances, “Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception,” which the FDA had released a month prior.
The December guidelines define a Human Cell or Tissue Therapy Product (HCT/P) under section 361 of the PHS Act and 21 CFR Part 1271 if the following apply (taken from FDA.gov):
- The HCT/P is minimally manipulated;
- The HCT/P is intended for homologous use only, as reflected by the labeling, advertising, or other indications of the manufacturer’s objective intent;
- The manufacture of the HCT/P does not involve the combination of the cells or tissues with another article, except for water, crystalloids, or a sterilizing, preserving, or storage agent, provided that the addition of water, crystalloids, or the sterilizing, preserving, or storage agent does not raise new clinical safety concerns with respect to the HCT/P; and either:
1. The HCT/P does not have a systemic effect and is not dependent upon the metabolic activity of living cells for its primary function; or
2. The HCT/P has a systemic effect or is dependent upon the metabolic activity of living cells for its primary function, and:
a.Is for autologous use;
b.Is for allogeneic use in a first-degree or second-degree blood relative; or
c.Is for reproductive use.
This week, the Alliance for Regenerative Medicine (ARM), a large advocacy organization supporting the cell therapy industry, released a set of comments on the FDA guidelines, asking for the FDA to provide clarification on a number of concepts, one of them being “main function.”
From the release:
“ARM requests more information on the concept, origin and application of the draft guidance’s inclusion of the new and yet-undefined term “main function” of the human cells, tissues, cellular and tissue-based products (HCT/Ps) when assessing minimal manipulation, as these products may have more than one function and it is not clear which of these functions would be considered primary.”
ARM calls on the FDA to “hold a public hearing to engage with stakeholders in a full airing of the topic.”
Similar comments and requests for clarification followed the release of the FDA’s previous guideline. Nonetheless, the industry appears to agree on the consensus that the FDA’ efforts in standardizing the development of cell therapy products are commendable