Hard Cell: Potency Testing for Cellular Therapy Products

Today, we hear from Tom Pritchett and Laureen Little who published the article "Hard Cell": Potency Testing for Cellular Therapy Products and look at the Regulatory Requirements and Expectations in BioProcess International.  

Regulatory Requirements and Expectation

Human cells are regulated as biologics if any of the following criteria are met: They are more than minimally manipulated, they are combined with an article other than a preservation or storage agent, they are used in a way that is not homologous to their normal function, or they have a systemic effect and depend on the metabolic activity of living cells for their primary function.

As biological products, cellular therapies are regulated under section 351 of the Public Health Services Act (42 U.S.C. 262) and the Food, Drug and Cosmetics Act (21 U.S.C. 321 et seq.) They also must comply with FDA’s current good manufacturing Practice (CGMP) regulations for Finished Pharmaceuticals (21 CFR Parts 210 and 211) and with relevant sections of the biologics regulations at 21 CFR 600. Compliance with these requirements applies to all biological products, including autologous and single patient allogeneic products, in which a lot may be defined as a single dose(2). In addition, cell therapy products must comply with applicable parts of the current good tissue practice (CGTP) rules in 21 CFR part 1271. The FDA pairs cellular therapy products with gene therapy products for regulatory purposes and refers to them as CGT (cellular and gene therapy) products. Regulatory submissions for CT products are reviewed by FDA’s Office of Cellular, Tissue and Gene Therapies (OCTGT) in the Center for Biologics Evaluation and Research (CBER).

The FDA feels that “These regulations provide considerable freedom when evaluating suitable potency assays” (6). For approval of a biologics license application (BLA), CT products must meet the requirements of safety, purity, and potency prescribed in the above regulations. However, as stated in FDA’s guidance on CGT potency testing, “FDA regulations allow for considerable flexibility in determining the appropriate measurement(s) of potency for each product.” As is typical, the FDA evaluates the adequacy of potency tests for such cutting-edge products case by case. (2).

In reviewing a potency assay to be used for release testing of a licensed product, the agency considers whether the method meets the following regulatory requirements and expectations (2, 6).

Lauren Little will be joining us at Biological Assays  to present the white paper report What are We Monitoring to Maintain our Potency Assays?.  To find out more about her presentation and the rest of the program, download the agenda.  Would you like to join Lauren?  As a reader of this blog,when you register to join us this May 5-7, 2014 in Berkeley, California and mention code B14177JT, you can save 20% off the standard rate.

References
2 CBER. Guidance for Industry, Potency Tests for Cellular and Gene Therapy Products, January 2011.
6 Gavin DK (CBER Office of Cellular,Tissue and Gene Therapies, Division of Cellular and Gene Therapies). Presentation at the Cellular, Tissue, and Gene Therapies Advisory Committee Meeting, 9 February 2006. Slides and transcript available at www.fda.gov/ohrms/dockets/ac/cber06.html#CellularTissueGeneTherapies.

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