In November, the FDA released its long-awaited regenerative medicine policy framework consisting of four guidance documents; two final and two draft. The guidance documents arrive at a time of rapid growth and expansion in the commercialization of regenerative medicines and represent the FDA’s attempt to ensure safety and effectiveness of the therapies, while spurring innovation and streamlining the regulatory process in the burgeoning industry.
“We’re adopting a risk-based and science-based approach that builds upon existing regulations to support innovative product development while clarifying the FDA’s authorities and enforcement priorities,” said FDA Commissioner Scott Gottlieb in a statement. “This will protect patients from products that pose potential significant risks, while accelerating access to safe and effective new therapies.”
One of the final guidance documents sets out to define “minimal manipulation” and “homologous use”, two of several criteria for classification of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) that are confusing and often misinterpreted.
“Homologous use” refers to cells or tissues that perform the same basic function or functions in the recipient as in the donor. “Minimal manipulation” refers to processing that does not alter the original relevant characteristics of the tissue or the relevant biological characteristics of cells or tissues.
The second final guidance document clarifies when a tissue/cellular product is not required to meet the regulatory requirements in 21 CFR 1271 Good Tissue Practices. Tissue/cellular products that are autologous, taken during the same surgical procedure, and remain in original form are not subject to 21 CFR 1271.
The guidances were viewed by many as a response to the recent proliferation of unapproved direct-to-consumer (DTC) stem cell products. In addition, while some industry experts argued the new guidelines do not go far enough to regulate unscrupulous parties, others, including Ann Kemp, Senior Manager, Regulatory Compliance at Be The Match BioTherapies, were encouraged by the level of detail provided.
“The FDA really honed in on those terms, made the criteria clear and provided many examples,” said Kemp. However, while the finalized guidance documents were expected, it was one of the draft guidance documents that grabbed the attention of industry experts.
The guidance document Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, November 2017 (DRAFT) would allow companies to apply for a designation as a Regenerative Medicines Advanced Therapies (RMAT) for certain cellular therapy products. Once granted, this status would allow companies to take advantage of other FDA expedited approval pathways, such as Fast Track Designation, Breakthrough Therapy Designation, Accelerated Approval, and Priority Review Designation.
“I expect we’ll see a lot of companies applying for RMAT designation,” says Kemp. There is a significant need for regenerative therapies and many start-up and mature companies wanting to get into the market, thus wanting a streamlined FDA approval process.”
“This is a sign that the FDA gets it and they are willing to work with the industry,” said Kemp. “They recognize the complexity and the pace of this field and want to help us expedite approval, while at the same time ensuring these therapies are safe and effective.”