FDA addresses cell-based regenerative medicine in comprehensive new policy



The Food and Drug Administration has announced a new policy that addresses the rapid growth and development of regenerative medicine products, which include novel cellular therapies, with the aim of ensuring their safety and effectiveness.

“The framework – outlined in a suite of four guidance documents – builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products,” the FDA announced in a statement released on Nov. 16.

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“We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease,” FDA Commissioner Scott Gottlieb, MD, said in the statement.

He added: “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.” But, while advances have benefited many patients, he referred to a small number of “unscrupulous actors” that have provided treatments that have harmed patients, which is why stricter FDA enforcement is needed.

Clarification of the existing regulations will “promote responsible and flexible regulation that leverages science to advance public health,” Dr. Gottlieb said during a media briefing held by the FDA to discuss the new framework.

During the briefing, in response to a question concerning adipose tissue injections and their associated risks, Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research, explained that the guidance documents will clearly delineate when adipose tissue will be classified as a structural tissue and a stem cell product. If a provider is purveying dangerous products, the FDA can “when necessary, undertake seizures, ask for injunctions, and in some cases when it has been determined that certain violations have occurred even criminal actions can be taken,” he said.

Earlier this year, a report of three patients who had severe visual loss after treatment with intravitreal injections of autologous adipose tissue for age-related macular degeneration was published (N Engl J Med. 2017;376:1047-53).

The new framework is composed of two final guidance documents and two draft guidance documents. The first final guidance document provides details about regulations concerning cell and tissue-based products and when those products are subject to regulation in surgical procedures. The second final guidance document elaborates on the definition of “minimal manipulation” and “homologous use” with the hopes of clarifying what products are subject to regulation. These documents will also explain how the FDA will provide a framework for premarket authorization for cell-based regenerative products.

One draft guidance outlines the FDA’s plan to simplify and expedite the application of the regulatory requirements for devices used in relation to regenerative medicine advanced therapies; the second draft guidance outlines the expedited programs that may be available to sponsors of regenerative therapies.

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