Regenerative Medicine Policy Issued by FDA

The FDA recently announced a novel policy for the development of regenerative medicine products. The policy adds to the existing risk-based regulatory approach to define what products are characterized as drugs, devices, and biologics, according to a press release.

“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,” said FDA Commissioner Scott Gottlieb, MD. “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.”

The proposed framework is described in 4 guidance documents, 2 of which support an efficient, science-based process to ensure the safety and efficacy of regenerative medicine products, while also encouraging innovation, according to the FDA. The guidance documents also address how the agency plans to take action against products that have potential safety concerns.

The FDA said the policy aims to balance safety concerns with mechanisms that help bring novel products to market.

“With the policy framework the FDA is announcing today, 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,” Dr Gottlieb said. “This will protect patients from products that pose potentially significant risks, while accelerating access to safe and effective new therapies.”

Finalized Guidance Documents

• The first finalized guidance document clarifies when cell- and tissue-based products would be excepted from the regulations if they are derived from and implanted back into the same patient during surgery and the cells remain in their original form, according to the release.
• The second guidance document clarifies how current regulatory criteria applies to regenerative medicine products by detailing how the FDA defines “minimal manipulation” and “homologous use.” The FDA said there are numerous products that require pre-market authorization and the new guidance discusses how the agency provides the risk-based framework for oversight, according to the release.

Draft Guidance Documents

• The first draft guidance document builds on provisions in the 21^st Century Cures Act by streamlining regulatory requirements for devices used in the recovery, isolation, and delivery of regenerative medicine advanced therapies, according to the FDA.
• The second guidance document aims to speed the approval of regenerative medicine products, including a new Regenerative Medicine Advanced Therapy (RMAT) designation. Additionally, the framework outlines the types of products eligible for RMAT designation, such as cell therapies, therapeutic tissue engineering products, human cell and tissue products, combination products, and gene therapies, according to the release.

The first gene therapies to gain FDA approval are tisagenlecleucel (Kymriah) for refractory B-cell precursor acute lymphoblastic leukemia and axicabtagene ciloleucel (Yescarta) for diffuse large B-cell lymphoma. It is likely that many more will come to market over the next few months, according to the FDA.

This guidance provides a new framework for the therapies to ensure that treatments are safe.

“As a molecular and cell biologist and physician, it has been exciting to witness the approval of the first 2 gene therapies in the US this year. Given the great opportunities that the field of regenerative medicine presents, we have undertaken a rigorous process to clarify our regulations that included solicitation of public input, and I believe today marks a significant step forward for all stakeholders,” said Peter Marks, MD, PhD, director of the FDA Center for Biologics Evaluation and Research. “In addition to clarifying some of the more complex areas of the regulations, we have taken meaningful new steps to encourage and expedite the development of innovative therapies. We welcome public comment on our draft guidance documents as we work toward finalizing this framework.”