FDA releases framework guidelines for regenerative med development

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The FDA this week laid out guidelines for a new framework it’s implementing for the development and oversight of regenerative medicine products including novel cellular therapies.

The framework is laid out through four guidance documents which build on the FDA’s existing regulatory approach and describe more clearly which products are regulated as drugs, devices and biological products, according to a new FDA release.

The documents include new science-based processes for ensuring the safety and effectiveness of such products and a risk-based framework for how it plans to enforce actions against potentially harmful products.

“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. This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine. But this field is dynamic and complex. As such, it has presented unique challenges to researchers, health care providers, and the FDA as we seek to provide a clear pathway for those developing new therapies in this promising field, while making sure that the FDA meets its obligation to ensure the safety and efficacy of the medical products that patients rely upon. Alongside all the promise, we’ve also seen products marketed that are dangerous and have harmed people. 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. This will protect patients from products that pose potential significant risks, while accelerating access to safe and effective new therapies,” FDA Commissioner Dr. Scott Gottlieb said in a press release.

The guidance documents released by the federal watchdog include two final guidance documents and two draft documents. The two final guidance documents cover the FDA’s view on the risk-based criteria manufacturers use to determine whether products are subject to premarket review.

The first final guidance document provides extra clarity on products that would be excepted from regulations if they are removed from one patient and implanted into the same patient, and the second clarifies how the agency interprets the regulatory definitions of “minimal manipulation” and “homologous use.”

The first of its draft guidances looks to build on regenerative medicine provisions in the 21st Century Cures Act and address how the FDA looks to simplify regulatory requirements for regenerative medicine advanced therapies.

The second draft guidance describes expedited programs that may be available to sponsors of regenerative medicine therapies, including the regenerative medicine advanced therapy designation created by the 21st Century Cares Act.

“As a molecular and cell biologist and physician, it has been exciting to witness the approval of the first two gene therapies in the U.S. 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. 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,” FDA Center for Biologics Evaluation and Research director Dr. Peter Marks said in a press release.

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