FDA announces policy framework to develop regenerative medicine products

The FDA announced a comprehensive policy framework for the development and oversight of regenerative medicine products outlines in a suite of four guidance documents.

Perspective from Cato T. Laurencin, MD, PhD

Scott Gottlieb

To more clearly describe what products are regulated as drugs, devices or biological products, the framework of the guidance documents builds upon the FDA’s existing risk-based regulatory approach, according to a press release. The guidance documents not only propose an efficient, science-based process for helping to ensure the safety and effectiveness of these therapies while supporting development in this area, the release noted it also define a risk-based framework for how the FDA intends to focus its enforcement actions against those products that raise potential significant safety concerns.

Final, draft documents

Broken into two final guidance documents and two draft guidance documents, the final documents help explain how the FDA will provide a risk-based framework for the oversight of regenerative medicine products undergoing premarket authorization. The first guidance document focuses on identifying “when cell and tissue-based products would be excepted from the established regulations if they are removed from and implanted into the same individual within the same surgical procedure and remain in their original form”, according to the release. The second final guidance document clarifies how the agency interprets the existing regulatory definitions “minimal manipulation” and “homologous use” for stakeholders to better understand how existing regulatory criteria apply to their products.

To help spur development and access to innovative regenerative therapies, the release noted the first draft guidance document builds off the regenerative medicine provisions in the 21st Century Cures Act by addressing how the FDA intends to simplify and streamline its application of the regulatory requirements for devices used in the recovery, isolation and delivery of regenerative medicine advanced therapies (RMATs), including combination products. The second draft guidance describes both the expedited programs that may be available to sponsors of regenerative medicine therapies, as well as the regenerative medicine therapies that may be eligible for RMAT designation, the release noted. Both draft guidance documents will have a 90-day comment period.

Paradigm shift

“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; an adult stem cells can generate replacements for cells that are lost to injury or disease,” Scott Gottlieb, MD, commissioner of the FDA, said in the release. “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.”

PAGE BREAK

However, Gottlieb added this dynamic and complex field has presented unique challenges to not only the FDA, but to researchers and health care providers as well, as they “seek to provide a clear pathway for those developing new therapies in the 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,” Gottlieb said. “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.”

Reference:

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585345.htm

Perspective

Cato T. Laurencin, MD, PhD

Christopher Murdock

The FDA’s establishment of concrete rules to increase safety, but also allow innovators to provide effective therapies to patients as proficiently and safely as possible, is a mandate that is necessary and invaluable to the field of regenerative engineering. In the announcement, Dr. Gottlieb hits the nail on the head when describing the novel innovations in the regenerative field as a “movement past science fiction that needs a clear pathway for those developing new therapies in this promising field.” Along with recent actions addressing clinics that claim miracle solutions without empirical evidence, the streamlined process the FDA has issued with a focus on risk-based approaches combined with scientific principles is the remedy that is necessary for the field to advance authentically. As scientists here at the Institute for Regenerative Engineering at University of Connecticut working to regenerate whole limbs by 2030 among other grand challenges, we sincerely hope the FDA continues to allow innovation in the field while continuing to establish guard rails that keep the safety of patients at the forefront of discovery.

Cato T. Laurencin, MD, PhD

University professor and Van Dusen Distinguished Professor of Orthopaedic Surgery

University of Connecticut

Farmington, Connecticut

– Christopher Murdock, BSE

Research assistant, University of Connecticut

Farmington, Connecticut

Disclosure: Murdock reports no relevant financial disclosures.

Disclosures: Laurencin reports he is a stockholder/owner in Healing Orthopaedic Technologies, Healing Orthopaedic Technologies-Bone, Natural Polymer Devices and Soft Tissue Regeneration; he is a consultant for/receives royalties from Globus Medical Inc. and is a consultant for Epibone Inc.