FDA releases comprehensive regenerative medicine policy framework; draws support from ISCT and ISSCR

International Society for Cellular Therapy and International Society for Stem Cell Research have announced support for the new guidelines.

The FDA recently released new comprehensive guidelines describing how products should be regulated as drugs, devices or biological products and proposed new processes for determining safety and efficacy. Following on from recent controversy around unregulated stem cell clinics and ‘pay-to-participate’ clinical trials and new approvals for cell-based therapies, these new guidelines have been hotly anticipated.

“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,” said FDA Commissioner Scott Gottlieb.

The framework consists of two final guidance documents and two draft guidance documents:

• The first final guidance document clarifies when cell or tissue-based therapies would be exempted from established regulations if they are removed from and implanted into the same individual within the same surgical procedure and remain in their original form.
• The second final guidance discusses how the FDA agency interprets the existing regulatory definitions of “minimal manipulation” and “homologous use”.
• The first draft guidance document builds on regenerative medicine considerations outlined in the 21^st Century Cures Act, discussing how the FDA seeks to streamline its application of regulations surrounding medical devices for the recovery, isolation and delivery of regenerative medicine advanced therapies.
• The second describes the myriad expedited programs that are available to new regenerative medicine therapies, as well as which therapies may be eligible for the Regenerative Medicine Advanced Therapy (RMAT) designation.

These new guidelines have been welcomed by the International Society for Cellular Therapy (ISCT) and International Society for Stem Cell Research (ISSCR) who released statements commenting on these new documents.

Find out more about US regulation of regenerative medicine therapies:

• “Pay-to-participate” stem cells studies: an interview with Leigh Turner
• Supporting the FDA’s tougher stance on unproven cell therapies: an interview with Massimo Dominici
• Texas expands patient access to unproven stem cell therapies

“This new FDA effort closes significant regulatory loopholes that have been exploited by businesses promoting unproven treatments that have not been proven safe nor effective for patient use,” explained ISSCR President Hans Clevers. “It puts clinics on notice that the agency is paying attention and asserting its authority,” he continued.

“ISCT welcomes the efforts of the FDA to provide additional regulatory clarification for all those operating in the cell therapy field,” commented Catherine Bollard, President, ISCT. However, she cautioned: “it is essential that the FDA is now cautious and measured in its introduction and application of the framework. The FDA needs to balance bringing those operating outside the regulatory pathways to compliance, taking action against those that remain outside the licensing and regulatory frameworks, and continuing to foster the ongoing innovation and considerable potential for the majority of the sector operating within the regulatory frameworks.”

Sources: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/UCM585345.htm, ISCT press release, ISSCR press release