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FDA to create clearer framework with regenerative medicine guidance documents
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In 2005, the FDA established and implemented a regulatory framework for the use of cell-based therapies in regenerative medicine. However, advances in the field of regenerative medicine over the last decade have led to challenges to researchers, health care providers and the FDA. To provide a clear, efficient pathway for product developers, as well as ensure the safety and efficacy of these medical products, the FDA recently released four guidance documents—two finalized and two draft documents—that build upon the existing regulatory approach by more clearly describing what products are regulated as drugs, devices, and/or biological products.
“We wanted to set forth guidance about what the threshold was between products that would fall within FDA’s regulatory scope and would require premarket approval prior to going to market and those that were not,” Scott Gottlieb, MD, commissioner of the FDA, told Healio.com/Orthopedics.
Creating a clearer framework
While the guidance documents cover multiple criteria of the regulatory framework, Gottlieb said two of the most significant criteria include defining what constitutes minimal manipulation of a cell and implementing components of the 21st Century Cures Act.
“The way we think of the totality of this package is that by creating a clearer framework in guidance, establishing where the line is between FDA regulation, we provide more consistency and predictability to the market place for people who want to develop products responsibly and want to know whether or not they fall across the line of demarcation defining whether or not they are going to be regulated by the FDA,” Gottlieb said. “At the same time, it gives us the ability for those who are crossing the line and creating patient risks to take enforcement actions.”
With some selective enforcement actions already enacted by the FDA in the past, Gottlieb noted the possibility of additional enforcement actions in the future for products that are “creating substantial risks for patients” To reduce the risk enforcement actions being taken, the FDA invites individuals to discuss where they stand in the development of regenerative medicine products over a 36-month period.
“If they think that they are going to be subject to regulation, we want them to come in and talk to use to understand whether or not in fact that is true because we think that, even with the guidance, there is still going to be some uncertainty about where different products stand, but also to understand what that regulation would look like,” Gottlieb said.
New approval processes
One process that is different includes the allowance of individual investigators to pool their data collectively receive FDA approval, Gottlieb noted.
“In cases where you have ... an academic institution that is developing a product where they might not see enough patients or be applying this product to enough patients in order to reach a sufficient number to file an application with the FDA ... in those cases, if the researchers want to band together so long as they are following a common manufacturing protocol, they can band together and pool their data and submit one comment application and then each individual investigator would get an individual FDA approval,” he said.
The importance of dialogue
With the implementation of the guidance documents, Gottlieb noted the FDA intends to to educate academic institutions and companies working with regenerative medicine products on the parameters of the guidance. The FDA also welcom feedback on the draft guidance documents through a 90-day comment period hopefully tailor the “traditional regulatory approach in a unique way that is properly matched to, not just the kind of technology that regenerative medicine represents, but also the way the field is developing” according to Gottlieb.
“It is important for us to have a dialogue with the medical community, with physicians, with academic centers who are developing these kinds of products to say we are interested in making sure that this framework gets implemented in a way that allows innovation to go forward and also allows it to go forward in a way where we are adapting our regulatory approach to the unique sort of opportunities and challenges offered by this field,” Gottlieb said. – by Casey Tingle
Reference:
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585345.htm
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585342.htm
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Cato T. Laurencin, MD, PhD
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