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Developers of regenerative medicine therapies will have greater flexibility in the FDA regulations to test their products and can expedite reviews of treatments for serious conditions.
The Food and Drug Administration unveiled Nov. 16 a new regulatory framework to oversee stem cells and other products designed to repair or replace damage tissue or organs, updating regulations that went into effect in 2005. FDA Commissioner Scott Gottlieb said it’s a move to account for an advancing field and to clarify when premarket activities such as clinical trials and surgeries would be subject to the FDA’s regulations. At the same time, the agency will continue to crack down on stem cell clinics that regulators believe are peddling unapproved and potentially harmful treatments.
“In the last decade, we’ve seen improbable advances that hold out great hope for patients,” Gottlieb said during a Nov. 16 media briefing. “With the ability to facilitate the regeneration of parts of the human body, we’re bearing witness to the beginning of a real paradigm shift in the practice of medicine.”
The announcement doesn’t introduce any new laws or regulations but modernizes the agency’s interpretation of them through four guidances, he said. “Our aim is to make sure we’re being nimble and creative when it comes to fostering innovation, while also taking steps to protect the safety of patients.”
R. Alta Charo, a University of Wisconsin-Madison bioethicist and former FDA policy adviser, told Bloomberg Law the plan reassures the clinical community that many existing therapeutic interventions won’t inadvertently be swept into a new regulatory category, such as bone marrow transplants that currently meet the surgical exceptions. Charo was co-chairwoman of the National Academies committee that wrote some of the first guidelines on embryonic stem cell research.
The four guidances are:
Michael Werner, co-founder and senior policy adviser for the Alliance for Regenerative Medicine, called the framework a significantly positive development for patients and their families. “Therapeutic developers now have further clarity on the regulatory process requirements to meet the FDA’s high product approval standards,” Werner told Bloomberg Law in a Nov. 16 email. “The ongoing implementation of the RMAT designation also is a boon to regenerative medicine and advanced therapy manufacturers.”
Leslie Miller, chairman of the Alliance for the Advancement of Cellular Therapies board, told Bloomberg Law the new framework responds in part to FDA public hearings. Miller is the chief medical officer for Santa Monica, Calif.-based Leonhardt Ventures, which operates laboratories for regenerative medicine technologies.
“These new guidelines provide some clarification of the important topics of minimal manipulation and homologous use,” Miller wrote in a Nov. 16 email expressing the alliance’s support for the framework. “We all want to see only the highest standards applied to use of stem cells.”
The framework also includes a proposal to allow individual academic investigators who follow the same manufacturing protocols and share combined clinical trial data in support of approval from the FDA to work under the same biological license application.
For the next three years, the FDA will exercise enforcement discretion for low-risk products that don’t comply with the guidance, Regulatory Considerations for Human Cell, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use .
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said this grace period allows sponsors to have a dialogue with the agency, and if necessary, file the appropriate regulatory documentation as soon as feasible to allow sufficient time for the application to process. “The level of clarity provided by the two final guidance documents may cause reconsideration of the regulatory pathway that many sponsors thought applicable to their products,” he said on the same media call as Gottlieb.
Lower-risk products include those administered by a syringe just underneath the skin. The FDA will prioritize enforcement of higher-risk products, such as those injected into a vein or applied directly to the central nervous system. “FDA does not intend to exercise such enforcement discretion for those products that pose potential significant safety concerns.”
Calling this grace period “extremely generous, Charo said it’s not enough to pursue the most risky interventions. Patients need to be better informed on the status of the interventions, on the basis on which clinics have reason to believe the intervention is safe and effective, and on how they retain their legal right to complain if there has been negligence. “No patient should be denied access to a remedy in case of injury by virtue of a waiver buried in the consent they give to the intervention,” she said, adding these are generally matters of state law, and aren’t reached by FDA’s guidances.
The framework follows an announcement Gottlieb made in the summer as part of an FDA crackdown on rogue stem cell clinics. The FDA will continue to go after these clinics, and he said the new framework provides a better understanding of the requirements when conducting work in regenerative medicine. “By exploiting the lack of consumer understanding in this area as well as the fear and uncertainties posed by the diseases these bad actors claim to treat, they’re jeopardizing the legitimacy and the advancement of this entire field,” Gottlieb said.
In addition to administrative actions such as warning letters, Marks said, the FDA can ask for court injunctions or seizures, or pursue criminal actions. “We intend to use the full range of enforcement provisions provided to us in order to make sure bad actors do not continue doing what they’re doing,” the CBER director said.
Charo said the guidances clearly identify the limits of the exceptions that have been abused by clinics offering unproven stem cell interventions. The guidances put clinics on notice that using a patient’s own cells doesn’t automatically exempt them from premarket requirements and lets them know use of a patient’s own cells to perform a function different than their original function doesn’t fall under the “homologous” defintion that exempts these activities. “Together, this should put an end to clinics claiming that, for example, extracting stem cells from adipose tissue and injecting them into joints is somehow outside FDA’s purview,” she said. “The guidances make clear that just this sort of practice falls squarely within the definition of a regulated medical product because it is more than minimally manipulated.”
To contact the reporter on this story: Jeannie Baumann in Washington at firstname.lastname@example.org
To contact the editor responsible for this story: Randy Kubetin at RKubetin@bna.com
The FDA's regulatory framework is available at https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm585218.htm.
Copyright © 2017 The Bureau of National Affairs, Inc. All Rights Reserved.
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