FDA on Thursday announced a new framework for regulating gene and cell therapy treatments—releasing two draft guidance documents and two final guidance documents on the oversight of such products.
According to CQ HealthBeat, the draft and final guidance documents are not legally binding but aim to help device and pharmaceutical companies navigate FDA requirements. The two draft guidance documents will be subject to comment periods.
According to the Times, the guidance issued Thursday is intended to help drug and medical device developers determine whether a product requires an FDA licensing application for premarket approval or whether it qualifies as "lower risk" and does not need such approval.
FDA in draft guidance described plans to make regenerative medicine therapies for serious medical conditions eligible for its expedited review and approval processes. FDA in the draft guidance stated expedited review would be available for gene therapies "to treat, modify, reverse, or cure serious conditions." The process, according to the Times, would still require clinical trials.
FDA in another draft guidance document outlined the agency's plans to evaluate medical devices involved in the "recovery, isolation, or delivery of regenerative advanced therapies." According to the draft guidance, FDA plans to simplify application requirements for combination device and cell or tissue products, describing "the least burdensome approach to demonstrate how a device may be used with more than one cell type."
FDA in final guidance stated that tissue establishments and health care professionals do not need FDA approval to remove cells or tissue from a patient and transplant them back into the patient "without intervening processing steps beyond rinsing, cleansing, sizing, or shaping, [because such a procedure] raise[s] no additional risks of contamination and communicable disease transmission beyond that typically associated with surgery."
FDA in second final guidance clarified FDA's oversight of products designed to manipulate cells or tissue.
FDA says it will target clinics offering unapproved stem cell treatments
FDA in the announcement said the agency plans to take a "risk-based framework … to focus its enforcement actions against … products that raise potential significant safety concerns," such as unapproved stem cell treatments.
FDA Commissioner Scott Gottlieb in a statement Thursday said, "[T]he rapid growth and promise of this field has increasingly sowed the ground for the entry of some unscrupulous actors, who have opportunistically seized on the clinical potential of regenerative medicine to make deceptive claims to patients about unproven and, in some cases, dangerous products."
Gottlieb said, "By exploiting the lack of consumer understanding of 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 advancement of the entire field [which] … underscores the importance of having a clear regulatory framework for developers, and ensuring that those who skirt these regulations are held accountable."
For example, some clinics claim to offer stem cell injections to treat dozens of medical conditions, such as Alzheimer's disease, arthritis, and multiple sclerosis. In some cases, patients have lost their eyesight after having cells derived from fat implanted back into their bodies, according to FDA.
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As part of FDA's effort to crack down on such procedures, the agency issued a consumer update advising patients to determine whether a procedure has been approved by FDA before undergoing treatment.
Peter Marks, FDA's director of the center for biologics evaluation and research, said under the new framework, health care providers would not get "a free pass to do anything they want." He said FDA's "goal … is not to flood the market with products" but rather "to get products on [the market] that are safe and effective, and to clear up the field so that people know what they have to do."
Michael Werner, a partner with Holland & Knight and co-founder of the Alliance for Regenerative Medicine, said, "This is an incredibly significant development for the gene therapy, regenerative medicine sector." Werner said, "This should really open the door to a lot more gene therapies coming on the market quickly."
However, Michael Carome, director of Public Citizen's health research group, said he "think[s] there is excessive hype." Carome said, "We are talking about rushing to market very complex biologics products where we are still in the infancy of this field."
Paul Knoepfler, a stem cell researcher, called FDA's announcement a "positive sign" but said it remains to be seen whether the agency will be able to follow through with needed enforcement. Knoepfler said, "Now that the FDA's policies are clear, will it back them up with action?" (Kaplan/Grady, New York Times, 11/16; Siddons, CQ HealthBeat, 11/16 [subscription required]; AP/Sacramento Bee, 11/16; FDA release, 11/16; Gottlieb statement, 11/16; FDA draft guidance, 11/16 ; FDA draft guidance, 11/16 ; FDA final guidance ; FDA final guidance ).
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