For nearly 20 years, regenerative medicine such as cell and gene therapies have gradually shown much promise in repairing or replacing damaged and infected cells, tissues and organs in order to cure ailments including organ failure, spinal cord injuries, multiple sclerosis and other traumatic wounds and diseases.
While many regenerating treatments continue to be developed, the U.S. Food and Drug Administration (FDA) has approved only a small number of possible cures in the United States. Holly Fernandez Lynch, J.D., MBE, University of Pennsylvania’s Perelman School of Medicine Presidential Assistant Professor of Medical Ethics, told FDA Reporter that the FDA must be paying close attention to regenerative products that may not be safe for patients if so few are being authorized.
“[The FDA] has a legal mandate to make sure that products are safe and effective prior to marketing approval, a mandate that is very important to protecting patients from unsafe and ineffective products,” Fernandez Lynch said. “Considering the numerous examples of unscrupulous stem cell clinics that have been reported in recent years, I think FDA regulation is critical to protect patient interests.”
The Pew Research Center predicts an increase in approved products within the next several years, citing successful treatment trials previously performed in many medical clinics and the FDA already pledging to increase enforcement against “high-risk, unapproved interventions” being provided, among other reasons. While some of the public may believe the FDA works rather slowly in approving certain treatments, Fernandez Lynch hears frequently that FDA approval standards have slipped in recent years, calling it “problematic” for patients.
“There is always a delicate balance between speedy product approvals and ensuring patient safety,” Fernandez Lynch said. “Some claim the FDA is too slow, but increasingly, the arguments I am hearing more often are that the FDA has lowered its approval standards, requiring less and less evidence for product approval.”
Fernandez Lynch is hopeful for more regenerative therapies to be FDA-approved, but wishing for tighter regulation and oversight over these advanced products.
“Overall, I would absolutely not hold regenerative medicine out as an area in which the market has done an adequate job promoting patient interests,” Fernandez Lynch said. “Regulation and oversight are sorely needed.”