Biologics therapies have the promise to revolutionize orthopedics and sports medicine, but unauthorized operators pose a serious threat to emerging therapies.
Randy Hubbell, CEO of biologics company Carmell Therapeutics, is working to develop and commercialize plasma-based bioactive materials. Mr. Hubbell and the company hope their therapeutic products can usher in a new era of regenerative medicine.
Here, he discusses the challenges facing biologics and elaborates on what the FDA is doing to regulate the development of new therapies.
Note: Responses were edited for style and clarity.
Question: Why are regenerative medicine operators dispensing treatments without FDA approval?
Randy Hubbell: The FDA allows for certain tissue-based materials to be used in patients without a regulatory approval process, called a "361," named for section 361 of the Public Health Service Act. There are definite criteria that must be met to be a 361 product and some companies, clinics and doctors are misinterpreting these criteria, either knowingly or not.
Q: What sort of benefits could biologics have to the orthopedic market?
RH: The orthopedic market has reached its limits on what can be accomplished mechanically with traditional medical devices. "Active" therapeutics in the form of drugs or biologics delivered directly to the injured site are the future for improving, accelerating and enhancing healing. This was accomplished in the cardiology space when Johnson & Johnson launched the world's first drug eluting stent, CYPHER, in 2003. Now, there are many applications in the heart and peripheral vasculature where drugs are being delivered locally to improve outcomes.
Q: Why is a more rigorous review process needed?
RH: There are increasing demands for safety and efficacy, as well as assurances that claims are substantiated. As an example, hospitals have been asking medical technology companies to demonstrate new technology that has gone through a rigorous regulatory process to ensure that the product is safe and effective, and equally important, that it does what the manufacturer says it does. Ninety-five percent of the medical devices are class 1 or class 2. The class 2 devices only need to show that they are substantially equivalent to something that has already been approved. That makes it difficult for hospitals, doctors, payers and patients to understand why a new technology is declared to be better by the manufacturer, when the 510(k) process says it is the same as another product already approved.
Q: Realistically speaking, how long do you think it'll be before the healthcare market has easily accessible biologics-based treatments?
RH: The market is very fragmented today, with products that have little regulatory review, but still make substantial claims on their benefits. This, at a minimum, could be a waste of money for the patient, and in some cases, could be dangerous. There are very few regenerative medicine technologies in the orthopedic space that have gone through the most appropriate approval process for a biologic: a biologic license application. To date, there are no BLAs for orthopedics bone healing and just a couple for wound-healing.
Q: How will Carmell Therapeutics usher in those treatments?
RH: Carmell's technology is a biologic delivered directly to the injured site to accelerate and enhance healing after surgery and major injury. This requires a very rigorous and demanding regulatory process, of which Carmell has already completed a substantial amount. Our product will be supported by two confirmatory phase 3 studies demonstrating superiority over standard of care. We are currently on a path to be the first company in the orthopedic bone healing space with a BLA supported by two prospective, randomized, multicenter clinical studies.