It wasn’t too long ago that the FDA was considered one of the biggest enemies of regenerative medicine. Between onerous regulations, enforcement ambiguity, and the incredibly high cost of gaining approval for new medical devices and procedures, the FDA made it extremely difficult to get new regenerative medicine procedures to market. Things have started to change thanks to the 21st-Century Cures Act of 2016.
The overarching goal of the legislation was to streamline the federal approval process for new medical devices and drugs. By eliminating as much red tape as possible, Congress hoped to improve U.S. healthcare by making it easier to get new therapies to market. Thankfully, quite a bit of progress has been made in regenerative medicine since the act became law.
From the regenerative medicine industry’s perspective, the 21st-Century Cures Act has helped tremendously by modernizing the FDA’s clinical trial process. This is observed in two new regulatory designations: the regenerative medicine advanced therapy (RMAT) and breakthrough therapy designations.
The RMAT Designation
The RMAT designation is a special regulatory designation intended to account for many of the unique aspects of regenerative medicine. In fact, creation of the designation was seen as critical to helping regenerative medicine move forward. The unique nature of this new form of medicine is such that old designations simply do not work well.
A new regenerative medicine product or procedure can receive the RMAT designation if it meets the following two conditions:
- It modifies, reverses, or cures a serious or life-threatening disease or condition; and
- It demonstrates evidence of an ability to address an as yet unmet medical need.
In simple English, a private sector company or research organization can submit a regenerative medicine product for the RMAT designation. If the designation is awarded, the product is then eligible for an expedited review process on its way to FDA approval.
The Breakthrough Therapy Designation
The breakthrough therapy designation applies to new therapies that demonstrate significant improvement over a currently approved therapy, based on a statistically significant endpoint. The chosen endpoint is critical for gaining this designation.
The designation allows for fast tracking regenerative medicine therapies that demonstrate a measurable improvement over existing treatments. For example, steroid injections are commonly used to treat the chronic pain associated with osteoarthritis. If a new regenerative medicine procedure could demonstrate better results than steroid injections, that procedure would be eligible for expedited FDA review.
A Good Start
Utah-based Apex Biologix, a company that supplies regenerative equipment and processing kits, says that the two new designations are a good start. But they also say that the regenerative medicine industry would like to see the FDA go further. A best-case scenario would see the FDA removing any and all unnecessary barriers that currently prevent regenerative medicine research from progressing any faster.
The reality is that getting a new procedure, drug, or medical device to market can cost millions of dollars and more than a decade of research work. FDA requirements are so stringent that companies literally have to bet their financial futures on the hope that regulators will not give them the cold shoulder when the time for final approval finally arrives. This is no way to advance medical science.
Congress took an important first step when they passed the 21st-Century Cures Act. Likewise, the FDA has taken an important first step in creating a pair of new designations that should help streamline the approval process for regenerative medicine procedures. Let’s hope these first steps lead to more steps that eventually take the shackles off regenerative medicine completely.