RE:RE:RE:Interesting past news...And finally....the "21st Century Cures Act" ..... is this where we are at???....
The RMAT program is housed at CBER. Products granted designation are eligible for increased early interactions with the FDA, including all the benefits available to breakthrough therapies. Because these are considered investigational products, the FDA does not identify which products have gained RMAT designations unless the company makes an announcement itself, as Mallinckrodt did with StrataGraft.1 In November 2017, the FDA released two draft guidance documents describing the expedited programs available to sponsors of regenerative medicine and describing how CBER will encourage flexibility in clinical trial design to facilitate the development of data to demonstrate the safety and effectiveness of regenerative medicine therapies. The first addresses how the FDA intends to simplify and streamline its application of the regulatory requirements for devices used in the recovery, isolation, and delivery of RMATs, including combination products.7 The second draft document describes the expedited programs that may be available to sponsors of regenerative medicine therapies, including priority review and accelerated approval.8
Michael Werner, of ARM, explains that the RMAT designation is significant for two reasons. First, it sends a signal to the regulated industry and patients waiting for therapy that there will be a specific pathway for new products. There wasn’t one before. That puts the U.S. on the same footing as countries such as Japan, which have been more aggressive. Second, the designation allows a company to have meetings early in the clinical trial process with the FDA, which are, according to Werner and many others, “a real factor in determining success.” Designees also acquire the ability to use RWE, which is particularly important where a chemical is seen as having an impact on a rare disease but where recruitment for large clinical trials is close to impossible. There is also access to expedited approval pathways.
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