Developers submitted more than 173 applications for RMAT designation to the FDA from 2017 to 2021, but only 66 of those applications — less than 40 percent — were granted this exclusive designation during that same time frame. As reflected in this statistic, we find developers of cutting-edge advanced therapies such as cell and gene therapies (CGTs) are encountering new challenges as they navigate complex clinical and regulatory hurdles.
Indeed, there is no formal playbook for CGT development due to the unique patient-specific nature of each dose and potentially limited patient populations for each therapy. However, our regulatory experts have the experience needed to best prepare CGT companies applying for RMAT designation. Learn 7 key areas of focus when applying for RMAT designation.