Lessons learned from RMAT submission, denial and successful resubmission
A successful strategy for obtaining the US FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation requires a strong understanding of the FDA/CBER defined designation criteria and potential pitfalls. Equally important to the RMAT strategy is understanding how/if/when regulatory flexibility applies, including GMP challenges to overcome and understanding of the effect of potential government agency interactions. Attendees will gain a better understanding of the possible outcomes from RMAT designation requests, be better positioned to determine if a clinical indication is ready for request of RMAT designation, understand how they can use RMAT designation to expedite clinical development, and explore additional avenues for product development.
What will you learn?
- Understand key content to submit for RMAT consideration including clinical data requirements
- Identify potential flexibility around clinical and CMC approaches and associated challenges
- Understand regulatory options that exist for RMAT designated products including increased interactions with the FDA
- Understand the sequence of meetings that follow the RMAT designation
Who may this interest?
- Biopharmaceutical executives
- Regulatory professionals
- Cell and gene therapy investors
- Biopharma clinical science professionals
Director of Regulatory Affairs
Stephen Westover is the Director of Regulatory Affairs at Cook MyoSite Incorporated, where he is the point of contact with health authorities for the clinical investigation and eventual commercial licensing of cellular therapy products. He also oversees the regulatory interactions and submissions to health authorities including the US Food and Drug Administration (US FDA), Health Canada and European competent authorities. Stephen is a certified manager of quality and operational excellence through the American Society of Quality (ASQ) and is certified in RAC Drugs through the Regulatory Affairs Professionals Society (RAPS).
Manager, Regulatory Affairs
Brett Snyder started at MyoSite in January 2010 in the QA department, moved into Compliance, and finally into Regulatory Affairs. In her tenure at MyoSite, Brett has helped grow systems to fit cGMPs throughout the organization, helped regulatory move from paper submissions to electronic submissions, and is putting systems in place for the department to become an industry leading group. Brett is a certified quality auditor (CQE) through the American Society of Quality (ASQ) and certified as a regulatory affair professional (RAC) through RAPS.