International – Regulatory Intelligence: Update on Regenerative Medicine Advanced Therapies Designations

This article discusses the scope and purpose of the special designation for Regenerative Medicine Advanced Therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT, such as keeping the US globally competitive in the field. They provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.

Introduction

Section 3033 of the 21st Century Cures Act, titled “Accelerated Approval for Regenerative Advanced Therapies,” created a special designation for Regenerative Medicine Advanced Therapies (RMAT). A product is eligible for RMAT designation if it is a regenerative medicine therapy, such as cell therapy, therapeutic tissue engineering product, human cell and tissue product, gene therapy or any combination product using such therapies or products, and is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.
 
Other national bodies have in recent years made special regulatory provisions to recognize the value of regenerative medicine products. For example, Japan’s SAKIGAKE designation,  was introduced in 2015 to promote R&D of regenerative medicines and other innovate pharmaceuticals and medical devices.
 
RMAT designation has helped keep the US competitive in the global field, while addressing specific needs and requirements of regenerative medicine advanced therapy products. The benefits of an RMAT designation are the same as the benefits for breakthrough therapies and include interactions with FDA to expedite development and review of the product and consideration of the product for priority review or accelerated approval. Receiving RMAT designation also allows for increased flexibility in clinical trial design, for instance, in the number of clinical trial sites. Developers also have the potential to use patient registry data and other real-world sources in post-approval pathways.

While developers seeking breakthrough designation are required to show that their therapeutic candidate would provide a substantial improvement over existing therapies, RMAT designation only requires that the therapy have the potential to address unmet medical need…