Global Tools to Expedite Advanced Therapy Product Development

Description: Advanced therapies, such as cell and gene therapies, are a group of highly innovative products that is growing at a rapid pace. Many of these products are designed to treat and/or potentially cure life-threatening and less common or rare diseases. Developers increasingly aim to develop these products for the global market since there are fewer patients to treat on the national level.

Due to the complexity of advanced therapy development, various global regulators have implemented programs and tools to expedite these development programs. For global developers, however, this has resulted in the need to navigate a heterogeneous array of global frameworks and programs. For example, the U.S. offers the RMAT and breakthrough therapy designations, the E.U. offers the PRIME scheme and the U.K. initiated the Innovative Licensing and Access Pathway.

This session will provide an overview of these various expedited pathways and tools, with a focus on applicability and potential benefits. Industry experts will share their experience with expedited advanced therapy pathways across multiple jurisdictions, addressing questions such as: Which designations were more efficient to obtain? Which provided the most benefit? What were the challenges and opportunities for each pathway/tool?