Navigating Global Regulatory Frameworks for Cell and Gene Therapy Development

The potential of advanced therapies to treat and/or cure many rare diseases and life-threatening conditions has drawn the industry’s interest for product development. The number of innovative technologies under development has increased almost exponentially over the past few years. Additionally, medicinal product developers increasingly aim to develop investigational products for the global market in part because some of these therapies may only treat a few patients globally. However, global development also means navigating a heterogeneous array of global frameworks for advanced therapies.
There are differences, for example, between how the U.S. FDA regulates advanced therapies and how they are regulated in Europe. In the E.U., new legislative proposals currently being examined by the European Parliament and Council may result in additional new requirements. Expedited programs can accelerate therapy development. Each jurisdiction has their own support systems (e.g., U.S. breakthrough therapy and regenerative medicine advanced therapy designations, the E.U.’s PRIME pathway).
The session will provide a brief overview of the regulatory frameworks for the U.S. and E.U. The experts will also discuss the benefits, regulatory support, expedited pathways and challenges of each framework, including potential new legislation.

* One lucky attendee will win a copy of RAPS Regulation of Regenerative Medicines: A Global Perspective book.