Plenary Session 8 - Global Regulatory Updates and Panel Discussion & Closing Remarks

The cell and gene therapy field spans an enormous range of product types and technologies with the potential to provide curative treatments for a wide range of serious diseases. This tremendous potential has led to thousands of investigational treatments entering clinical development and many new technologies being applied to manufacture and control these complex products. Global health authorities have had to add capabilities and capacity to support the emergence of this diverse and complex portfolio of cell and gene therapy products.

In this session, we will hear from regulatory leaders from health authorities across the world about the work being done to support cell and gene therapy products and ensure timely and safe access of effective therapies to patients. Since many cell and gene therapy products are developed in rare diseases, the benefit of harmonized guidelines for global development is of particular relevance. Multiple activities on different levels are ongoing on the level of WHO and ICH to bring harmonized requirements and expectations for developers of these products. Initiatives are also being created to allow for coordination and work share across multiple health authorities to review a product. These efforts have the potential to bring efficiency to the review process for sponsors and health authorities allowing parallel authorization and facilitating access to patients around the world. Another opportunity that is gaining momentum with regulatory agencies and industry is the potential application of regulatory reliance mechanisms to allow for broader reach of a product into additional markets based on the robust review from a major health authority such as EMA, FDA, or PMDA. In this panel, we will discuss the activities being considered to enable these key levers such as workshare and reliance pathways that are key to maximizing the potential of these advanced therapies.

Panelists:

Yoshiaki Maruyama, PMDA

Steven Oh, CBER, FDA

Ilona Reischl, Austrian Medicines and Medical Devices Agency