Project Management Consultant Integrated Project Management, Inc.
Description: Gene therapies offer the unique opportunity to cure previously incurable diseases demanding regulatory and innovators to trailblaze new guidance and information to support achieving this goal safely and effectively. Gene therapy regulatory strategies are key in supporting successful regulatory outcomes and can be influenced by factors such as models and surrogate models, patient populations and primary endpoints that are accessible and acceptable to health authorities. In this session the discussion will focus on current approaches to planning gene therapy regulatory strategies, gaps in regulatory guidance, and recent success and challenges currently experienced in the US. Finally, an open discussion on current or related regulatory experiences will be discussed to level set the community on recent interactions to support planning regulatory strategies in these innovative life changing therapies.
Learning Objectives:
List three characteristics of a successful gene therapy regulatory strategy
Explain three key challenges to gene therapy regulatory strategies
Describe two key gaps in understanding at academic and regulatory levels requiring further development