Description: Advanced therapies have garnered increasing interest in recent years due to their potential to treat and/or cure rare and life-threatening diseases. A myriad of innovative technologies is being developed – often conceived during bench research. Few products have been approved globally. However, the use of increasingly innovative technologies also provides for special operational and regulatory challenges at any stage of the product development. Clinical studies have been put on hold due to emerging safety issues. Although they are in most cases extremely rare, there may be need for further preclinical exploration. Regulatory agencies have emphasized the need to establish product quality early in the development and insist on the need to use the intended commercial product for pivotal clinical studies. Innovative trial designs such as using umbrella trials in early development could support faster development programs. How can developers address regulatory requirements early and how can interactions with regulators support the product development program from very early stages through approval and beyond? The session will discuss innovative advanced therapy technologies and provide case studies on challenges, pitfalls, hurdles, successes and lessons learned by industry stakeholders. Session speakers offer their insights and experiences in tackling challenges and opportunities of unique product development strategies. How are the regulatory considerations applied, interactions with regulators utilized and what future regulatory expectations are anticipated? The audience will have ample time to ask questions and support the session by providing responses to polls throughout the session, discussing their experience during the final panel discussion.
Learning Objectives:
Understand the regulatory, development and operational challenges associated with advanced therapies
Describe potential challenges that may be identified during an ongoing development program early
Identify regulatory support options to optimize development plans for time and cost savings