Spotlight session: Defining the right endpoints and measuring clinical benefits in advanced therapies

Endpoint selection is a critical step in demonstrating clinical benefit and securing regulatory approval for advanced therapies. With small patient populations and high variability, developers must adopt rigorous frameworks to identify relevant, patient-centered measures and validate them effectively. This session draws on lessons from developers navigating these complex decisions. 

Key Takeaways:  

• Define clinically relevant and patient-centered endpoints in rare or variable populations 

• Apply strategies to validate novel endpoints within small cohorts 

• Understand regulatory expectations for endpoint justification in advanced therapy trials 

• Leverage early patient and clinician input to improve endpoint selection 

• Learn from case studies of developers overcoming endpoint challenges