Tutorials are not included in the meeting registration and require a separate registration fee

Participants in this event will join the afternoon sessions of the meeting “Advancing the Science of Study Endpoints: Seeking Practical Solutions” for an in-depth view of the unique challenges of selecting and identifying study endpoints for rare disease clinical trials.

Experts will discuss strategies for demonstrating treatment benefit through the selection of optimal study endpoints and review key considerations for development and implementation of underlying assessments. Analysis and interpretation of endpoints will also be addressed. The session will explore adaptation of standard methods of study endpoint development to preserve scientific best practices while accommodating common issues facing rare disease development programs, such as limited knowledge of natural history of the disease, lack of treatment precedent, and absence of existing tools to evaluate treatment benefit. The perspectives of key stakeholder groups will be represented as we explore the many regulatory and clinical challenges, and address commercial considerations.