Exploring Multi-Stakeholder Approaches
Globally there are approximately 7,000 known rare diseases. About 4,000 – 5,000 of those have no available therapies. Consequently, there are many patients with a rare disease who are waiting for a new therapy to become available.
In this white paper we explore the various pathways for pre-approval access before regulatory approval, data generation to support regulatory approval and the benefits of early engagement with key stakeholders to secure timely, sustainable access to patients in need.