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.

Download the full
white paper here