Expediting Access to Rare Disease Therapies in Europe

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.

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