Heather Manna, VP, Global Regulatory Affairs, Inceptua Early Access, answers questions about Real World Data and Early Access.
Expediting Access to Rare Disease Therapies in Europe
- by flowerhaus
- | April 9, 2019
- Early Access, White Paper
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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