Explore 15 years of RWD collection in Early Access Programs, its evolution, regulatory challenges, and growing role in pharma strategy.
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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The easiest way to describe what we do is that there is a patient in need, a medicine available, and we are the bridge between the two.
Exploring the often-overlooked role of Medical Science Liaisons (MSLs) in Early Access Programs (EAPs)