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 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.
Latest News and Insights
Exploring the often-overlooked role of Medical Science Liaisons (MSLs) in Early Access Programs (EAPs)
Around two billion people lack regular access to essential medicines. In the poorest regions more than half the population is affected