Inceptua’s Site Order Portal is a web-based tool that allows Sponsors to match the supply of medicine to real-life patient recruitment
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.
Umbrella Access Programs (UAP) – A Pioneering, Multi-Stakeholder Approach to Early Access
Heather Manna, VP, Global Regulatory Affairs, Inceptua Early Access, answers questions about Real World Data and Early Access.