“Effectively Collect Real World Evidence for Early Access Programs”. We have asked Stuart a few questions to reflect on the topic …
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.
As we are strengthening our offering and presence in China, we are delighted to present Ryan Zhang as Regional Director Business Development.
Inceptua’s Site Order Portal is a web-based tool that allows Sponsors to match the supply of medicine to real-life patient recruitment