On February 22-23 Johan Prévot, IPOPI’s Executive Director, participated in the 2nd Multi-Stakeholder Symposium on Improving Patients’ Access for Rare Diseases. The Symposium brought together numerous participants from various stakeholder groups to express their concerns and interests in improving patients’ access to rare disease medicines.
Key topics discussed during the meeting included the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA), the new European Reference Networks and their role in generating quality data, the future of EU-level cooperation on health technology assessment as well as between payers and companies, pan-European disease registries, and innovative performance-based outcome agreements.
The Symposium called for a three to fivefold increase in the number of rare disease medicines approved by the European Medicines Agency and the US FDA per year by 2025 and for those medicines to be available at one third to one fifth of the price.
The meeting concluded with the announcement of a new multi-stakeholder group that will draft a Collaborative Action Plan for all parties to collaborate in a process to improve patients’ access to RD medicines.