Over 250 rare disease stakeholders gathered in Brussels on 13th & 14th February to develop and endorse Eurordis’ new proposed framework to sustainably improve patient access to rare disease therapies.
Mencía de Lemus, President of SMA Europe, delivered a clear message. Despite the speed with which treatments are being developed for SMA, many challenges still present themselves with just 20% of patients across Europe accessing today’s only therapy, Spinraza™. Although some countries benefit from full access, others, not at all. Eurordis' framework will offer solutions and set out commitments by all players in the rare disease community. Once adopted, it will be presented to the European Parliament ahead of the elections in May 2019, the European Commission and the Council of European Health Ministers, calling upon them to sign-up to the framework, implement the content and/ or continue the dialogue in the appropriate arena, at both European and national level.