This expert meeting Towards a New European Policy Framework: Building the future together for rare diseases bridges a series of technical and ministerial gatherings on proposals for a renewed strategy for rare diseases following the Slovenian and French presidencies.
In the spirit of continuity stakeholders in the rare disease community are particularly keen to continue discussions on rare diseases and focus on how the Czech EU Council Presidency can take continued steps towards a coordinated strategy for rare diseases that better addresses current unmet needs by setting meaningful goals for patients, families and for society at large integrated at the national and regional levels.
Despite tremendous progress demonstrated by the measures already implemented and the ongoing commitments and major investments in addressing the challenges of rare diseases from the side of the European Commission, the need for an updated framework of EU actions and support for national plans and strategies on rare diseases remains. The 2008 Communication on rare diseases which aimed to “encourage cooperation between the Member States and set out an overall Community strategy for support to Member States” was a cornerstone legislation for today’s progress, but drafted in an era during which scientific breakthroughs, technological potential and Community crisis and values were not the same as today.
Today’s strategy must strengthen the cooperation and coordination of Member States with regards to rare diseases by combining the critical building blocks discussed during this conference that make up the rare disease ecosystem along the patient journey (diagnosis, centres of expertise, treatment, and social care) and the life cycle of innovative solutions from “bench to bedside”.