Edition of 20 October 2022

In the context of the Czech EU Council Presidency, an expert meeting entitled “Towards a New European Policy Framework: Building the future together for rare diseases” will be held on 25-26 October in Prague. This conference will bridge a series of technical and ministerial gatherings on proposals for a renewed strategy for rare diseases following the Slovenian and French presidencies. This will be an opportunity 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.

Strengthened cooperation and coordination of Member States could be outlined in an European Action Plan for Rare Diseases that addresses remaining gaps by combining and supplementing current legislative actions (e.g. revisions of legislation of Orphan Medicinal Products Regulation, Paediatric Use of Medicines and adoption of the Pharmaceutical Strategy); non-legislative policy actions; and new Commission initiatives such as the European Health Data Space and the European Pillar of Social Rights Action Plan.

This conference will mark a major milestone in the proposal for a policy framework for rare diseases following the conclusions of  the European Court of Auditor’s report n°7/2019, the cross-sector consensus from over 250 stakeholders in the Rare 2030 Foresight Study, the 43 cosignatory members of the European Parliament in their letter of support for Europe’s Action Plan, the recognition of the “undeniable benefit” of stronger cooperation during the Informal meeting of Ministers of Health in Grenobleand the support of thousands of patients and key opinion leaders and policy makers presented at the High Level Ministerial Conference: Care and innovation pathways for an EU policy on rare diseases in support of the proposal for a European Action Plan on Rare Diseases.

The conference will focus on 5 priority areas:

• Block I. – A new goal-based and coordinated strategy for rare diseases
• Block II. – Early Diagnosis for Rare Diseases
• Block III. – Revision of the Orphan Drug and Paediatric Drug Regulations
• Block IV. – Instruments for improving access to treatments for rare diseases
• Block V. – Holistic healthcare pathways: Integrating European Reference Networks into European health care and social systems