Rare Disease Policy

EURORDIS represents the voice of patients and in this capacity plays a central role in the regulatory process. The contribution of EURORDIS has been key to the adoption of important rare disease and orphan medicine legislations at the European level, including the EU Regulation on Orphan Medicinal Products, the EU Regulation on Paediatric Drugs, the EU Regulation on Advanced Therapies, the Commission Communication Rare Diseases: Europe's Challenges, the Council Recommendation on a European action in the field of rare diseases, the EU Directive on Patients’ Rights in Cross-border Healthcare, and others.

By partnering with rare disease national alliances, EURORDIS also contributes to national processes, and facilitates the adoption and implementation of national plans and strategies for rare diseases in European countries.

Two milestones in EU Rare Disease Policy

The Commission Communication and Council Recommendation have established a strategy for meeting the challenge of rare diseases.

Major Recommendations shaping RD policy

The EU Committee of Experts on Rare Diseases (EUCERD, 2010-2013) and the European Commission Expert Group on Rare Diseases (CEG-RD, 2013-2016) were established to support EU policy on rare diseases

EU Joint Actions on Rare Diseases and on Rare Cancers

Involving a broad range of stakeholders across Europe to shape policies, make recommendations aimed at ultimately improving the lives of patients.

The European Conference on Rare Diseases & Orphan Products (ECRD)

The leading conference gathering all rare disease stakeholders in Europe

Parliamentary Advocates for Rare Diseases

A network of European and national members of parliament advocating to improve the lives of people living with a rare disease.

European Reference Networks

Information on European Reference Networks & the European Patient Advocacy Groups

Patients Access to Diagnosis & Care

EU level initiatives are improving access to rare disease diagnostics and care across Europe.

Patients Access to Treatment

EU level legislation and initiatives encourage the development of rare disease medicines and foster equitable access across Europe.

Breaking the Access Deadlock to Leave No One Behind

A contribution by EURORDIS and its members on possibilities for patients' full and equitable access to rare disease therapies in Europe This position paper is a contribution from EURORDIS and its members that offers a synthesis of their analysis, reflections and perspectives on the issue of access to rare disease therapies....