EURORDIS - The Voice of Rare Disease Patients in 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.

The position paper sets out a new four-pillar approach to tackling the challenges that prevent patients’ access to care and medicines, as well as the ambition to have 3 to 5 times more new rare disease therapies approved per year by 2025, 3 to 5 times cheaper than today.

In the paper, EURORDIS calls for a new model based on a collective conversation involving all stakeholders (patients, the pharmaceutical industry, national competent authorities, national health ministries, researchers, scientists and the European Medicines Agency).