Breaking the Access Deadlock to Leave No One Behind

A work-in-progress contribution on possibilities for patients' full and equitable access to rare disease therapies

This reflection paper is a contribution of the community of rare disease patient advocates. It is a work in-progress by EURORDIS and its members, offering a synthesis of their analysis, reflections and perspectives on the issue of access to orphan medicines.


The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases