EFPIA-EURORDIS Joint statement on Patient Access to Medicines for Rare Diseases

Today marks the publication of the EFPIA-EURORDIS Joint statement on Patient Access to Medicines for Rare Diseases. The statement is based on an 18-month structured dialogue between the two organisations focused on identifying proposals that can improve access to orphan medicines across Europe. At the same time the statement recognises areas where the two organisations have different perspectives.

Speaking about publication of the statement, EFPIA Director General, Nathalie Moll said. “EFPIA and EURORDIS share the goal of faster, more equitable access to medicines for patients living with rare disease across Europe. Both the structured dialogue and today’s Joint Statement are a proactive effort to find solutions to access issues together. We did not agree on all points but found enough common ground to bring forward a series of concrete proposals that we believe can make a real and tangible difference to Europeans living with rare disease.”


In the statement, both EFPIA and EURORDIS propose the consideration of a conceptual framework for Equity-Based Tiered Pricing (EBTP) to better align medicines’ prices with countries’ ability-to-pay. The statement includes a number of proposals, supported by both organisations aimed at improving Health Technology Assessment (HTA) and Pricing & Reimbursement processes, both at EU and Member State level. Recognising the significant number of rare conditions where no treatment options exist, the final proposal focuses on the launch of a Moonshot to stimulate translational research for the development of medicines that will address some of the remaining unmet medical needs of rare disease patients.

“Often European and national policies cannot progress in their will, because the analysis of the issues is unclear, the solutions are not identified, or there is no consensus among the stakeholders. Here, in rare disease therapies, there is a wide consensus on the issues of access and their root causes, there are concrete solutions designed over the years through robust work embedded in real-life experience and multi-stakeholder dialogues, and now there are clear common positions amongst stakeholders. This Joint Statement represents the progress that would not happen without taking ourselves out of our comfort zone in such discussions. We hope it comes at the right time to help align the will of policy makers and stakeholders for positive change” says Yann Le Cam, EURORDIS’ Chief Executive Officer.