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EFPIA-EURORDIS joint statement published on patient access to medicines for rare diseases

The statement contains a series of proposals to improve the lives of people living with rare diseases by addressing access barriers to transformative medicines


The EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases across Europe has been published.

The statement is based on an 18-month structured dialogue between the European Federation of Pharmaceutical Industries and Associations (EFPIA) and EURORDIS, a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

Commenting on the publication of the statement, EFPIA’s 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.”

The statement also recognises areas where the two organisations have different viewpoints. “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,” Moll said.

A conceptual framework for Equity-Based Tiered Pricing (EBTP) to better align medicines’ prices with countries’ ability to pay is proposed in the statement.

A number of proposals are also included, supported by both EFPIA and EURORDIS, that aim to improve pricing and reimbursement processes and Health Technology Assessment (HTA), at both EU and member state level.

The final proposal recognises the significant number of rare conditions where no treatment option exists and 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.

The report acknowledges that the recommendations it contains cannot “entirely alleviate the access challenge that rare disease patients face” but the organisations believe that these proposals “collectively represent an important step forward and a foundation for future collaboration”.

Yann Le Cam, chief executive officer or EURORDIS, said: “Often European and national policies cannot progress... because the analysis of the issues is unclear, the solutions are not identified, or there is no consensus among the 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… policy makers and stakeholders for positive change.”

Article by
Emily Kimber

15th June 2022

From: Regulatory, Healthcare



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