Rare Daily Staff

Eurodis-Rare Diseases Europe and the European Federation of Pharmaceutical Industries and Associations issued a joint statement that outlines proposals to improve access to orphan medicines in Europe, where access can be inequitable across countries and patients can face long delays before getting needed medicines.

The Joint Statement on Patient Access to Medicines for Rare Diseases grows out of an 18-month dialogue between the two organizations. The groups said the statement also recognizes areas where the two organizations have different perspectives. It comes ahead of revisions of the EU General Pharmaceutical Legislation, including the Regulation on Orphan Medicinal Products, and the EU Regulation on Pediatric Use of Medicines, later this year.

“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. Here, in rare disease therapies, there is a wide agreement 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,” said Yann Le Cam, Chief Executive Officer of Eurodis-Rare Diseases Europe. “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.”

Eurodis said it believes that the three sets of proposals put forward—increased equity of access for patients and solidarity between Member states, improved HTA and Pricing and Reimbursement processes, and accelerated needs-led innovation—will bring about tangible change in access to medicines for people living with a rare disease.

Eurodis has promoted differential pricing as a route for better access and more equity across Europe since 2012. A proposal on equity-based tier pricing has been a hallmark of Eurodis’ Position “Breaking the Access Deadlock” issued in 2018. It said EFPIA jointly agreeing to the conceptual framework reflects progress and advances in finding solutions at the European level for rare disease therapies.

The proposal to reinforce European collaboration on post-launch real-world evidence came from the shared views on the obstacles caused by evidential uncertainties at the time of marketing authorization for value assessment, and its impact on a well-informed discussion on pricing and reimbursement, recognizing that the solution comes from a continuum of evidence generation.

In addition, EFPIA’s commitment to a “moonshot” to develop science for rare diseases, is welcome given how much there is still to learn about rare diseases, especially in underserved areas. Funding in public-private partnerships is a critical success factor in both research and healthcare, for clinical research before and after marketing authorization.

“EFPIA and Eurodis share the goal of faster, more equitable access to medicines for patients living with rare disease across Europe,” said EFPIA Director General Nathalie Moll. “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.”

Photo: Yann Le Cam, Chief Executive Officer of Eurodis-Rare Diseases Europe

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