Skip to content


Com nossos programas e iniciativas

Generic filters
Exact matches only
Search in title
Search in content
Search in excerpt

Rare Disease patient advocates call for a bold, joined-up approach in the evolution of the Orphan Medicinal Products Regulation

Fevereiro 2023

Speaking at a conference hosted on Wednesday 8 February at the European Parliament, policymakers from across the EU institutions discussed the need for an evolution of the regulatory framework that further drives innovation and improves access to transformative treatments for people living with a rare disease.

Only six percent of all the known rare diseases have an approved treatment option, and 22% of people with a rare disease cannot get the treatments they need because they are not available where they live.

The conference, ‘For a patient-driven evolution of the Orphan Medicinal Product Regulation: Addressing unmet needs’, hosted by Frédérique Ries MEP and EURORDIS-Rare Diseases Europe, shone a spotlight on the expectations of the rare disease community from the Revision of the Orphan Medicinal Product (OMP) Regulation, which will amend the legislation first introduced by the EU in 2000 aimed at incentivising investments in the development of medicines for rare conditions. 

The Revision of the OMP Regulation will provide a pivotal opportunity to hone and enhance the system for spurring greater medical innovation and boosting the development of new medicines. Advocates with real life experience of the current OMP Regulation reported feeling cautiously optimistic about the prospects for positive change.

I’ve seen some orphan drugs disappear from pipelines. Altogether, there have been thousands of orphan drug designations in Europe, but only around 260 have been authorised! I’d like to see more orphan medicines cross the finish line to get approved, and to be made more affordable for healthcare systems and more available all over Europe. For me, it is key that the coming Revision of the OMP Regulation embraces patient partnership during the whole cycle of orphan drug development to make sure that the patients’ unmet medical needs are addressed.

Dominique Sturz, Rare Disease Week participant and advocate from the Austrian Rare Disease Alliance and Usher Austria

In the presence of representatives from the European Commission, the European Parliament, the European Medicines Agency (EMA), and the upcoming Spanish Presidency of the EU Council, EURORDIS took the opportunity to restate its proposed recommendations for the highly anticipated Revision of the Orphan Medicinal Products (OMP) Regulation. 

Yann Le Cam, Chief Executive of EURORDIS, reflected on the recently leaked draft Revision and commented that while the draft has “a lot of positive points” and “goes in the right direction”, there are also “many uncertainties and shortcomings”. Mr Le Cam specifically spoke of his concerns within parts of the draft, noting that some of the draft’s wording contains excess room for interpretation, potentially undermining the robustness of legislation aimed at delivering meaningful improvements in rare disease medicine development.

The opportunity that the coming Revision offers was recognised across political groups in the European Parliament. Conference host Frédérique Ries of the Alliance of Liberals and Democrats for Europe (ALDE), Deirdre Clune MEP of the European People’s Party (EPP) and Tilly Metz MEP of the European Green Party (EGP) were united in their aspiration for Europe to become a global leader in medicine development and access, and for more investment to be attracted to the development of therapies for life-threatening or debilitating diseases.

We have a duty to prove that health and caring for the most vulnerable is not an extra. It is in our DNA. At the very core of our EU values is solidarity. We have the responsibility to deliver.

Frédérique Ries MEP, Co-Chair of the network of Parliamentary Advocates for Rare Diseases, speaking at the conference.

The existing ecosystem has been a success, and we want to preserve what’s good in it and build on it.

Deirdre Clune MEP, speaking at the conference.

The conference formed part of the Brussels Rare Disease Week programme – a four-day series led by EURORDIS-Rare Diseases Europe aimed at empowering rare disease patient advocates to effect change at the EU-level.

Julien Poulain, Communications Manager
Communications Manager
EURORDIS-Rare Diseases Europe
+33 6 42 98 14 32