Following Brussels Rare Disease Week, we interviewed Yann Le Cam (EURORDIS-Rare Diseases Europe) and Julia Jenkins (EveryLife Foundation for Rare Diseases). We learned why engaging in advocacy events is so important and how these organisations are working to improve the lives of patients across borders

Interview by Geoff Case, RARE Revolution Magazine

In early 2023, EURORDIS-Rare Diseases Europe and US-based EveryLife Foundation for Rare Diseases announced a partnership to launch the second annual Brussels Rare Disease Week. Inspired by EveryLife Foundation’s Rare Disease Week on CapitolHill, which is now in its 12th year, EURORDIS hosted Brussels Rare Disease Week from 6–9 February 2023 with the goal to empower rare disease patient advocates to participate effectively in advocacy activities at the European and national levels.

Thanks to financial and consultative support from the EveryLife Foundation, EURORDIS brought together 44 patient advocates from 21 countries to share the challenges of their rare disease journey with EU decision-makers.

Can you tell me about the Brussels Rare Disease Week event itself?

Yann: Forty-four advocates representing large patient groups or national alliances of rare diseases from 21 countries came to Brussels to gain the knowledge and skills to effectively influence European Union (EU) policymaking throughout their future careers. The programme involved engagement with the three main EU Institutions—the EU Council, the Parliament, and the European Commission. They enjoyed meetings with Members of the European Parliament (MEPs) from their country, a meeting with the health attaché for the Spanish government, and meetings with several representatives of the European Commission. Frederique Ries and EURORDIS also co-hosted a conference on the upcoming Revision of the Orphan Medicinal Products Regulation, an important legislative file that the participants were talking to policymakers about.

Julia: The event was not just an event to lobby Parliament, it was about patient empowerment, democracy, giving patients a voice in public policy, and connecting patients together. Most importantly it was a catalyst for more advocacy.

What were the highlights for you?

Yann: Seeing so many patient advocates leave with a better understanding of the role that they can have in influencing EU policymakers. One of the participants said that we had “made the EU more tangible” for them; it’s great to know that people learned and got to grips with it. It was also amazing to see participants share their experience and expertise with one another to strengthen the European patient advocacy network. I’m also delighted that so many MEPs agreed to meetings, and were so engaged in their meetings.

Julia: Meeting the patients and learning their personal stories and about their organisations. I loved learning about the efforts of Spain’s FEDER (the Spanish Federation of Rare Diseases) and their efforts to reach out to Latin America with resources since they share the same language. I was inspired by a Wilson disease patient from France who wanted to become a professional patient advocate and help mobilise her disease community in Latin America. There were so many sparks lit during the event. It was so special to see patients realising their power as legislative and policy advocates and wanting to go back home and start advocating at the national level.

How does your partnership help address the global rare disease public health crisis?

Yann: We have long known that there is an added value of working at the EU level, so it was great to have patient advocates understanding legislation to better articulate their national and European advocacy. We had several requests that we took to prominent MEPs, including the call for a European Action Plan on Rare Diseases, which we believe is a necessary step to improve the lives of people living with a rare disease in Europe. The last comprehensive strategy on rare diseases—which sets the direction for Member State and European actions in this area—was 14 years ago. Over the week we saw momentum build for this initiative that would aim to reduce inequalities across Europe.

Julia: When it comes to developing treatments for rare disease, especially the ultra-rare or very rare diseases, companies have a global approach because they will need to find and treat most of the patients with the disease in the world for the treatment to be economically viable, yet patient organisations often due to limited resources stay very siloed in their home countries. Our partnership with EURORDIS is an important first step in understanding our organisations and identifying areas of strengths to allow us to learn from each other and better support the rare disease community. 

What are the next steps?

Yann: Rare Disease Day is coming up (28 February 2023) so we are hoping to engage many of the MEPs we met during Rare Disease Week to continue our call for a European Action Plan on Rare Diseases. We are also excited to bring some new MEPs into our Network of Parliamentary Advocates on Rare Diseases, who will have an important role in influencing upcoming legislation on the European Health Data Space and the future General Pharmaceutical Legislation.

Julia: Rare Disease Week on Capitol Hill is just a few days away (28 February–2 March). We are excited to be back in person with more than 500 advocates from all 50 states joining us in Washington, D.C. We are hosting a state-level advocacy event again this year and supporting other organisations hosting local advocacy events. In the US, rare disease patients are impacted by policies made on both the local and national level. Also, we will be reaching out to other organisations outside of the US to develop partnerships.

“Rare disease knows no borders. We must work together to address this global public health crisis.” – Julia

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