Eurordis - Rare Disease Europe

• EURORDIS celebrated its 25th anniversary.
• We welcomed 48 new members in 2022, reaching a total of 1,000 members by March 2022, which coincided with the anniversary.
• A strategic review initiated in 2021 was completed in 2022, resulting in the adoption of a new vision, mission, values, strategy, strategic objectives, and an organizational model by the General Assembly.
• Terkel Andersen stepped down as EURORDIS President due to health reasons, and Avril Daly assumed the role of Acting President until a new Board of Officers was elected in November 2022.
• Momentum for a European Action Plan for Rare Diseases was built through the Rare 2030 foresight study, with support expressed at the European Conference on Rare Diseases and by over 20 Member States and 50 Members of the European Parliament.
• In response to the invasion of Ukraine, EURORDIS launched a Ukraine Response programme, providing support to Ukrainians with rare diseases, including housing assistance in partnership with Airbnb.org.
• The Rare Barometer launched a survey on the journey to rare disease diagnosis in March 2022, reaching 13,307 respondents, with results to be published in 2023.

• The Rare2030 Foresight study, co-funded by the EU and led by an academic consortium with EURORDIS, concluded in March 2021, culminating two years of extensive work and resulting in policy recommendations for the next decade.
• EURORDIS initiated the #30millionreasons campaign to press for the creation of a European Action Plan for Rare Diseases. The advocacy campaign led to substantial political engagement, with the European Parliament recognising the need for such a plan and including rare diseases in the EU Trio Presidency agenda of France, the Czech Republic, and Sweden.
• The inaugural Rare Diseases Week in 2021, organised by EURORDIS, enabled 38 participants from 20 countries to engage online with European Parliamentarians, facilitating knowledge exchange and training in European policy advocacy.
• EURORDIS played a pivotal role in the United Nations General Assembly’s adoption of the first-ever resolution on addressing the needs of people with rare diseases in December 2021, marking a landmark achievement in acknowledging the challenges faced by this community globally.

The first-ever fully virtual European Conference on Rare Diseases & Orphan Products (ECRD) takes place, bringing together a record 1,500 registered participants.

• Rare Barometer reaches 10k people living with a rare disease, their family members and carers who participate in EURORDIS surveys.
• In February, the first Rare Disease Day Policy Event at the United Nations (the Second High-Level Event of the NGO Committee for Rare Diseases) is held at the UN headquarters in New York.
• The new Rare 2030 foresight study on the future of rare disease policy starts, led by EURORDIS.
• Following joint advocacy efforts by EURORDIS and Rare Diseases International UN Member States include rare diseases in Political Declaration on universal health coverage, adopted in September 2019. A victory as it was the first time rare diseases were included in a UN political declaration adopted by all UN Member States.
• The first face-to-face trainings of the EURORDIS Digital School on Social & Digital Media and the EURORDIS Leadership School on Healthcare & Research take place in Gothenburg, Sweden and Barcelona, Spain, respectively.
• The icon Audrey Hepburn becomes posthumous of EURORDIS.

• EURORDIS launched the EURORDIS Open Academy consolidating all of EURORDIS’ training activities and the first edition of the EURORDIS Winter School on Scientific Innovation and Translational Research takes place in Paris.
• The launch of the new EURORDIS EUROCAB Programme to facilitate the creation of community advisory boards.
• EURORDIS and resource centres for rare diseases gather in the new European Network of Resource Centres for Rare Diseases – RareResourceNet.
• EURORDIS is granted Special Consultative Status with the United Nations Economic and Social Council (ECOSOC), paving the way for our efforts to advocate for rare diseases to become an international health priority.

• EURORDIS celebrates its 20^th birthday and the 10^th edition of Rare Disease Day.
• After years of advocacy efforts by EURORDIS and the rare disease community, the European Commission launched the first European Reference Networks.
• EURORDIS launched a new network of Parliamentary Advocates for Rare Diseases.

• EURORDIS and the Ågrenska Foundation launch the NGO Committee for Rare Diseases, a substantive committee established under the umbrella of the Conference of NGOs in Consultative Relationship with the United Nations. The NGO Committee was inaugurated in November 2016 at the ‘Global Gathering for Rare Diseases’ event at the headquarters of the UN (New York), the first event focused on rare diseases to be held at the UN.
• In anticipation of the launch of the European Reference Networks (2017), EURORDIS creates European Patient Advocacy Groups (ePAGs) to structure representation of the patient voice in the development and governance of the future Networks.

• EURORDIS launches its new Rare Barometer survey programme.
• Rare Diseases International, the global alliance of people living with a rare disease, is created by EURORDIS.

• EURORDIS welcomes its 500^th member!
• The first EURORDIS Black Pearl Dinner takes place in Brussels, including the first EURORDIS Black Pearl Awards ceremony.

• Creation of IRDiRC, the International Rare Diseases Research Consortium. EURORDIS played an instrumental role in its creation and in representing the patient voice in the Consortium.

• EURORDIS supports national alliances around Europe to organise the first EUROPLAN conferences.
• Creation of the first EU Committee of Experts on Rare Diseases (EUCERD), including eight patient representatives as members, several of which representing EURORDIS.

• Launch of the EURORDIS Council of European Federations (CEF).
• EURORDIS forms its first international partnership by signing a Memorandum of Understanding with the US National Organization for Rare Disorders (NORD) for a 5-year strategic partnership.
• EURORDIS and other partners in the RAPSODY Project create the European Network of Rare Disease Help Lines.
• EURORDIS launches RareConnect, the online platform where rare disease patients, families and patient organisations develop online communities and conversations across continents and languages.
• Publication of the book The Voice of 12,000 Patients, a report based on the EurordisCare2 and EurordisCare3 surveys

• The first Rare Disease Day campaign takes place in Europe, created by EURORDIS and national alliances.
• The first EURORDIS Summer School on Medicines Research & Development takes place in Barcelona.

• Launch of the EURORDIS Council of National Alliances (CNA).

• Creation of the EURORDIS Round Table of Companies to foster dialogue with, and between, companies addressing rare diseases.

• The first EURORDIS Membership Meeting takes place in Barcelona.

• Organised by EURORDIS, the first European Conference on Rare Diseases & Orphan Products takes place in Copenhagen.

• Yann Le Cam becomes Chief Executive Officer of EURORDIS.
• Patient representatives including EURORDIS Chief Executive Officer Yann Le Cam are appointed for the first time as full members in a European Medicines Agency committee – the Committee for Orphan Medicinal Products.

Following advocacy efforts by EURORDIS and the wider rare disease community to bring into place legislation in Europe that mirrored the success of the Orphan Drug Act in the US (1983), the EU Regulation on orphan medicinal products is adopted.  

EURORDIS went on to play an instrumental role in advocacy efforts that led to the adoption of numerous EU legislations that have a positive impact on people living with a rare disease in Europe:

• EU Regulation on medicinal products for paediatric use (2006)
• EU Regulation on advanced therapy medicinal products (2007)
• EU Commission Communication Rare Diseases: Europe’s Challenges (2008)
• EU Council Recommendation on a European action in the field of rare diseases (2009)
• EU Directive on Patients’ Right to Cross-Border Healthcare (2011).

EURORDIS-Rare Diseases Europe was created in 1997 by four founding members: the Association Française contre les Myopathies, AIDES Fédération Nationale, Vaincre La Mucoviscidose and La Ligue Nationale Contre le Cancer. Our original goal was to advocate for EU laws to bring medicines to people living with a rare disease.