ECRD2022 : A week to make a better future for rare diseases possible
The European Conference on Rare Diseases & Orphan Products - ECRD 2022 organised by EURORDIS and co-organised by Orphanet took place online from 27 June to 1 July 2022. The event was designated as an official event of the 2022 French Presidency of the Council of the European Union and has gathered over 800 stakeholders from across the rare disease community, bringing this Presidency to a close and heralding the start of the Czech EU Presidency. The 11th edition was held under theme “Turning policy into action” and in the context of the Adoption of the first UN Resolution on People Living With a Rare Disease and their Families in December 2021. The conference aimed at exploring the political opportunities opened by this milestone and to discuss the future shapes of a European Policy on rare diseases, notably as a response to the Sustainable Development Goals.
The conference was opened by Avril Daly, Vice-President of EURORDIS-Rare Diseases Europe, who called for a new framework for policy in the European Union, based on the 2030 Foresight Project’s Recommendations. Stakeholders, including MEPs, channelled the need for a coordinated and comprehensive rare disease policy in Europe, and expressed the Parliament’s commitment to rare diseases. Frédérique Ries, Member of the European Parliament (MEP), stated that “The European Union needs a comprehensive policy framework to connect all policies and initiatives affecting people living with a rare disease at the European and national level.” Stella Kyriakides, European Commissioner for Health and Food Safety, also attended the conference and highlighted the Commission’s work in the field of rare diseases, including towards the Rare Disease Partnership, the ERNs and review of the Orphan Medicinal Products Regulation. WHO Regional Director for Europe, Hans Kluge, praised the role played by patient organisations in the adoption of the UN Resolution on PLWRD and stated that WHO will remain a strong partner to take the UN Resolution into an effective strategy for Europe. He asserted the importance of a European disability framework and a European digital health action plan, showing the commitment to Universal Health Coverage.
During the first day, the authors of the following four top scoring posters presented their poster live at the conference:
- “A long-term, prospective, multicenter, in clinic and online disease monitoring program (DMP) for patients with long-chain fatty acid oxidation disorders (LC-FAOD)”, Ultragenyx Pharmaceutical Inc.
- “Work participation in adults with rare diseases – a systematic scoping review of relevant research”, TRS National Resource Center for Rare Disorders
- “Using ADDIE learning model to design a multidisciplinary educational program centered on rare diseases addressed to high-school students in Romania”, Babeș- Bolyai University Cluj-Napoca
- “Operational Description of Rare Diseases – A common reference to improve the recognition and visibility of rare diseases”, Rare Diseases International
The other accepted posters were showcased on the event platform in a multimedia format.
The three following days were dedicated important discussions on how to reach the following three visionary goals for people living with a rare disease, inspired by the Rare 2030 Foresight Study and implemented through a new European policy framework for rare diseases.
- Ensuring healthy lives and promoting wellbeing for all people living with a rare disease at all ages
- Reducing inequalities for people living with a rare disease
- Building resilient infrastructure, promoting inclusive and sustainable industry and fostering innovation for people living with a rare disease
After four days of rich exchanges between stakeholders, the conference came to a conclusion with the moving speeches of young rare disease advocates, including Adéla Odrihocká, Young Patient Advocate from Czechia, who said “as rare disease patients and young citizens we have been told our whole life to wait. We don't have this time. We need hope for our future. We need to take action now, to ensure better management of symptoms and guarantee a better quality of life.” Danielle Drachmann, Executive Director of Ketotic Hypoglycemia International highlighted the need for stronger partnerships between experts and affected families for more efficient rare disease policies.
Yann Le Cam, Chief Executive Officer at EURORDIS-Rare Diseases Europe, closed the conference by stating that “The question is no longer if we need a new European Strategy, but how and when. We have witnessed the strong political momentum insisting that the current strategy is not enough, this is not the time to relinquish our efforts.”
While the conference highlighted France’s commitment to rare diseases, it also marked the beginning of the Czech presidency of the EU. Jakub Dvoracek, Deputy Minister of Health, stated that the Czech Republic will “Continue to support enhanced cooperation and coordination between the Members states and encourage the creation of the European Action Plan on rare diseases”. He also announced that a conference on rare diseases will take place in October 2022, to further discuss a coordinated strategy on rare diseases at the EU-level. The conference will address the keys aspects of the new framework, including, early diagnosis, access to treatment, the revision of paediatric legislations, and the role of the ERNs.
The next edition of the ECRD will take place in 2024. Recordings of the sessions are now available on the platform for registered attendees.
