To date, three groups of stakeholders in the field of rare diseases have been successively brought together by the European Commission to provide expert advice in this area to their services: the Rare Diseases Task Force (2004-2009), the European Union Committee of Experts on Rare Diseases (2010-2013) and the EC Expert Group on Rare Diseases (2014 onwards).
The Rare Diseases Task Force (2004-2009)
The Rare Diseases Task Force was established in January 2004 via Commission Decision 2004/192/EC of 25 February 2004 on the programme of Community action in the field of public health (2003 to 2008). The RDTF was replaced by the European Union Committee of Experts on Rare Diseases.
The members of the RDTF included current and former rare disease research project leaders, elected experts from Member States , and representatives from relevant international organisations (DG Research, DG Enterprise, EuroStat, EMA, WHO, OECD). Orphanet director Ségolène Aymé was the chair person.
Over 6 years, the RDTF played a pivotal role instigating key collaborative rare diseases initiatives in Europe. The RDTF met 12 times over the course of its mandate, and many key topics were brought forward for discussion in relation to rare disease research, policy and actions. The scientific activity of the RDTF was funded from 2005 onwards, which allowed the publication of OrphaNews Europe, the official newsletter of the Rare Diseases Task Force. Published every two weeks, this newsletter has served as a vital communication tool, bringing news on policy developments, medical and scientific findings, orphan drugs, research projects, patient activities, events and new relevant publications to the global rare disease community – including patients, healthcare professionals, researchers, industry professionals and health policy makers.
Various working groups were identified and constituted to meet the specific objectives of the RDTF: this included the Standards of Care working group, the Public Health Indicators working group, and the Coding, Classification and Data Confidentiality Group. These working groups produced various reports, recommendations and scoping papers.
One of the most notable contributions of the RDTF was its pivotal role between June and October 2007 in drafting the Communication Rare Diseases: Europe’s Challenges, in close collaboration with the European Commission. The process ultimately culminated in the adoption of the European Council Recommendation on an Action in the Field of Rare Diseases in June 2009.
The European Union Committee of Experts on Rare Diseases replaced the European Commission’s Rare Diseases Task Force (RDTF) in 2010.
The European Union Committee of Experts on Rare Diseases (2010-2013)
The European Union Committee of Experts on Rare Diseases was formally established via the European Commission Decision of 30 November 2009 (2009/872/EC) . This committee was charged with aiding the European Commission with the preparation and implementation of Community activities in the field of rare diseases, in cooperation and consultation with the specialised bodies in Member States, the relevant European authorities in the fields of research and public health action and other relevant stakeholders acting in the field.
The 51 member committee with representatives from all Member States and stakeholder groups was chaired by Ségolène Aymé and co-chaired by Kate Bushby, Yann Le Cam and Helena Kääriäinen. The EUCERD met 3 times a year and organised a number of workshops supported by the EUCERD Joint Action.
Over the 3 year mandate of the EUCERD, 5 recommendations were adopted in the field of rare diseases concentrating on centres of expertise, European Reference Networks, the clinical added value of orphan medicinal products information flow, registries & data collection, and indicators for national plans. An Opinion on potential areas of collaboration at European level in the field of new born screening was also issued.
The EUCERD was a highly successful forum for exchange of experience and cooperation between stakeholders appreciated greatly by the rare disease community. The EUCERD was replaced at the end of its mandate by the European Commission Expert Group on Rare Diseases from 2014 onwards.
The European Commission Expert Group on Rare Diseases (2014 onwards)
This group includes representatives from all the Member States plus nominated experts, representatives of patient organisations, patients’ organisations in the field of rare diseases, European associations of producers of products or service providers relevant for patients affected by rare diseases, and European professional associations or scientific societies acting in the field of rare diseases.