New European Union Committee of Experts on Rare Diseases braves the “storm of the (new) century” to meet in Luxembourg
A massive snowstorm arrived in Western Europe just as the newly formed European Union Committee of Experts on Rare Diseases (EUCERD) was set to convene for the first time on 9-10 December in Luxembourg. Rare disease experts are a hardy bunch, however, and long accustomed to tackling hurdles and obstacles of all kinds. Thus the majority of members and many of the alternative members were able to make their way to the day and a half event. Seasoned stakeholders in the field of rare diseases, including representatives from DG Sanco and DG Research funded projects, the rare disease patient organisations and the biopharmaceutical industry, especially welcomed the new EUCERD member representatives coming from each of the Member States and look forward to a positive and eventful collaboration.
The meeting kicked off with a review of the European Commission Decisions that led to the establishment of the EUCERD. The appointment of the Members of the Bureau of the Committee was the next order of business. Ségolène Aymé (Orphanet) was elected President of the Committee for a two-year term. The Bureau will have three Vice-presidents: Yann Le Cam (Eurordis), Kate Bushby (Treat-NMD) and Helena Kaariainen (National Institute for Health and Welfare Helsinki). Amongst other responsibilities, the Bureau establishes the agenda for upcoming EUCERD meetings. Following the Bureau elections, the Internal Rules of the EUCERD proposed by the Commission were discussed, amended and approved.
Next, Antoni Montserrat (Policy Officer for Rare and Neurodevelopmental Diseases – DG Sanco) proposed a Road Map for the Implementation of the EU policy on Rare Diseases 2010-2013. After discussion of certain elements, it was decided that a revised Commission would be sent to the EUCERD members for comment before the next meeting (tentatively scheduled for March 2011).
The second day of the EUCERD meeting kicked off with a presentation by Ségolène Aymé of the activities of the Scientific Support of the EUCERD. These include the OrphaNews Europe newsletter, the EUCERD website, the activities of the Working Groups of the EUCERD, and the initiatives on classification and codification on rare diseases, including the proposal for an EU/WHO Workshop on Classification and Codification for Rare Diseases. A report on the Initiatives and Incentive on Rare Diseases (2009) was presented by the corresponding Working Group. Plans were discussed for the next report which will cover the 2010 activities in this field.
Next, the state of the play of the Europlan Project was presented. This included the national plans or strategies for rare diseases; guideline documents proposed for the national Europlan conferences; a report on the outcome of the Europlan national conferences in 2010; and the closing Europlan conference taking place in Rome, Italy during February 2011.
The creation of the Network of Experts on Newborn Screening for some Rare Disorders was elaborated by Luciano Vittozzi in view of an expected Council Recommendation on the topic in 2012. An update was given by Gratiela Dobirta of the Medical and Public Health Research Unit (DG Research) on the Seventh Framework Programme and the Directorate-General Research activities in the field on rare diseases, as well as a report of a Workshop organised by the National Institutes of Health (USA) and the European Commission. A second Workshop, scheduled for Spring 2011 in Washington, DC (USA), will seek to form an international consortium on rare diseases to prioritise and organise research. It is possible that Japan will join this initiative.
Results of the DG-Sanco 2010 Call for Proposals of the Second Health Programme were presented by Stefan Schreck (Executive Agency for Health and Consumers). An update of the proposal for a Directive on Cross-Border Healthcare was delineated by Nick Fahy (Head of Health Information – DG Sanco). This proposal is in the final stages of adoption. The European Reference Networks on Rare Diseases are explicitly mentioned in the Directive. It should be determined whether this Directive has gone through by the next EUCERD meeting in March 2011. The first EUCERD meeting concluded with an overview of the decisions taken during the day and a half event. These include sending a letter to the WHO defining expectations for the revision process of the International Classification of Diseases (ICD 11) in the field of rare diseases; and sending a letter to the Member States urging them to develop a plan for their rare disease patients by 2013 as called for in the Council Recommendation. Also, three EUCERD Working Groups will be refined and will hold Workshops in 2011. These will focus on Centres of Expertise and European Reference Networks for rare diseases; Registries; and Coding and Classification. Furthermore, the EUCERD website will be adapted to meet the needs of members expressed during the meeting. Finally, a proposal for a future Joint Action to support the Scientific Secretariat of the EUCERD will be drafted.
While there is obviously an enormous amount of work to be done to meet the needs of rare disease patients and their families in Europe, the first meeting of the EUCERD held out hope that much can, and will, be accomplished.