8th EUCERD meeting: New recommendations adopted

June 19, 2013 in EUCERD Meeting, Recommendation

The European Union Committee of Experts on Rare Diseases (EUCERD) held its 8th meeting on 5-6 June 2013, marking the end of its three-year mandate. This last meeting was proof of the effectiveness of the Committee in successfully bringing together representatives of the European Commission, Member States, patient organisations, Industry and experts in the field of public health and research, as it saw the adoption of two sets of recommendations, on patient registration and data collection and indicators for national plans and strategies respectively.

During the meeting of the EUCERD a number of key topics were presented and discussed, including steps towards the establishment of a European platform for rare diseases registries, the role and content of a possible European Research Infrastructure Consortium on rare diseases information for research, the EUCERD opinion on newborn screening, and next steps towards the development of a EUCERD recommendation on genetic testing for rare diseases. Members were also informed of the outcomes of the working group on mechanisms of coordinated access to orphan medicinal products, the advancement of the elaboration and implementation of national plans/strategies for rare diseases due by the end of the year, the work of the EUCERD Joint Action in 2013, as well as plans for the European Conference on Rare Diseases and Orphan Products to be held on 8-10 May 2014 in Berlin.

The meeting also saw the unanimous adoption of the EUCERD’s Core Recommendations on Rare Disease Patient Registration and Data Collection. Rare disease registries are valuable instruments for increasing knowledge on rare diseases, and for supporting fundamental, clinical and epidemiological research, as well as for post-marketing surveillance of orphan medicinal products and medicines used off-label. This data is also crucial for the planning of healthcare services. The recommendation calls for the international operability of registries and databases and use of appropriate coding systems to enable the necessary pooling of data for public health and research purposes, gives advice concerning the establishment of registries and collection of data, highlights the various uses of patient data and how to best share this information, underlines the importance of adherence to good practice guidelines in the field, stresses the need for registries to be adaptable to meet future needs, and emphasises the importance of sustainability for the timespan of the registry’s utility.

The second set of recommendation to be adopted was the EUCERD’s Recommendation on Core Indicators for National Plans/Strategies for Rare Diseases. This recommendation provides a list of 21 indicators which are intended to capture relevant data and information on the process of planning and implementing of these plans and strategies on a regular basis. These indicators would provide information notably to the European Commission on the implementation of the Council Recommendation on an Action in the field of Rare Diseases (June 2009) which encourages Member States to establish a national plan or strategy in the field by the end of 2013. They will also serve as a basis for the elaboration of indicators at national level tailored to the specific actions foreseen in the plans/strategies. This set of recommendations will be revised in the future to take into account the experiences of the Member States.

To mark the end of this mandate, the Director of Public Health at the EC’s Directorate-General for Health and Consumers, Mr. John Ryan, joined the meeting to thank the Members of the Committee, and the Chair, Dr. Ségolène Aymé, for their work. He highlighted the important advisory role the EUCERD had played during their term and gave assurance that the next committee, to be established within the coming months, will continue to play a key supporting role in European rare disease policy in the future.

Estabished in 2009, the EUCERD held its first meeting in December 2010, and has succeeded in fulfilling its mission of assisting the European Commission in the field of rare diseases by elaborating and adopting five sets of recommendations on a variety of topics : in addition to the aforementioned recommendations adopted during the 8th Meeting, the EUCERD has issued recommendations on Quality Criteria for Centres of Expertise for Rare Diseases in Member States, Improving Informed Decisions Based on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow, and European Reference Networks for Rare Diseases. In addition, the Committee has helped elaborate, in collaboration with national stakeholders, the annual Report on the State of the Art of Rare Disease Activities in Europe which provides an indispensable overview of activities at European and Member State level: the new edition will be published this summer. Members have also contributed to the discussions and conclusions of a number of expert workshops organised by the EUCERD Scientific Secretariat, a number of which within the framework of the EUCERD Joint Action.

All the recommendations elaborated by the EUCERD during the past three years are also available here. The summary report of the 8th Meeting of the EUCERD is available here.