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.

New EUCERD Recommendation on RD European Reference Networks

February 21, 2013 in News, Recommendation

On the 31 January 2013, during the seventh meeting of the European Union Committee of Experts on Rare Diseases, the EUCERD Recommendations on European Reference Networks for Rare Diseases (RD ERNs) were unanimously adopted by the 51-member EUCERD.

There are around 6000 rare diseases and most are unknown to healthcare professionals so rare diseases patients suffer from not knowing where to consult. To overcome this, some Member States have established centres specialised in some rare diseases/groups of rare diseases which have proven to be very efficient in improving quality of care. Now the European Commission is planning to link these centres together through European Reference Networks (ERNs) in order to gather expertise and improve healthcare for rare disease patients.

The development of centres of expertise and European Reference Networks in the field of rare diseases is encouraged in the Council Recommendation on an Action in the Field of Rare Diseases (2009/C 151/02) (8 June 2009) and in the Directive on the application of patients’ rights in cross-border healthcare (2011/24/EU) (9 March 2011) as a means of organising care for the thousands of heterogeneous rare conditions affecting scattered patient populations across Europe. European Reference Networks (ERNs) are one of the structures foreseen by the aforementioned Cross-Border Healthcare Directive to share knowledge, facilitate the mobility of expertise, and to allow Member States to provide highly specialised services of high quality for patients where this would have been impossible without European networking, such as in the case of rare diseases.

The EUCERD, formally the EC Rare Diseases Task Force, has already issued a series of reports investigating the state-of-the-art in the field, and a recommendation in 2011 on Quality Criteria for Centres of Expertise for Rare Diseases in Member States The 21 recommendations on RD ERNs build upon these previous achievements and will serve both to inform the Commission services and expert groups working on criteria for the creation and designation of ERNs in the context of the Cross-Border Healthcare Directive, as well as the Member States who are developing their healthcare pathways at both the national and EU levels in the field of rare diseases in the context of national plans/strategies for rare diseases which the Council has urged all Member States to elaborate by 2013.
The recommendations cover a range of points including the mission, vision and scope of ERNs, their governance, their composition, their funding and evaluation, as well as their designation.

The recommendations have been presented to the Cross-Border Healthcare Expert Group at the European Commission to inform their work on the designation criteria for ERNs on 6 February 2013.

Consult the recommendation.

New recommendation for a CAVOMP Information Flow

October 10, 2012 in News, Orphan medicinal products, Recommendation

EUCERD’s recommendation for improving the assessment of the Clinical Added Value of Orphan Medicinal Products encourages the creation of an Information Flow

Equitable and timely access to market-authorised orphan medicinal products is an ongoing issue for thousands of rare disease patients and their families across the European Union (EU). Large disparities in access exist between and even within the European Member State countries. Increasing cooperation between EU-level authorities and the Member States (MS) has been identified as a means of improving access to Orphan Medicinal Products (OMP) and reducing inequities. Global auditing firm Ernst & Young was mandated by the European Commission to identify and assess the possible options for the creation of a mechanism for the exchange of knowledge between the MS and European authorities on the scientific assessment of the relative effectiveness of orphan medicines and issued a reportin December 2011. The European Commission consequently asked the European Union Committee of Experts of Rare Diseases (EUCERD) to make recommendations in the area of facilitating the exchange of scientific information on orphan medicinal products that would support the clinical assessment processes undertaken by the MS.
No need to reinvent the wheel…
The EUCERD recommendation involves the creation of an Information Flow between individual MS and between MS and the EU that would bridge existing knowledge gaps, especially at the time of marketing authorisation. This flow would fit into existing regulatory, clinical, Health Technology Assessment (HTA), pricing and reimbursement processes. The recommendation thus capitalises on existing mechanisms, procedures, and regulatory frameworks, and does not bring new obstacles to the assessment process. The EUCERD Recommendation emphasises the continuum of evidence generation that takes place during the life cycle of an individual OMP and the Information Flow would be harboured by the corresponding institution responsible during each particular point in time (see the diagram).
The EUCERD Recommendation Improving Informed Decisions Based on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow will ultimately accelerate access to approved orphan medicinal products, while encouraging pricing and reimbursement decisions based on the value of the OMP and promoting good medical practices throughout the EU. The Recommendation has been submitted to the European Commission.

Recommendations for Centres of Expertise adopted unanimously

January 11, 2012 in News, Recommendation

On 24 October, during the third meeting of the European Union Committee of Experts on Rare Diseases (EUCERD), the Recommendations on Quality Criteria for Centres of Expertise for Rare Diseases in Member States were unanimously adopted by the 51-member EUCERD, which has representatives from all 27 EU Member States and from all domains relevant to the fields of rare diseases and orphan drugs, including academia, government, the biopharmaceutical industry and patient organisations. This is the first set of recommendations adopted by this committee. Developing Centres of Expertise and European Reference Networks in the field of rare diseases has been proposed in the Council Recommendation on an Action in the Field of Rare Diseases and more recently in the Cross-Border Healthcare Directive as a means of organising care for the thousands of heterogeneous rare conditions affecting scattered patient populations across Europe. In order to share knowledge and expertise more efficiently, the EUCERD recommendations seek to introduce harmonious standards of quality practices by elaborating criteria for the Member States to incorporate into their process to designate Centres of Expertise.

EUCERD, formally the Rare Diseases Task Force, has already issued a series of reports investigating the state-of-the-art in the field. The 45 Recommendations build upon this work already achieved and assist the Member States to develop their healthcare pathways at both the national and EU levels in the field of rare diseases. The recommendations cover the Mission and Scope of the Centres of Expertise; the Criteria for Designating Centres of Expertise; the Process of Designating and Evaluating National Centres of Expertise; and the European Dimension of Centres of Expertise.

The EUCERD Recommendations on Quality Criteria for Centres of Expertise for Rare Diseases in Member States are available on the EUCERD website.