Recommendations for Centres of Expertise adopted unanimously by the European Union Committee of Experts on Rare Diseases
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.
The final report Recommendations and Proposed Measures for a Belgian Plan for Rare Diseases was submitted to the Minister of Social Affairs and Public Health last month. The report is also available online on the website of the King Baudouin Foundation in English, French, German and Dutch languages. The proposed plan consists of 42 recommendations and measures that can be grouped into five central themes: Expertise and multidisciplinarity; Collaboration and networking; Knowledge, information and awareness; Equity in access; and Governance and sustainability. 
Off-label drug use has come under the spotlight in Europe following a scandal in France involving the widespread use of a product for an indication other than that which it had been authorised and which transpired in numerous deaths. However, off-label medicine use, which refers to the use of a medicinal product for an indication other than those for which it has received marketing authorisation, is critical in the field of rare diseases - an area that suffers from a serious lack of tested and authorised treatments. In the UK, the National Institute for Health and Clinical Excellence (NICE) will start commissioning expert assessments for off-label medicine use starting next spring. These assessments will not constitute formal guidance, but rather will provide “a summary of available evidence on selected unlicensed drugs to inform local decision-making”. A NICE press release on the topic observes that, “…Patients with rare diseases, such as certain cancers like lymphoma or autoimmune diseases, may have limited access to medicines as there are often not enough patients to run the clinical trials needed to develop a licensed drug. Some of these patients could benefit from treatment with unlicensed or off-label drugs”. According to the press release, the National Health Service (NHS) in England receives some 1000 specific requests for off-label use annually. The announcement for the off-label product assessments has been met with approval from the rare disease community. In the NICE press release, Andrew Wilson, Chief Executive of the Rarer Cancers Foundation, is quoted as saying: “This announcement is good news for patients with rare cancers and will help ensure all patients get access to good standards of care. No patient should be disadvantaged simply because they are unlucky enough to have a rare disease”. 
In Switzerland, the Federal Office of Public Health is working on a project that will facilitate the reimbursement of rare disease medicinal products. According to a news source, a round table meeting held in September gave health professionals, members of the biopharmaceutical industry, and local government representatives the opportunity to exchange views. Amongst the topics broached were strategies for reimbursing products and evaluating their benefits, as well as ways to improve diagnosis, for which the French model of identifying and creating networks of expertise was evoked. Finally, the issue of negotiating prices for rare disease treatments was discussed, as well as the necessity for clinicians and researchers to collaborate to enhance the understanding of rare disease treatments. A second round table will be held early next year. The project should then be open for consultation later in the year. The issue of reimbursement for orphan drugs has been catalyzed in Switzerland by the case of a patient with myopathy whose treatment was deemed cost-prohibitive. The particular treatment is presently being reimbursed, under certain conditions related specifically to efficacy. Reimbursement is limited to one year after which it must again undergo evaluation.
