The Eleventh European Health Forum Gastein considers the challenge of managing rare diseases
The eleventh European Health Forum took place in Gastein, Austria, from 1-4 October with sessions devoted especially to rare diseases. The first forum, Meeting the challenge: an opportunity for demonstrating European added value took the form of a panel discussion on several related themes: Integrating European initiatives with national activities reviewed EU-level initiatives for rare diseases, considered Romania as an example of the pertinence of national rare disease plans, and looked at the initiatives within the scope of the French presidency of the European Council. France is the only country in the world that has adopted a rare disease plan with a specific budget. France’s plan was adopted in 2004 with a budget of 100 million euros for a four-year period. Ten strategic objectives were delineated to meet the overriding aim of ensuring equitable access to rare disease diagnostics, care, and treatment: increasing knowledge; recognising rare diseases within the long-term disease framework of care; providing accurate information for patients and professionals; improving training for professionals; implementing a coherent screening policy; ameliorating access to treatment via centres of reference and via the availability of reimbursed orphan drugs; developing new products; meeting the accompanying needs of RD patients by linking various institutions and supporting patient groups; improving RD research by establishing a voluntary research policy; and coordinating partners at both the national and EU levels. France’s plan has been considered a model by several other countries and with France currently assuming the presidency of the European Union and thus extending its patronage to rare disease initiatives at the EU level, this is an ideal time to spotlight rare disease policies and patient needs across Europe. Several events are being held under the patronage of the French EU presidency.
The second forum, The need for patient centred efforts: centres of reference – equal access addressed the difficult issue of how to foster equitable access to rare disease care. Not every country (particularly smaller ones and those with limited resources) can have expertise for every rare disease. The role of European networks of centres of expertise is particularly relevant to rare diseases, as is the role of patient involvement. Improving professional and patient access to information and quality management is being developed by pilot projects such as ECORN-CF. Epidermolysis bullosa was presented as an example of the critical role networks of centres of expertise play in the field of rare diseases. Expert networking combined with skilled fundraising for this devastating rare genetic disorder resulted in a successful gene therapy trial. Recent developments, such as cross-border health care, need to be scrutinised to be rendered most effective for all players. Rare diseases, perhaps more than any other domain, need a collaborative effort between the 27 member states. The EU action in the field of rare diseases is intended to bring together the different elements in order to create an efficient strategy. The adoption of a European Commission Communication and proposal for a Council Recommendation (taking place in November 2008) will then require implementation, including the development of EU guidelines on many different aspects, the classification of diseases, an analysis of orphan drug bottlenecks, and many other actions.
The next subject to be tackled asked How to enforce the development of orphan medical products and how to fund them? Considering the various aspects of value, rarity, risk and pricing, a model developed by the EU High Level Pharmaceutical Forum’s Pricing and Reimbursement Working Group is sustainable because it is ultimately in the best interest of all society. Raising both public and policy maker awareness of the impact rare diseases have on patients and their entourage is critical in order to sustain the principle of solidarity that guides public opinion vis-a-vis orphan medicines.
Stakeholders are considering this year’s European Health Forum a success for that very reason: it enabled those who champion rare disease and orphan drug research, policies and funding to present the concerns and issues facing rare disease patients and professionals to key European health decision-makers.
Consult the European Health Forum rare disease presentations
