Belgium and the Czech Republic advance their national rare disease strategies
On 9 June, 2009, the European Council adopted the Recommendation on an Action in the Field of Rare Diseases, calling on each European Union (EU) Member State (MS) to elaborate and adopt a strategy for their rare disease patients by the end of 2013. The Council Recommendation defines seven strategic areas for rare disease care, of which the first - Plans and strategies in the field of rare diseases – urges the MS to develop actions that encompass the elements of the following strategic areas: Adequate definition, codification and inventorying of rare diseases; Research; Centres of expertise and European reference networks; Gathering expertise on rare diseases at European level; Empowerment of patient organisations; and Sustainability.
In October, the Czech Republic released a ten-year strategy for its rare disease patients, estimated to be roughly 20,000 in the country of 10.5 million habitants. The Czech strategy, “intended to ensure the effective diagnosis and treatment of rare diseases, ensure that all patients with rare diseases have access to the indicated, high-quality health care, and ensure their subsequent social integration on the basis of equal treatment and solidarity”, is “fully compliant with the European Council's recommendation mainly concerning improved identification of rare diseases, support for the development of health policy and the development of European-level cooperation, coordination and regulation in this field”. The Strategy outlines existing efforts and proposes major targets and measures for improving the situation in the Czech Republic, which are to be subsequently specified in more detail in the context of a three-year National action plan that will establish “sub-tasks, instruments, responsibilities, dates and indicators for fulfilling individual tasks”. The first meeting of the working party for the preparation of the National action plan was scheduled to convene on 12 November in Prague. The Czech ten-year strategy, available on the European Commission’s Public Health Rare Diseases website, reveals the budgetary sources for the plan, which will include “existing budgetary chapters and domestic and foreign subsidies” such as the Ministry of Health and the country’s public health insurance. Consult the Czech Republic ten year rare disease strategy
The Czech Republic is not alone in its effort to develop a rare disease strategy. In February 2009, the Belgian House of Representatives adopted a resolution for a plan of action for rare diseases and orphan drugs. The Rare Diseases and Orphan Drugs Fund, managed by the Fondation Roi Baudouin, was mandated to coordinate the elaboration of a Belgian rare disease plan. Patient representatives, physicians and other specialists, paramedical staff, insurance organisms, social service representatives, members of industry, and political authorities participated in the working groups that developed a set of recommendations which groups specific measures into four central domains: diagnostics and treatment; codification and inventory; information, awareness, and patient empowerment; and access and cost. Under these four areas come a series of proposed measures that include the creation of centres of expertise/coordination; the establishment of a national platform; the development of a national registry; the creation of a national information portal; support for Orphanet Belgium; the modification of existing legislation to allow reimbursement of DNA samples tested abroad; a coordinated approach to non-DNA testing; establishment of a mechanism for the early access (pre-marketing authorisation) and reimbursement of orphan drugs (similar to the system currently used in France); establishment of an inventory of off-label product use; assurance that primary materials for treating rare diseases can be used legally; the modification of existing criteria used to confer an orphan designation when a suitable primary material is available and utilised; and support and monitoring for orphan drug use in the home setting. It is anticipated that the Belgian plan, to be unfurled in phases, would generate costs between 9 and 30 million Euros per year over a five-year period for the Belgian health insurance system. While Belgium continues to establish its new government, work on the second phase of the strategy is nonetheless progressing with further defined recommendations in the areas of non-medical costs of rare diseases, international networking, research, adherence, advanced therapy medicinal products (ATMP), ethical issues, teaching and education, therapeutic education, and clinical trials.
Consult the Belgian rare disease strategy recommendations (in French language).
Visit the European Commission Public Health Rare Diseases webpage for national strategies