European market fragmentation and the lack of cooperation between the authorities responsible for setting prices and reimbursement are the main barriers to creating an efficient policy on rare diseases. This was the message sent out on Wednesday 22 February by the Eurordis organisation, during a conference on rare diseases.
A disease is considered “rare” if it affects fewer than 5 out of 10,000 people. Little is known about these diseases due to their rarity and their medical, psychological and social care is often very inconsistent. Overall, there are between 6-8000 diseases of this kind. The number of people affected in the European Union is estimated to be around 30 million, which corresponds to approximately 6% of the population.
With less than a week to go until International Rare Diseases Day on 28 February, Eurordis decided to highlight reflection on this topic by organising a two-day conference in Brussels. The title is informative and emphasises the importance of a cooperative process for developing “mutually acceptable solutions that respect all stakeholders” and improve common understanding and sustainable mutual confidence. The event opened in the evening at 21 February with Eurordis awarding prizes to individuals and organisations that help patients or support the cause for tackling rare diseases and generate funds for Eurordis programmes. One of the prize winners included Frédérique Ries MEP (ALDE, Belgium), who received the 2017 political decision maker prize, particularly because of the work he has undertaken in his “Rare 2030” pilot project, which obtained funding of €500,000 and a team of researchers that will work over two-year period for developing best strategies against rare diseases.
During his opening comments at the multipartite conference, Yann Le Cam, the director-general of Eurordis stated, “We need to demonstrate that medicines can be developed more quickly and cheaply for the regeneration of productivity in the pharmaceutical industry”. According to a study carried out by Eurordis, 24% of respondents have been unable to access the treatment they required because it was not available where they lived. Yann Le Cam identified several obstacles to accessing orphaned medicines: prices, productivity in the industry, market fragmentation and the allocation of resources. In a reflection document, the organisation, a federation of 738 associations of patients with rare diseases in 65 countries, indicated that orphan medicines continue to represent a limited fraction (5%) of pharmaceutical budgets in EU member states. (Original version in French by Sophie Petitjean)