New Eurordis survey depicts the real life situation of European rare disease patients when it comes to accessing orphan drugs
The European rare disease patient alliance Eurordis has made available the results of a survey involving ten European countries that attempts to capture the price and access of orphan drugs at the national level. Working with the national rare disease alliances of Belgium, Denmark, France, Greece, Hungary, Italy, the Netherlands, Romania, Spain, and Sweden, the Eurordis study demonstrates the complexity of the process through which approved orphan products are brought to market across Europe. The principal actors in facilitating orphan drug availability are identified (national competent authorities, national insurance systems, the biopharmaceutical industry, and the patient organisations and national alliances) and the report distinguishes between orphan drug marketing authorisation – largely a European-level process - and orphan drug access – largely a national-level process.
Trying to chart which products are available in what countries and at what price is a difficult task and stakeholders from many different strands of the rare disease community lament the lack of transparency in this area. To begin with, one must define what is meant by “available”. A product can be on the market but not reimbursed by the national insurance scheme. Technically, such a product is available but if the patient cannot afford it, such a product is not accessible.
The survey reveals that while patient organisations can access information about the availability of the medicinal products used by their members, other data – particularly pricing information - remains inconclusive, as data on the official prices in individual countries as well as the actual prices paid by their healthcare system are not easily obtained. The information presented shows the diversity of product availability between countries, and identifies Denmark, France and the Netherlands amongst the countries enjoying wide access to orphan drugs, in contrast to Greece, Romania or Spain, which have a limited number of the 60 EU approved orphan drug products available.
The Eurordis inventory contributes the experience and knowledge of the patient alliances toward orphan drug access in their individual countries. What is needed now is full access to the hard pricing and reimbursement data and the national decision-making processes in order to better identify the bottlenecks that are preventing full and equal access to all the EU market-approved orphan drugs.
Consult the Eurordis inventory