Rare diseases: a public health priority

Read "Rare Diseases: A Public Health Priority" >

“To promote rare diseases as a public health priority” is one of Eurordis' most important stated objectives. With this in mind, Eurordis has come to the conclusion that a further step to take is to actually clarify what rare diseases are as a public health concept. In its work with national and EU health authorities, Eurordis regularly stumbles across the fact that policy- and decision-makers know very little about rare diseases. “We should never take for granted that people know what rare diseases are,” says Yann le Cam, CEO of Eurordis. And when people are aware of them, they often don't know the specificities or the impact these diseases have on the lives of patients and their families. An example of confusion is the concept of rarity: rare diseases are rare (defined as less than one citizen in 2,000 in Europe), but rare disease patients are many. For Flaminia Macchia, in charge of European Public Affairs at Eurordis, it is crucial to realise that “it can happen to anybody, at any stage of life. Indeed, it is not unusual to have a rare disease.” Another frequent confusion is made between rare diseases and neglected diseases (common, communicable diseases mainly affecting patients living in poor, developing countries). “The consequences of this confusion are manifest in the World Health Organisation (WHO) Report on Priority Medicines,” recalls Flaminia Macchia, “even if it was very clear in the background paper from the Dutch EU Presidency”.

To clarify the concept of rare diseases as a public health issue, Eurordis took the initiative to create a unique document covering all aspects of rare diseases: definition of the concepts involved; characteristics and specificities of rare diseases; figures; description of the social, medical and financial impact of rare diseases on the lives of patients and their families; need for raising public awareness; need for appropriate funding and for appropriate health systems and professional care; and, last but not least, the absolute necessity to empower patients to emerge from their isolation. No such a document existed before, and Eurordis decided to take the lead. The paper was written by Eurordis based on its experience, with additional input from the European Conference on Rare Diseases in Luxembourg in June 2005. Partners of Eurordis as well as members and interested parties were directly consulted in the process and had many opportunities to contribute to the document and give their feedback. An exemplary process of wide, public consultation was then put in place before the final document was adopted in December 2005.

But a document is only a tool, and to achieve its aims, it has to be used extensively. Eurordis will now disseminate this founding document widely to everyone playing a role, closely or not, in the rare disease community: Eurordis' members; rare disease patients; the public at large; EU policy- and decision-makers; health professionals; and journalists. Eurordis encourages everyone to use the document, especially at the national level. Print it! Forward it! Disseminate it! Your comments and feedback are always welcome. The rare disease community will be grateful to you for helping it put forward rare diseases as a much needed public health priority in the EU!

For more information:
Read the document "Rare Diseases: Understanding this Public Health Priority" >

This article appeared in the January 06 issue of our newsletter.

Author: Jerome Parisse-Brassens
Photo credits: © Eurordis