European rare disease organisations - alike and yet so different

"Who are you?" That is the question Eurordis asked of 1402 organisations throughout Europe between September and December 2003. Organisations were surveyed on the availability and circulation of information on rare diseases.

But first the organisation was asked to give details about itself: when it was established, its mission, who runs it and how it is financed. This is vital information in support of the proposal to the European Union of a common public health policy across Europe, which takes into consideration individual countries' specificities. Many of you took part in the project. Here is a profile of you drawn by Lesley Greene, the volunteer leader of the project, as a token of thanks for your participation.

With 20 years of experience in the field of rare diseases, the survey results do not surprise Lesley Greene. Rare disease organisations across Europe resemble one another and encounter the same obstacles: lack of visibility and shortage of funds. “Eurordis carried out a survey of the organisations distributing information on rare diseases (PARD 3), as part of a project funded by the European Commission and the Association Française contre les myopathies (French Muscular Dystrophy Association)”, explains Lesley Greene, the project leader. She is the mother of a young woman living with cystinosis and works at CLIMB, a national metabolic diseases information and advice centre in Great Britain. “The aim of the survey was to build the case for a common public health policy across Europe which takes into consideration individual countries' specificities. We had hypotheses, now we have objective arguments to back up our proposals”.

The survey was sent to 1402 organisations at the end of 2003, largely through channels provided by the national rare disease alliances. The study covered 18 countries and profiled 372 participating organisations. Eighty-eight per cent of the respondents were patient organisations. Information services, research bodies, government organisations and specialist health services made up the remainder. A standard profile emerged, regardless of location. An organisation generally targets a single rare disease rather than a collection of diseases, is principally run by volunteers, is largely financed by its members - receiving occasional private donations - and suffers from severe lack of funding, particularly as regards the state and the European Commission. Its main activity is patient-orientated, providing information in 97% of the cases and services in 76%. Advocacy and research are undertaken by fewer than one in two of the organisations.

Despite these similarities, four major regions do emerge with differing characteristics. In Northern Europe, organisations have a long and extensive history, undertake advocacy and receive state funding. This is possibly due to the prominence of volunteer work and the care culture in these societies. Legacies and private donations are more important here than elsewhere. In Western Europe, the impact of fund-raising is considerable, perhaps as a result of the Telethon. In Central Europe, organisations are more newly established. They often provide volunteer patient services. In Southern Europe, the baby of the family of Europe, organisations employ salaried staff and successfully campaign for local and regional recognition.

Lesley Greene draws the conclusion that “the funding gap clearly shows how little consideration is given to rare diseases. At first sight, rare diseases do not seem to constitute a public health issue and people are more concerned with high profile causes such as cancer. To overcome this attitude, we must increase our co-operation and networking to alter the scale of the problem. We can show that rare diseases, when taken as a whole, have a significant impact socially, medically and scientifically. Not to mention the fact that rare disease research often produces benefits in other areas of health.

European organisations have a breadth of expertise, and have grouped together and enhanced their image through Eurordis. They look set to make themselves heard - at every level. Even more so as they are on the increase: half of all the rare disease organisations surveyed started up in the past ten years and the most recent are among the most active.

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This article appeared in the March 05 issue of our newsletter.

Author: Anne-Laure Murier
Translator: Helen Lea
Graphics: © Eurordis