EURORDIS surveys patient organisations

The results of a survey conducted by the European Organisation for Rare Diseases (EURORDIS), entitled European Rare Disease Patient Groups in Research: current role and priorities for the future, have been announced at the Rare Diseases Day Workshop, held in Brussels this week, following the third annual Rare Disease Day event on February 28.  

The survey aimed to discover how, and to what extent, patient organisations (POs) support research and, from the patient's perspective, which are the priority research areas. Key findings are that POs are very interested in, and are committed to, research, despite great differences in terms of numbers of members and budgets. They are keen to collaborate with researchers, not only by orchestrating meetings between clinicians, researchers and patients, but also by giving logistical and financial support. POs in Europe allocated more than €13m to research last year, excluding funding from the Association Française contre les Myopathies (AFM), which gives about €60m to research annually. 

The survey concludes that rare disease POs are playing an important role as catalysers of research on their respective diseases. Their members' intimate knowledge of their diseases and lay expertise puts them in a strategic position at the crossroads of all fields in research (from basic to therapeutic); enables them to highlight the setbacks in basic research and clinical applications, and also to flag up weaknesses of translational research.   

POs want to see basic and clinical research conducted in parallel and through an intense dialogue between scientists, clinicians and patients. They are calling for partnering of all stakeholders. 

The findings show that, despite the enthusiasm, of their members, POs do not have the means nor the ambition to control and support all research by themselves. Their role is not to replace public institutions, but they can be valuable partners in research through identifying the gaps and supporting early research in areas not covered by the public or private sectors. Further, they can provide seed money or non-financial support to trigger interest in an 'orphan' field of research.  

The results help to acknowledge the role of patient organisations in research, and will contribute to shaping the future research agenda of the European Union. They will also help national patient alliances advocate for more and better research for rare diseases at national level. 

The survey was conducted between October and November 2009 in collaboration with the Centre de Sociologie de l'Innovation, École des Mines, France. Of the 772 rare disease POs invited to complete the online questionnaire, there were 309 valid responses, representing 110 rare diseases and 1.3m patients in 29 European countries.