EURORDIS Survey on “European Rare Disease Patient Groups in Research: current role and priorities for the future”

researchersConducted by EURORDIS between October and November 2009, this survey aimed to determine and bring to the forefront, in which ways and to what extent patient organisations support research. The survey was designed to find out which research areas should be given priority from the patient’s perspective.

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

Over 770 rare disease patient organisations received an invitation to complete the on-line questionnaire, which was available in 6 languages (English, French, German, Hungarian, Italian, and Spanish). EURORDIS received 309 valid responses (a 40% response rate) representing 110 different rare diseases and 1.3 million patients in 29 European countries. The survey was undertaken in collaboration with the Centre de Sociologie de l'Innovation (Ecole des Mines, Paris, France).

EURORDIS Position Paper on Rare Disease Research 

Key findings:

  • Patient Organisations (POs) show a high interest in research
  • POs have a strong commitment to research despite great differences in terms of number of members and budget
  • POs show a strong willingness to collaborate with researchers, not only by arranging encounters between clinicians, researchers and patients, but also by giving them logistical and financial support
  • POs in Europe devoted a total minimum of 13 million euros to research in 2009 (not including funding from the Association Française contre les Myopathies – AFM - which gives on average 60 million euros to research every year)



  • 37% of POs had funded research in the last 5 years prior to the survey
  • Amongst them, half spend more than 30 000 euros yearly and one quarter spends more than 112 000 euros a year
  • Some devote more than 100% of their budget, which means they organise fundraisers specifically for research
  • POs who are 10 years or older spent about half of their budget on research


Amongst those POs who funded research:

  • 70% had not only funded a specific research project but had also initiated it
  • 75% had helped fund the operating budget of a research project
  • 54% bought equipment and 47% granted fellowships for young researchers



  • POs fund mostly basic research
  • Patients are interested in and concerned by all areas of research (basic, clinical, genetics, therapeutics, social science, etc)
  • 1 out of 2 patient organisations fund basic research and 1 out of 3 fund human and social science research. Contrary to the common belief that patients only support therapeutic research, it is clear that patient groups understand and invest in long-term research projects, as well


PATIENTS SUPPORT RESEARCH IN OTHER WAYS TOOmap of Europe showing survey results

  • POs support research mainly through actions aiming at creating links between patients, researchers and physicians
  • 1 in 2 POs support research by helping to identify patients to participate in clinical trials or by providing information and counselling for potential participants in clinical trials
  • Almost 1 in 2 POs collaborated in clinical trial design and helped define research projects by highlighting patients' needs and expectations
  • 1 in 3 POs participate in scientific committees within institutions, demonstrating that their legitimacy is increasingly acknowledged and that their desire to support research is also political


Correlation between the age of the PO and degree of involvement in research:

  • 25% of the POs who responded have spent more than 20 years supporting research. They are the ones who support research the most and are most satisfied with the results
  • The smaller, younger patient groups (25% of the POs who responded were less than 5 years old) are also actively involved in research by providing non-financial support. They give encouraging signs of increasing cooperation and progress in their relations with researchers



  • Patients say the highest priority for the allocation of public funds should be clinical research (therapeutic and diagnosis). Nevertheless, their funding allocation priorities do not coincide with the funding allocation priorities they expect from the public sector. Patient groups fund mostly basic research and epidemiological studies
  • Patient groups see themselves as partners, identifying the gaps and supporting the first steps of research left out by the public or private sectors



  • According to patients, the biggest obstacle in the progress of research is the lack of researchers and clinicians specialising in rare diseases and the lack of coordination amongst them. Most obstacles encountered are the direct result of the rareness of the disease. However, they also point to organisational problems, such as lack of coordination and multidisciplinary collaboration



  • Relations with researchers are very good for the biggest, oldest and most financially robust patient groups, (such as groups for neuromuscular disease and cystic fibrosis)
  • For the youngest POs with smaller budgets, their enthusiasm to support research is not matched by researchers, who are less willing to collaborate with them
  • Nevertheless, the older and most established patient organisations have increasingly good relations with researchers, which means that there is a virtuous circle of collaboration between patients and researchers – increased collaboration improves trust and leads to more collaboration
  • This means that there is hope for the smaller and younger patient groups to build increasingly good relations with the research community over time
  • In practice, it is important to address the expectations of young and small patient groups regarding closer ties with the research community



  • Rare disease patient organisations are playing an important role as catalysers of research on their respective diseases
  • Due to their intimate knowledge of their diseases and lay expertise, patient organisations are in a strategic position at the crossroads of all fields in research (from basic to therapeutic). Patients are in the best position to highlight the setbacks in both basic research and clinical applications as well as the weaknesses of translational research for their disease
  • Rare disease patient organisations are calling for a “research model” where basic and clinical research is conducted in parallel and through an intense dialogue between scientists, clinicians and patients. They are calling for partnering of all stakeholders
  • It is clear that despite their enthusiasm, POs do not have the means or the ambition to control research and conduct research by themselves. POs will not substitute public institutions but they can be valuable partners in research by:
  • Identifying the gaps and giving financial or logistic support to the initial stages of research projects that can be continued later by the public or private sector
  • By contributing their unique knowledge of each disease, and by establishing relations of trust between patients and researchers


A summary of this survey was presented at the European Workshop on Rare Disease Research, held on the occasion of Rare Disease Day 2010 (Brussels, 1 March, 2010)


Author: Paloma Tejada
Photos: Patient groups with umbrellas, Map of Europe © Eurordis;  3 researchers, handshake ©


Page created: 09/04/2010
Page last updated: 08/03/2016
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