INTRODUCTION

Conducted by the European Organisation for Rare Diseases (EURORDIS) between October & November 2009 this survey aimed to find out and bring to the forefront, in what ways and to what extent, patient organisations support research. The survey was designed to find out which are the research areas that should be given priority to, from the patient’s perspective.

The results of this survey will help to 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.

PARTICIPANTS' BACKGROUND & RESPONSE RATE

772 rare disease patient organisations received an invitation to fill out the on-line questionnaire, which was available in 6 languages (EN, FR, DE, IT, ES, HU)

EURORDIS received 309 valid responses from members and non-members (40% response rate); representing 110 rare diseases and 1.3 million patients in 29 European countries.  

This work was undertaken in collaboration with the Centre de Sociologie de l'Innovation (Ecole des Mines, Paris, France)

PATIENTS FUND RESEARCH

• 37% of POs funded research in the last 5 years
• Amongst them, half spend more than 30 000 euros yearly and one quarter spend 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% had granted fellowships for young researchers

WHAT KIND OF RESEARCH DO PATIENTS FUND?

• POs fund mostly basic research.
• Patients are naturally interested in all areas of research since they are concerned by many areas of research (genetics, therapeutics, social, 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 TOO

• Patient organisations support research mainly through actions aiming at creating links between patients, researchers and physicians.
• 1 in 2 PO 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 PO participate in scientific committees within institutions, which reveals 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 PO who responded have less than 5 years of existence) 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.

WHAT ARE PATIENTS’ PRIORITIES?

• 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

WHAT ARE THE OBSTACLES THEY’VE ENCOUNTERED?

• 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 multidisciplinarity.

RELATIONS BETWEEN PATIENTS AND RESEARCHERS: BRIDGING THE GAP

• Relations with researchers are very good for the biggest, oldest and richest patient groups, (such as Neuromuscular and Cystic Fibrosis)
• For the youngest PO with smaller budgets, their level of enthusiasm to support research is not matched by that of 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 links with the research community.
  

CONCLUSIONS

• 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 basic research and clinical applications and the weaknesses of translational research for their disease. 
• Rare diseases 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 carry all 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 taken on 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, March 1st, 2010)

Read more about the Major outcomes from the Rare Disease Day 2010 EU workshop