Patients and researchers: Lifelong partners!
Rare disease patient organisations can be valuable partners in research – by funding research teams, equipment and training or by helping recruit patients for clinical trials. Patients often act as catalysers for increased collaboration amongst researchers, clinicians, industry and patients. Therefore it makes sense for patients and researchers to work closer together!
This is one of the main conclusions drawn from a survey recently conducted by EURORDIS in collaboration with a team of researchers from the “Centre de sociologie de l'innovation” (Ecole des Mines, Paris, France). The survey aimed to find out and bring to the forefront, in what ways and to what extent, patient organisations support research. It also examined priority areas and bottlenecks encountered, from the patient’s perspective.
772 patient organisations were invited to participate by filling out a questionnaire on-line, available in 6 languages. In total,
EURORDIS received 309 valid responses (an impressive 40% response rate) from members and non-members, representing 110 rare diseases and 1.3 million patients in 29 European countries.
The first part of the questionnaire shed light on the type of financial support that patients give to research. 37% declared having funded research, mainly by financing a specific project but also by granting fellowships to young researchers or by purchasing equipment. Of those funding research, a total contribution of 13 million euros was recorded over the last year (not including the AFM which alone gives an average 60 million euros to research every year!)
The survey revealed that patient organisations support research in others ways too. Almost all respondents declared having organised events to link researchers, clinicians and patients. They also aided clinical development by identifying patients for participation in clinical trials or by providing information and counselling for participation in clinical trials.
Concerning patient collaboration with researchers, most respondents reported being satisfied with the information provided by researchers about the results of the projects supported. ‘Interaction between patients and researchers is not always easy,’ says Francois Faurisson, Clinical Research Advisor at EURORDIS. ‘However, we can observe, that the older and most established patient groups (such as Neuromuscular and Cystic Fibrosis patient organisations) have increasingly good relations with researchers, which means that there is hope for the smaller and younger patient groups of increasing contacts with research personel and generating positive change in the years to come.’
When asked what areas of research the public sector should fund, the highest ranking went to clinical research (therapeutic and diagnosis). The survey also revealed that patient groups fund mostly basic research and epidemiological studies. ‘It is hardly surprising that their funding allocation priorities do not coincide exactly with the funding allocation priorities they expect from the public sector,’ argues Francois Faurisson. ‘Patient organisations are looking for complementary roles, as they are fully aware that understanding the disease (basic research) and curing the disease (therapeutic research) are two axes that must be supported concurrently as progress in one contributes to the other. The long history of several patient groups’ continuous commitment to basic research demonstrates their understanding that research is a slow and long-term process.’
Amongst the lowest ranking areas supported by patient groups were research infrastructures and collection of biological samples. ‘Patient groups seem to consider that these areas do not fall in their field of action or under their responsibility,’ argues Fabrizia Bignami, EURORDIS’ Research and Therapeutic Development Director. ‘It is obvious that patients do not and cannot replace public or private research institutions, but rather collaborate with them, as partners, bringing their own important contribution.’
The most commonly reported obstacles were lack of researchers and clinicians specialising in their diseases, and the lack of coordination amongst them. This was ranked higher the difficulty in identifying patients, which came in as the third biggest obstacle. ‘It is clear that patients are asking for more translational research, which will only happen with increased coordination,’ explains Fabrizia Bignami.
‘Patient organisations have a high commitment to research and have a strong willingness to collaborate with researchers. The survey confirms they fund research directly and that their participation is a crucial support in clinical trials,’ says Yann Le Cam, EURORDIS’ Chief Executive Officer. ‘But patients have limited budgets!
However valuable, their modest contribution will never meet the kind of investment needed to advance research into rare diseases. Therefore they are calling for more public funding, especially for clinical research.’
The results of this survey were presented at a European Workshop on Rare Disease Research organised by EURORDIS in Brussels as part of Rare Disease Day awareness raising activities. The main aim of this Workshop was to advocate for rare disease research to be included in and remain high on the EU and national research policy agendas.
Read more:
Newsletter article on the European Workshop “Building the Future Agenda of Rare Disease Research in Europe”, 1 March 2010, Brussels
Read more about the Major outcomes from the Rare Disease Day 2010 EU workshop
Author: Paloma Tejada
Photo credits: © Inserm & EURORDIS
This article first appeared in the May 2010 issue of the EURORDIS newsletter.
Page created: 01/05/2010
Page last updated: 16/08/2011
