What support the associations of patients with rare diseases are to researchers and what are their expectations of the research It is the subject of the survey of a sample of associations of the European Union by Eurordis (1) and the Centre for Sociology of innovation of the Ecole des mines de Paris. This study is presented today in Brussels, in a symposium on the theme "Building a bridge between the patients and research", organized by Eurordis and E-Rare (2), under the aegis of the European Commissioner for research, Máire Geoghegan-Quinn.
Better understanding of the disease
Rare diseases are a case in part because they are still poorly understood and often devoid of treatment. The isolation of patients, because of their low numbers in each pathology (sometimes 10 only at the European level) to group together in associations to try to be of interest to researchers and doctors to their cause. The spectacular growth of the Association against myopathies (AFM), organizer of the Telethon, which is at the head of a EUR 60 million research budget is of exception in European landscape where more than half of the associations have a total budget of less than 30,000 euros, or even no budget at all for more than 5. But they are small or large, rich or poor, they are very invested in research.
More than a third of the sample associations contribute to the budget of the laboratories. They finance mostly co-finance specific projects relating to their disease, allow the acquisition of a well-defined equipment or or supports a thesis or a postdoctoral fellowship. "These funds speakers in the margin to the operation of laboratories, but can play a crucial role of catalyst research start-up," analysis Vololona Rabeharisoa, Professor at the Centre of Sociology of the innovation of the Ecole des mines de Paris. Contrary to what one might imagine on the part of patients involved in a race of speed with a fatal disease, support for basic research (on the genetic origins or mechanisms of the disease) is a priority. Two-thirds the sample associations support and it is at the top in terms of contribution (50) in the research of treatments (38) or diagnostic tests (36). "The sick have integrated that without a good understanding of the disease, they fail to treat them," explains Vololona Rabeharisoa.
"So far, the financial contribution is not the essence of the contribution of associations", she continues. Most of them are, above all, actively to build links with specialists to develop knowledge about their disease. This is true for almost half of them up to the definition of research projects with scientists or participation in the design of clinical trials.
Progress remains to be done
The associations also play a crucial role in the mobilization of patients likely to participate in these clinical trials, as well as in the establishment of the clinical profile of the disease. Indeed, in conditions where patients are few and diverse symptoms know the experience of the disease is valuable for researchers. "The unique assets of associations, are sick they represent," observes Vololona Rabeharisoa. Without them, the work of researchers and physicians would be much more difficult, if not impossible. Many are aware and see associations as true partners (see below).
So far, the associations believe that there is still progress to. A third party are not satisfied with the information they have obtained in return projects that they had funded. And 40 feel unable to judge whether or not the results of the research they have supported were relevant. More generally, the low number of researchers and physicians working on these diseases, the lack of coordination between specialists and the difficulty to identify patients remain in the eyes of the main obstacles to the progress associations research on rare diseases.
Finally, if they are very active, associations do not have to aim to substitute for the public authorities. Because their financial means are limited, they are waiting in particular of the latter that they continue to support that therapeutic as well fundamental research, including by funding infrastructure and research institutions.
The point of view of Annick Schwebig, of Leem on lesechos.fr/interviewlesechos.fr/interview
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