Rare diseases: Launching of the RaDiCo platform and sixteen initial rare disease cohorts

The RaDiCo project, which is funded by the French government under the first “Investments for the Future” programme, aims at providing France and Europe with cohorts that lead to a better understanding of rare diseases and thus improve their management and the development of treatments. The first call for proposals has now ended, and the platform yesterday officially launched sixteen studies.

The term "rare diseases" designates a group of highly diverse pathologies characterized by low frequency of occurrence (less than one person affected out of 2,000). Some 7,000 pathologies of this type have been described. Although each of these diseases is rare, the total number of people affected is high: more than 30 million people in Europe and 3 million in France. Knowledge about these diseases and therefore the available treatments remains limited at present. Consequently, these diseases represent a major public health issue.

A centralised platform of resource expertise

With funding totalling almost €10 M under the "Cohorts" action of the first "Investments for the Future" programme, the RaDiCo project aims at allowing the large-scale collection of data on patients affected by rare diseases: state of health, symptoms, quality of life, etc. These data will provide inputs for research work, from the most fundamental (mechanisms subtending the disease, determinants/risk factors, etc.) to the most applied, to ultimately achieve progress in the diagnostic methods, the treatments, and more generally the management of these diseases.

Coordinated by Inserm (French National Health and Medical Research Institute) and hosted by the Trousseau Hospital, RaDiCo constitutes a centralised platform of resources and tools for setting up and monitoring cohorts. The project will be funded by the French State until December 2019. The project is also supported by a large network that brings together 131 reference centres for rare diseases (CRMR) grouping highly specialised research hospital teams, 501 competence centres for rare diseases (CCMR) at regional level, 54 research hospital molecular diagnostic laboratories, biological resource centres, research laboratories and the patients associations concerned.

Sixteen initial pilot projects

Once it had completed the phase of setting up its governance and operational platform, RaDiCo launched a call for proposals in January 2014 aiming at selecting a first series of pilot projects that it accompanies for the development of rare disease cohorts. In July 2014, sixteen projects were thus selected. On completion of the joint preparatory work between the RaDiCo operational team and the chosen project principal investigators, sixteen cohorts concerning rare diseases or groups of diseases were officially launched yesterday.

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What is a cohort study?

A cohort study consists in monitoring a population of subjects, healthy or ill, for several years or several decades in order to study numerous health determinants and their interactions with genetic and environmental factors over the long term.

The "Cohorts" action of the Investments for the Future programme

The aim of the Cohorts call for proposals was to guarantee the long-term funding of cohorts subtended by health problems, whether they were general population cohorts or patient cohorts. In this context, ten projects are funded for a total sum of €74.5 M over ten years (€7.4 M per project on average). Among the ten chosen cohorts, two are "general population" cohorts while eight are cohorts of patients suffering from the following diseases: rare diseases, multiple sclerosis, psychiatric illnesses, renal insufficiency, breast cancer, paediatric cancers, bladder cancer, and lastly, graft-versus-host disease.

ANR and Investments for the Future

In 2010, ANR was designated principal manager of the French Government for the actions of the Investments for the Future programme in the area of higher education and research. The actions of the Investments for the Future programme managed by ANR concern the centres of excellence, health, biotechnologies and the field of technology transfer and research valorisation for a total budget of more than 20 billion euros. Within the framework of the second Investments for the Future programme, ANR has been confirmed as manager on the themes of higher education and research for a budget of 4.015 billion euros for the following actions: Equipment of excellence (EQUIPEX), Initiatives of excellence (IDEX), Research hospitals and generic key technologies.