The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with expected impact to use the results in the future for benefit of patients.

Topic: Natural History studies addressing unmet needs in Rare Diseases

The objective of this call is to conduct efficient, innovative and high-quality natural history studies which will facilitate understanding of the disease's or group of disorders' progression throughout the lifespan of a patient. The goal of these studies is to collect and analyze comprehensive patient data to define targets for future therapies, taking into consideration innovation, safety, and efficacy.

Research proposals should cover at least one of the following areas:

1. Estimation of disease prevalence
2. Identification of biomarkers/companions for the diagnosis/prognosis of a RD
3. Identification of biomarkers/indicators/predictors of a rare disease or group of disorders (e.g. having the same aetiology) onset/progression (including collection of genetic, physiological, environmental data or variables....)
4. Identification of relevant endpoints for future studies that include potential biomarkers, querying patient-reported outcomes (PROs) and quality-of-life measures to identify the most
5. Identification of biomarkers/variables for therapeutic approaches (pharmacology, drug repurposing, gene therapy, RNA therapy, cell therapy, medical devices...)

The call is expected to be opened in the following countries/regions:Austria, Belgium, France, Canada, Germany, Hungary, Ireland, Israel, Italy, Lithuania, Luxembourg, Poland, Slovakia, Spain, Sweden, Switzerland, Türkiye. 

Projects shall involve a group of rare diseases or a single rare disease following the European definition. 

Applicants are encouraged to assemble groups of rare diseases based on solid criteria and commonalities if these leverages added value in sharing resources or expertise and has the capacity to elucidate common disease mechanisms and therapeutic targets.

Partners belonging to one of the following categories may request funding under a joint research proposal (according to country/regional regulations):

1. academia (research teams working in universities, other higher education institutions or research institutes),
2. clinical/public health sector (research teams working in hospitals/public health and/or other health care settings and health organizations),
3. enterprises (all sizes of private companies) when allowed by national/regional regulations,
4. patient advocacy organizations.

The maximum duration of the project is three years.

Only transnational projects will be funded.

Each consortium submitting a proposal must involve four to six eligible principal investigator partners from at least four different participating countries. No more than two eligible partners from the same country can be present in each consortium.

The number of partners can be increased to 8 in two cases:

1. The inclusion of partners from participating countries usually underrepresented in projects (Slovakia, Hungary, Lithuania, Poland, Sweden and Türkiye).
2. The inclusion of Early Career Researchers as full partners.

Each transnational proposal must nominate a project consortium coordinator among the project partner principal investigators. The project coordinator will represent the consortium externally, to the JCS and to CSC, and will be responsible for its internal scientific management (such as controlling, reporting, and intellectual property rights issues). This workload should be taken into account in the estimation of the budget of the coordinator. A single principal investigator will represent each project partner. Within a joint proposal, the principal investigator of each project partner will be the contact person for the relevant country/regional funding organization.

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