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:

• Estimation of disease prevalence;
• Identification of biomarkers/companions for the diagnosis/prognosis of a RD;
• 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….);
• Identification of relevant endpoints for future studies that include potential biomarkers, querying patient-reported outcomes (PROs) and quality-of-life measures;
• Identification of biomarkers/variables for therapeutic approaches (pharmacology, drug repurposing, gene therapy, RNA therapy, cell therapy, medical devices…).

It is possible to use cellular and animal models for validation of endpoints and biomarkers uncovered in the natural history studies where relevant.

Timeline and eligibility criteria:

• 12^th of December 2022 – Call opening
• 15^th of February 2023 – pre-proposal submission
• 14^th of June 2023 – full-proposal submission
• DFG and BMBF funding
• Funding for transnational joint projects with min four partner from four participating countries
• maximum of six partners (eight und certain conditions)

Participating countries:

Belgium, Canada, France, Germany, Hungary*, Ireland, Israel, Italy, Lithuania, Luxembourg, Poland, Slovakia, Spain, Sweden, Switzerland, Turkey

*Decision pending

Further information:

https://www.ejprarediseases.org/joint-transnational-call-2023/

Contact at EPC:

© TU Dresden

Project Manager

Name

Mr Dr. Stefan Schuldt

ERA-Nets, BMBF Complementary measures

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Contact Information

Organization Name

European Project Center

European Project Center

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