Pre-announcement European Joint Programme on Rare Diseases (EJP RD) – Joint Transnational Call 2023 -“Natural History Studies addressing unmet needs in Rare Diseases”.

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…).

It is possible to use cellular and animal models for validation of the new diagnostic approaches in the subtopics listed above where relevant.
Furthermore, additional elements need to be considered in the application and there are some approaches and topics excluded from the scope of the call JTC2023 (see additional information).

Maximum duration of the project: 3 years.

Number of partners: 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 (further national limits may apply). The number of partners can be increased to 8 in two cases (see additional information).

Timetable: There will be a two-stage submission procedure for joint applications: pre-proposals and full proposals (see additional information).

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

Plazo de Presentación: 

Pre-proposal: 15/02/2023

Full proposal: 14/06/2023