European Join Programs on Rare Diseases (EJP RD)

Call for Proposals 2023: «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 (eg 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;

5. Identification of biomarkers/variables for therapeutic approaches (pharmacology, drug repurposing, gene therapy, RNA therapy, cell therapy, medical devices...).

Deadline:

Pre-proposal: February 15th, 2023

Full proposal: June 16th, 2023

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