JTC 2020 on Rare Diseases within European Joint Programme on Rare Diseases (EJP RD)

The French National Research Agency will launch, in partnership with 23 countries, the second joint transnational call of the European Joint Programme on Rare Diseases for funding “Pre-clinical research to develop effective therapies for rare diseases”.

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 a clear benefit for patients. Projects shall involve a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10.000 persons in the European Community, EC associated states and Canada.

Transnational research proposals must cover at least one of the following areas, which are equal in relevance for this call:

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

1.    Development of novel therapies in a preclinical setting (including small molecules, repurposing drugs, cell and gene advanced therapies) focusing on condition(s) with unmet medical needs
2.    Use of disease models suitable for medicinal product’s development according to EMA guidelines
3.    Development of predictive and pharmacodynamics (PD) biomarkers (with appropriate analytical methods e.g. OMICS) in a preclinical setting (e.g. in the validated model or in pre-collected human samples) for monitoring the efficiency of the therapy. The model chosen must mimic the human diseases and be transposable so that the biomarker identified in animals can be valid for humans
4.    Proof of principle studies fostering an early (pre-clinical) stage of drug development (excluding interventional clinical trials of phase 1-4).

The following approaches and topics are excluded from the scope of this call: (1) Approaches concerning rare infectious diseases or rare cancers; (2) Approaches concerning rare adverse drug events/medical complications in treatments of common diseases; (3) Studies that focus on research to accelerate diagnosis or to set up new registry/cohort studies to explore disease progression and mechanisms as these were the focus of JTC 2019; (4) Interventional clinical trials; (5) Rare neurodegenerative diseases which are within the main focus of the Joint Programming Initiative on Neurodegenerative Disease Research (JPND; http://www.neurodegenerationresearch.eu/).

Projects shall involve a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10.000 persons in the European Community, EC associated states and Canada. Applicants are encouraged to assemble groups of rare diseases based on solid criteria and commonalities if this leverages added value in sharing resources or expertise and has the capacity to elucidate common disease mechanisms and therapeutic targets.

The French National Research Agency will finance solely French partners of selected projects, and for a maximum duration of 3 years.