European Joint Programme

European Joint Programme on Rare Diseases 2020 Proposal: Pre-Clinical Research to Develop Effective Therapies for Rare Diseases

The European Joint Programme on Rare Diseases (EJP RD) encompasses over 130 institutions from 35 countries. Together, they work to achieve a more sustainable and supportive environment for improved research, care and medical innovation.

The EJP RD have announced a call for funding into “Pre-Clinical Research to Develop Effective Therapies for Rare Diseases”. All proposals must incorporate rare diseases at the centre of their projects. Guidelines for research proposals can be found below.



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).


Visit the EJP RD website for more updates  and check out the Joint Transnational Call 2020 (JTC 2020) for a more in-depth description of the Pre-Clinical Research proposal.


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