PRE-CLINICAL RESEARCH TO DEVELOP EFFECTIVE THERAPIES FOR RARE DISEASES
This is a Preliminary Announcement. The content and procedures of the call described in this pre-announcement may be subject to change and are not legally binding.
The ERA-Net E-Rare has successfully implemented ten Joint Transnational Calls for rare disease research projects since 2006. This effort continues in the framework of the European Joint Programme on Rare Diseases (EJP RD) that has been established to further help in coordinating the research efforts of European, Associated and non-European countries in the field of rare diseases and implement the objectives of the International Rare Disease Research Consortium (IRDiRC).
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 future benefit for patients.
Research proposals must cover at least one of the following areas:
- 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
- Use of disease models suitable for medicinal product’s development according to EMA guidelines
- 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
- 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 the call:
- Therapeutic approaches concerning rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases
- Interventional clinical trials
- Surgery or radiation therapies
- 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.
- Rare neurodegenerative diseases which are within the main focus of the Joint Programming Initiative on Neurodegenerative Disease Research (JPND). These are: Alzheimer’s disease and other dementias; Parkinson’s disease (PD) and PD-related disorders; Prion disease; Motor Neuron Diseases; Huntington’s disease; Spinal Muscular Atrophy and dominant forms of Spinocerebellar Ataxia. Interested researchers should refer to the relevant JPND calls.
Childhood dementias/neurodegenerative diseases are not excluded.