Fundação para a Ciência e Tecnologia (FCT) participates in the European Joint Programme on Rare Diseases EJP RD that brings together over 130 institutions from 35 countries to create a comprehensive, sustainable ecosystem allowing a virtuous circle between research, care, and medical innovation. Portugal is also represented in this programme by the Ministry of Health through the Instituto Nacional de Saúde Dr. Ricardo Jorge (INSA).
The Recommendation 2009/C 151/02 from the Council of the European Union recognised rare diseases (RD) as a prime example of a research area that can strongly profit from coordination on a European and international scale. RD research should be improved to overcome fragmentation, leading to efficacious use of data and resources, faster scientific progress and competitiveness, and most importantly, to decrease unnecessary hardship and prolonged suffering of RD patients.
In a time of massive generation of data, need for the reuse and efficient interpretation of data, introduction of omics into care practice, and structuration of RD care centres in European Reference Networks, it is crucial and timely to maximize the potential of already funded tools and programmes by supporting them further, scaling them up, linking, and most importantly, adapting them to the needs of end-users through implementation tests in real settings. To achieve this goal, the European Joint Programme on RD (EJP RD) has two major objectives:
(i) To improve the integration, the efficacy, the production and the social impact of research on RD through the development, demonstration and promotion of Europe/world-wide sharing of research and clinical data, materials, processes, knowledge and know-how;
(ii) To implement and further develop an efficient model of financial support for all types of research on RD (fundamental, clinical, epidemiological, social, economic, health service) coupled with accelerated exploitation of research results for benefit of patients.
To this end, the EJP RD is organised in four major Pillars assisted by the central coordination and transversal activities: Pillar 1: Funding of research; Pillar 2: Coordinated access to data and services; Pillar 3: Capacity building; Pillar 4: Accelerated translation of research projects and improvement outcomes of clinical studies.
18 December 2019: Call Launch
18 February 2020: Deadline for submission of pre-proposal (2:00 p.m. CET)
16 June 2020: Deadline for submission of full-proposal
Please consult this webpage on a regular basis for possible news/updates.
The "Call Documents" contain detailed information on the call topic, participating countries and eligibility. Please consult the documents and selected links available in the yellow box on the right.
Pre-clinical Research to Develop Effective Therapies for Rare Diseases
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).
Austria, Belgium, Canada, Czech Republic, Finland, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Lithuania, Luxembourg, Poland, Portugal, Slovakia, Spain, Sweden, Switzerland, The Netherlands and Turkey.
1- Please consult the Call Documents in the yellow box on the right.
2- Consult the National Documents available in the yellow box on the right. Also read the specific National funding regulations of all the participating countries involved in your consortium (including your own), available in the Guidelines for Applicants. Non-eligible partners may lead to the rejection of the whole consortium.
3- Access to the online Submission Platform.
4- Each Portuguese partner (or coordinator) must send to the Call National Contact Point a Statement of Commitment (a single pdf file) duly signed, dated and stamped by the Head of the Portuguese applicant organization and by the Principal Investigator, until 10 days after the submission deadline. FCT may later require the original of the statement of commitment.
National funding commitment for the call: 300.000 €
Maximum requested funding for a consortium coordinated by Portugal: 250.000 €
Maximum requested funding for a consortium with Portuguese participation : 150.000 €
Note: if more than one Portuguese institution participates in a given consortium, the budget has to be shared. Portuguese institutions must follow FCT's Regulation on Projects Funded Solely by National Funds. The dedication (FTE) in transnational projects is not taken into account for the 100% (FTE) dedication to national projects.