Introduction

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).

Aim

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.

Topic: PRE-CLINICAL RESEARCH TO DEVELOP EFFECTIVE THERAPIES FOR RARE DISEASES

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 this 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.

Consortium Make-up

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.

Partners belonging to one of the following categories may request funding under a joint research proposal (according to country/regional regulations):

• academia (research teams working in universities, other higher education institutions or research institutes)
• clinical/public health sector (research teams working in hospitals/public health and/or other health care settings and health organisations)
• enterprises (all sizes of private companies). Participation of small and medium-sized enterprises (SMEs) is encouraged when allowed by national/regional regulations
• patient advocacy organisations (PAOs are eligible to obtain funding for their participation in research projects)

The maximum duration of the project is three years.

Only transnational projects will be funded. Each consortium submitting a proposal must involve four to six eligible principal investigator partners. No more than two eligible partners from the same country can be present in each consortium (further national limits may apply). This limit applies to Early Career Researchers and partners from underrepresented countries (see below). PAOs requesting funding do not count toward this total.

The number of partners can be increased to 8 in two cases:

• The inclusion of partners from participating countries usually underrepresented in projects (Czech Republic, Slovakia, Hungary, Lithuania, Poland, and Turkey).
• The inclusion of Early Career Researchers as full partners.

Eligible Countries

Austria; Belgium; Canada; Czech Republic, Finland; France; Germany; Greece; Hungary; Ireland; Israel; Italy; Lithuania; Luxembourg; Poland; Portugal; Slovakia; Spain; Sweden; Switzerland; The Netherlands; Turkey.

Budget

The funding depends on national/regional rules. For Spanish entities, up to € 250,000 for coordinators and € 175,000 for partners.

Duration

The maximum duration of the project is 3 years

Deadlines

There will be a two-stage submission procedure for joint applications: pre-proposals and full proposals.

Important

Whilst applications will be submitted jointly by applicants from several countries/regions, individual groups will be funded by the individual funding organisation of their country/region that is participating in the EJPRD JTC 2020. The applications are therefore subjected to eligibility criteria of individual funding organisations.

Due to the complexity of the call, if you are interested in submitting an application we strongly recommend that you contact FSJD- Proyectos Internacionales (linternacional@fsjd.org) well in advance of the deadline.

Web

http://www.ejprarediseases.org/index.php/open-call-jtc2020/