Overview

Country(s): Austria, Belgium, Canada, Czech Republic, Estonia, Finland, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Lithuania, Netherlands, Poland, Portugal, Slovakia, Spain, Sweden, Switzerland, Turkey,

Deadline: 14-02-2019 Pre-proposals (TBC - Call opening 14 December 2018)

Deadline:11-06-2019 Invited full proposals (TBC - full proposals to be invited by early May 2019)

PROGRAMME SUMMARY

The ERA-Net E-Rare has successfully implemented ten Joint Transnational Calls for rare disease research projects since 2006. This effort is now continued in the frame 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 benefit for patients.

The topic of this Call is ‘Research projects to accelerate diagnosis and/or explore disease progression and mechanisms of rare diseases’.

The FNR, along with funding organisations in 22 other countries in January 2019 will open the first EJP RD Joint Transnational Call (JTC 2019) for funding multilateral research projects on rare diseases together with the European Commission (EC) under the EJP-COFUND mechanism.

VIEW AIMS OF EJP RD CALL

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

  1. Research to accelerate diagnosis, e.g:
  • New schemes for finding diagnosis for undiagnosed patients;
  • Improved annotation and interpretation of variants and development of diagnostic tests for the more prevalent variants;
  • Novel modalities of functional analysis of candidate variants through in vitro, cell, tissue or animal studies.
  • -omic or multi-omic integrated approaches for discovery of disease causes and mechanisms including development of relevant bioinformatic tools;
  1. Research to explore disease progression and mechanisms, e.g:
  • Natural history studies and patient registries (also for clinical trial readiness). Whenever possible these should include development and use of patient reported outcome measures. In addition, the exploration of the use of standardized M-Health-based surveillance instruments and of patient entered data to gather information for natural history studies is welcome;
  • Identification of clinical biomarkers, clinical outcome measures and surrogate endpoints;
  • Identification of novel pathophysiological pathways in appropriate disease models that effectively mimic the human condition.

Furthermore, additional elements need to be considered in the application:

  • The design of the study (sample collection, statistical power, interpretation, relevant models for hypothesis validation) must be well justified and has to be part of the proposal;
  • For natural history studies and patient registries: strategies and timelines for patient recruitment, retention, assessment, and analysis must be included. Data supporting the proposed recruitment numbers is mandatory. The study design and objectives should take into consideration what information regarding the rare disease population would be needed in order to pursue clinical trials or other health care related studies in that rare disease. There always need to be clear research questions that are addressed in the study/registry. Clear plans for sustainability of the resources must be described. Consideration of common data elements as outlined in the recent publication “Set of Common Data Elements for RD Registration” (http://www.erare.eu/sites/default/files/SetCommonData-EU%20RD%20Platform_CDS%20_final.pdf) is highly recommended;
  • Appropriate bioinformatics and statistical skills should constitute, whenever justified, an integral part of the proposal, and the relevant personnel should be clearly specified;
  • The new research data resulting from the project should be treated permissible according to the FAIR[1] principles, and deposited and shared, according to the national/regional rules of the countries involved. It is strongly advised to make data accessible through RD-Connect (http://rd-connect.eu/ – connecting databases, patient registries, biobanks and clinical bioinformatics data into a central resource for researchers worldwide) and through Elixir (https://www.elixir-europe.org/platforms/data/elixir-deposition-databases – compiling a list of resources for the deposition of experimental, biomolecular data). To make research data findable, accessible, interoperable and re-usable (FAIR), a data management strategy for the proposed full project is mandatory in the full proposal stage. Some countries involved in EJP RD JTC 2019 will also ask for a data management plan (DMP) at national level at the stage of full proposal or after granting of the project.
  • To ensure that the needs and priorities of rare disease patients are adequately addressed, they or their representatives should be appropriately involved in all projects wherever relevant. For examples, inclusion and involvement of patient representatives includes but is not restricted to natural history studies / registries where patients should be involved in the governance of the registry. Please consult the INVOLVE website for information on various ways to involve patients: http://www.invo.org.uk/resource-centre/resource-for-researchers/. For additional guidance and practical advice on patient involvement in research studies, please consult also the JPND guidelines: http://www.neurodegenerationresearch.eu/wp-content/uploads/2013/11/JPND-guide-for-Patient-and-Public-Involvement.pdf .

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 pre-clinical therapy development and/or validation in cellular or animal models. These will be addressed in future calls;
  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/). These concern: 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. Not excluded through this specification are childhood dementias/neurodegenerative diseases.

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 research projects submitted within this call must be based on novel ideas stemming from consolidated previous results or preliminary data and must be clearly endowed with benefit for the patients, i.e. studies allowing a rapid implementation into public health-related decisions or into the clinics. To achieve this goal, the necessary expertise and resources should be brought together from academia, clinical/public health sector and private companies whenever relevant. The research teams within a consortium should include investigators from complementary scientific disciplines, research areas and expertise necessary to achieve the proposed objectives.

The research proposals must demonstrate complementary and synergistic interaction among the partner teams. There should be clear added value in the transnational collaboration over the individual projects, in terms of:

  1. Gathering a critical mass of subjects/patients and or subjects/patients databases and corresponding biological materials that would not be possible otherwise;
  2. Sharing of resources (biobanks, models, databases, diagnostic tools, etc.), of specific know-how and/or innovative technologies including “-omics”, and of expertise. The projects should clearly demonstrate the potential health impact.

[1] FAIR: Findable, Accessible, Interoperable, Reusable (for more information: see “The FAIR Guiding Principles for scientific data management and stewardship” (https://www.nature.com/articles/sdata201618)

VIEW GENERAL APPLICATION CONDITIONS

Joint research proposals may be submitted by applicants belonging to one of the following categories (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)
  • enterprise (all sizes of private companies). Participation of small and medium-size enterprises (SMEs) is encouraged when allowed by national/regional regulations
  • patient advocacy organisations (PAOs – see more information below and refer to the INSERM contact point)

Please note that the inclusion of a non-eligible research partner (principle investigator) in a proposal leads to the rejection of the entire proposal without further review. 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 2019. The applications are therefore subjected to eligibility criteria of individual funding organisations. Applicants are strongly advised to contact their corresponding national/regional representative and confirm eligibility with their respective funding organisations in advance of submitting an application (see national/regional contact details).

The consortium coordinator must always be eligible to receive funding from the funding organisations participating in the call and cannot be a partner that joins only with their own funding. Only groups that contribute substantially to at least one of the work packages are considered as partners and should be indicated in the project.

Consortia are also encouraged to include Early Career Scientists as principal investigators in their proposal. Early career PIs must prove that they are scientifically excellent and independent, for example that they lead or have led a research group or project. They also must clearly be eligible according to national/regional funding regulations. Early Career Scientists should be clearly identified in the proposal and their CV.

Additional research partners that secure their own funding may join consortia. However, their number is limited to two and depends on the number of research partners requesting funding (see table below). These additional research partners can only come from countries that are not involved in the EJPRD JTC 2019 funding or are not eligible for the respective funding organization due to national/regional rules. These research partners must state clearly in the proposal if these funds are already secured or if not, how they plan to obtain funding in advance of the project start, as well as what the concrete amount of contributed funding will be. It will be required to document the availability of their funds before October 1, 2019. In the (pre)proposal form these research partners are mentioned in the category «Associated research partners not asking for funding».

To collect the necessary patient data and/or samples for the proposed study, a consortium may need to collaborate with other centres. If the unique role of those centres is providing patients data and/or samples for the study only, they will not be considered as research partners of the consortium but can be included otherwise, e.g. via cooperation agreements or subcontracting.

View full text on EJP RD website

FUNDING SPECIFICS

The FNR supports Luxembourg partners in this Call with a maximum of 300,000 EUR.

APPLICATION INFORMATION

Submission

Both pre- and full proposals must be written in English and must be submitted to the Joint Call Secretariat (JCS) by the coordinator through the electronic submission system exclusively. Specific information to follow.

In addition, Luxembourg partners must submit both pre and full proposals, along with the FNR INTER Call documents to the FNR no later than 5 working days after the EJP-RD deadline. Applications to the FNR must be submitted via the FNR’s Online Grant Management System.

Call timeline

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

  • Call opening: Friday, 14 December 2018
  • Pre-proposal indicative deadline: Thursday, 14 February, 2019.
  • Invitation to submit full proposal: by early May 2019
  • Full proposal indicative deadline: Tuesday, 11 June 2019
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