ERDERA Joint Transnational Call 2025 "Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation"

Beneficiary & submission

European Rare Diseases Research Alliance (ERDERA) Joint Transnational Call (JTC) 2025 "Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation" For a complete set of information, including the full call (incl. annexes and templates), please refer to the ERDERA website ERDERA Research consortia who intend to submit a transnational project proposal should register as soon as possible via the electronic proposal system: https://funding.erdera.org There will be a two-stage submission procedure for joint applications: a pre- and full proposal stage. In both cases, one joint proposal document (in English) shall be prepared by the partners of a joint transnational proposal and must be submitted by the coordinator only to the Joint Call Secretariat (JCS) via the electronic submission system. Proposals must be prepared using the templates provided in the electronic system. Proposals not conforming to template instructions will be rejected. 10th December 2024: Launch of the call 17th December 2024: Information webinar for potential applicants 13th February 2025: Pre-proposal submission deadline 3rd March 2025: Pre-proposal eligibility check Early May 2025: Invitation to full proposal 6th May 2025: Information webinar for applicants invited to submit a full proposal 9th July 2025: Full proposal submission deadline 18th July 2025: Full proposal eligibility check December 2025: Notification of funding decision Full proposals will be accepted only from those applicants who were explicitly invited by the JCS to submit them. In general, fundamental changes between the pre- and full proposals concerning the composition of the consortia, objectives of the project, or requested budget can only be applied with a detailed justification provided to the JCS for consideration by the Call Steering Committee. Potential justifications may include advice gathered on the feasibility of the project indicating the need for additional expertise and/or resources, or the addition of partner(s) through the widening scheme. However, the national/regional regulations on eligible partners and budget caps will still apply and the budget change needs to be pre-approved by relevant national/regional funding organisation(s). Further information on how to submit pre-proposals and full proposals electronically (including “Guidelines for Applicants” and submission templates) is available at the ERDERA website. Evaluation Process At the pre-proposal stage, applicants should focus on presenting the scientific idea/hypothesis and supporting preliminary results, studies or data. The proposal should describe the project, starting from an unmet need, and follow through to the expected endpoint of the study. At the full proposal stage, in addition to the scientific content, a full description of the patient engagement plan, data management, statistical methods, and ethical and legal issues will be required in compliance with EC requirements. Applicants should anticipate these requirements and ensure that they have consulted relevant experts to verify the feasibility of the project, and that the proposal can be completed within the defined timelines and budget (considering budget limits listed in the “Guidelines for Applicants”). Evaluation Criteria Evaluation scores will be awarded according to specific evaluation criteria that are in line with Horizon Europe rules (see below), using a common evaluation form. 7.1.1 Scoring system 0: Failure: The proposal fails to address the criterion in question or cannot be judged because of missing or incomplete information. 1: Poor: The proposal shows serious weaknesses in relation to the criterion in question. 2: Fair: The proposal generally addresses the criterion, but there are significant weaknesses that need corrections. 3: Good: The proposal addresses the criterion in question well, but certain improvements are necessary. 4: Very good: The proposal addresses the criterion very well, but small improvements are possible. 5: Excellent: The proposal successfully addresses all aspects of the criterion in question. 7.1.2 Criteria 1.    Excellence (0-5) a.Clarity and pertinence of the objectives; b.Credibility of the proposed approach and methodology; c.Soundness of the concept; d.Innovative potential: description of existing development landscape, relationships with technology transfer offices, plan for ongoing development, involvement of industry partners; e.Competence of participating research partners in the field(s) of the proposal (e.g., previous work in the field, specific expertise), and; f.**Active and meaningful participation of Patients Organisations (PAOs) and patient partners in the project (including in the design and definition of research priorities, interpretation and implementation of results, their dissemination, and communication) as well as clarity of the patient partner involvement plan. 2.    Impact (0-5) a.    *Potential and readiness of the expected results to be translatable for future clinical applications for diseases without approved treatment options in Europe; b.*Benefit to patients, their families, and carers; c.*Added value of transnational collaboration: gathering a critical mass of patients/material, sharing of expertise and resources, harmonisation of data, sharing of specific know-how and/or innovative solutions, and; d.      **Effectiveness of the proposed measures to exploit and disseminate the project results (including management of intellectual property rights (IPR)), to communicate the project, and to manage research data. A data management strategy in the full proposal is mandatory. 3.    Quality and efficiency of the implementation (0-5) a.Feasibility of the project (coherence and effectiveness of the work plan, including appropriateness of the allocation of tasks, resources and timeframe, access to data and material); b.Complementarity of the participants within the consortium, including the integration of PAOs or patient partners; c.**Appropriateness of the management structures and procedures, including risk management, contingency plans and innovation management, and; d.**Plan for sustainability of infrastructures or resources initiated by the project. *Sub-criteria 2a, 2b and 2c will be prioritized for assessing the impact of proposals (pre- and full proposal stage). **Sub-criteria 1f, 2d, 3c, 3d will only be assessed as part of the full proposal evaluation step. Pre-proposal Review Eligibility check The JCS will check all pre-proposals to ensure that they meet the call’s formal criteria (completeness of information in submission platform, general eligibility criteria). The JCS will forward the proposals to the CSC members who will perform a check for compliance to country/regional eligibility rules. Please note that proposals not meeting the formal criteria or the national/regional eligibility criteria and requirements will be declined without further review. Peer review of pre-proposals Pre-proposals that pass the eligibility check will be forwarded to the Scientific Evaluation Committee (SEC) members with biomedical expertise for a first evaluation (see evaluation criteria above). These SEC members will perform the assessment of the pre-proposal and fill the evaluation forms with scores and comments for each criterion. Each pre-proposal will be assessed by 2 SEC members. The SEC members will then meet online to establish a ranking of the pre-proposals. This ranking will be used by the CSC to decide which pre-proposals will be accepted for full proposal submission. Applicants will receive their individual consensus review report and corresponding SEC recommendations regarding full proposal submission. Scoring rules Each criterion will be scored out of five (5). The weighting factor for the Excellence criterion will be two. Therefore, the maximum overall score for each proposal will be 20 points. To be approved for the full proposal stage, the application must receive a minimum threshold score of 3 (out of 5) for each individual evaluation criterion; in addition, the application must receive a minimum overall score of 15 points per expert vote. Widening At the end of this stage research teams of underrepresented or undersubscribed countries may join successful pre-proposals after approval by the relevant funding organizations (see 5.2 in “Guidelines for Applicants” for more details). ERDERA support services  Applicants that are invited to submit a full proposal are strongly encouraged to make use of the ERDERA support services for FAIR data, translational mentoring and regulatory advice (see 5.3 in “Guidelines for Applicants” for more details).  Full Proposal Review Formal criteria check The JCS will check the full proposals to ensure that they meet the call’s formal criteria with the help of the CSC. SEC meeting evaluation The JCS will send full proposals to the SEC members. The SEC will meet to discuss the proposals, assign their final scores and create a ranking list with proposals that are recommended for funding. The final summary review report for each consortium will be prepared by the SEC members and sent to respective applicants. Scoring rules Each proposal will be evaluated by three biomedical reviewers (including methodological reviewers) and one patient reviewer. For biomedical SEC members, each criterion will be scored out of five. The weighting factor for the criterion excellence will be two. Therefore, the maximum overall score will be 20 points. The threshold for an individual criterion is three, with an overall threshold of 15 points per expert vote. In addition, the full proposals will be reviewed by a patient reviewer according to the relevant evaluation criteria listed above (see 7.1.2; subcriteria 1f, 2b, 3b). The maximum score per patient expert is 15 points with a threshold of 10 points. Patient reviewers will be present at the SEC meeting to discuss proposals and provide their feedback. Based on the above, the overall maximum score that can be given to a consortium, through the addition of individual expert scores, is 75 points. The minimum threshold to be considered for funding will be 55 points, with each expert meeting their respective threshold as described above. Ethical evaluation After the second SEC meeting, full proposals recommended for funding by the SEC will be remotely evaluated by independent experts in ethics. These experts will report on the feasibility of a given proposal to comply with the ethical requirements. If necessary, they will list those tasks to be done and documents to be submitted by the consortium in order to receive approval for funding from an ethics standpoint. Only those proposals approved by both the scientific and ethical evaluations (complying with all central Horizon Europe and regional/national ethical requirements), will be funded. Funding Decision Based on the ranking list established by the SEC, the ethical evaluation and available funding, the CSC decides on the list of projects recommended for funding. Each CSC member organisation makes the final decision on its funding contribution according to its respective regulations, calendar and legal frameworks. If necessary, the CSC will determine a priority order for funding proposals which have been awarded the same score within a ranked list. Prioritization will be based on (in descending order): Availability of national/regional funding; Maximization of use of national/regional funding; Proposals with participation of underrepresented or undersubscribed countries; Proposals that address diseases not otherwise covered by more highly ranked proposals. The JCS will notify all project coordinators of the final funding decision and disseminate the SEC consensus report. Partnership: Regarding the eligibility criteria on the consortium composition, in addition to Annex 5 of the Horizon Europe rules on the cascade funding calls (the projects supported must be transnational, involving at least two independent legal entities from two different EU Member States or Horizon Europe associated countries as recipients of the financial support), it is specifically required that each consortium submitting a proposal must involve four to six eligible principal investigator partners (referred to as partners below) from at least four different participating countries (see list in section 2). One of these four to six partners must be an Early Career Researcher (ECR; see section 5.6). In specific cases this number of consortium partners can be increased to eight partners (see below). No more than two eligible partners from the same country can be present in each consortium; further national/regional limits may apply, see “Guidelines for Applicants”. Patient advocacy organisations (PAOs) requesting funding do not count toward the total.

Further information

There are at least 7000 distinct rare diseases, the great majority being of genetic origin. Although individually rare, taken together rare diseases affect at least 26-30 million people in Europe. Moreover, they represent a major issue in health care: many of these diseases have an early or very early onset and/or lead to a significant decrease in life expectancy. Moreover, most of them cause chronic illnesses with a large impact on quality of life and the health care system. Research on rare diseases is needed to provide knowledge for prevention, diagnosis, better care and everyday life improvement for patients. Yet, research is hampered by lack of resources at several levels: (1) few scientists work on any given disease, (2) there are few patients per disease and they are scattered over large geographic areas, causing difficulties to assemble the necessary cohorts, (3) existing databases and bio-material collections are usually local, small, and not accessible or standardized, (4) the complex clinical phenotypes of these diseases require interdisciplinary cooperation to improve research and treatment outcomes. The specificities of rare diseases - limited number of patients per disease, scarcity of relevant knowledge and expertise, and fragmentation of research - single them out as a distinctive domain with very high potential for an added-value through a European collaboration. Rare diseases are a prime example of a research area that necessitates collaboration/coordination on a transnational scale. In this context, the European Rare Diseases Research Alliance (ERDERA) 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). These actions follow the five Joint Transnational Calls for rare diseases research projects launched previously by the European Joint Programme on Rare Diseases (EJP RD) since 2019. For a complete set of information, including the full call (incl. annexes and templates), please refer to the ERDERA website ERDERA An information webinar will be held on December 17th, 2024, 14.00-16.00 (CET). You may register to participate in the webinar here: ERDERA JTC 2025 Information Webinar Registration Contact Joint Call Secretariat DLR Projektträger (DLR-PT, Germany) Dr. Katarzyna Saedler Dr. Michaela Fersch Dr. Ralph Schuster Tel: +49228-38212453 E-Mail: SelteneErkrankungen@dlr.de