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

The European Rare Diseases Research Alliance (ERDERA) has announced the Joint Transnational Call (JTC) 2025 aimed at supporting pre-clinical therapy studies for rare diseases, specifically utilizing small molecules and biologicals for development and validation. This initiative intends to coordinate research efforts across Europe and aligns with the objectives of the International Rare Disease Research Consortium (IRDiRC).

The call will open on December 10, 2024, featuring multiple cut-off deadlines, with the pre-proposal submission deadline set for February 13, 2025, and full proposal submissions due by July 9, 2025. The total funding allocated for this call is €32,625,162. Individual project funding can vary but typically falls within a range determined by national and regional budget caps.

Eligible applicants include research consortia comprising four to six principal investigators from at least four different participating countries, though the number can increase to eight under specific circumstances. One partner must be an Early Career Researcher (ECR). Patient advocacy organizations may participate but will not count towards the investigator total. Each proposal must demonstrate transnational collaboration among partners from different EU Member States or Horizon Europe-associated countries.

The application process involves a two-stage submission, beginning with a pre-proposal that outlines the scientific idea and supporting data. Only those invited after preliminary review can proceed to submit a full proposal, which must contain comprehensive details including patient engagement plans, data management strategies, ethical considerations, and implementation quality.

Evaluation criteria will focus on excellence, potential impact, and implementation quality. Proposals will be assessed for their clarity, innovative approaches, collaboration effectiveness, and the feasibility of implementation. The scoring system ranks proposals on a scale from zero to five, and proposals must meet specific threshold scores to advance.

An information webinar will provide additional details to potential applicants on December 17, 2024. The Joint Call Secretariat, located in Germany, will oversee the process, and detailed guidance will be made available on the ERDERA website.

In summary, this call presents a significant opportunity for collaborative research initiatives targeting the challenges of rare diseases, emphasizing the necessity for innovative therapeutic development and interdisciplinary cooperation across Europe.

The European Rare Diseases Research Alliance (ERDERA) is launching a Joint Transnational Call (JTC) 2025 for "Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation". The call aims to coordinate research efforts in the field of rare diseases and implement the objectives of the International Rare Disease Research Consortium (IRDiRC). This call follows the five Joint Transnational Calls for rare diseases research projects launched previously by the European Joint Programme on Rare Diseases (EJP RD) since 2019.

The opening date for the call is December 10th, 2024. There are multiple cut-off deadlines. The pre-proposal submission deadline is February 13th, 2025, at 17:00 (Brussels time), and the full proposal submission deadline is July 9th, 2025, at 17:00 (Brussels time). The expected duration of participation is a maximum of three years. The total funding available is 32,625,162.00 €. The project acronym is ERDERA, and the full name of the EU-funded project is the EUROPEAN RARE DISEASES RESEARCH ALLIANCE. The grant agreement number is 101156595. The topic is HORIZON-HLTH-2023-DISEASE-07-01 - European Partnership on Rare Diseases.

The submission process involves a two-stage procedure: a pre-proposal stage and a full proposal stage. Research consortia intending to submit a transnational project proposal should register as soon as possible via the electronic proposal system. One joint proposal document (in English) must be prepared by the partners and submitted by the coordinator to the Joint Call Secretariat (JCS) via the electronic submission system. Proposals must be prepared using the templates provided in the electronic system; non-conforming proposals will be rejected.

Key dates include:
December 10th, 2024: Launch of the call
December 17th, 2024: Information webinar for potential applicants
February 13th, 2025: Pre-proposal submission deadline
March 3rd, 2025: Pre-proposal eligibility check
Early May 2025: Invitation to full proposal
May 6th, 2025: Information webinar for applicants invited to submit a full proposal
July 9th, 2025: Full proposal submission deadline
July 18th, 2025: Full proposal eligibility check
December 2025: Notification of funding decision

Full proposals will only be accepted from applicants explicitly invited by the JCS. Fundamental changes between the pre- and full proposals regarding consortium composition, project objectives, or requested budget require detailed justification to the JCS for consideration by the Call Steering Committee. National/regional regulations on eligible partners and budget caps still apply, and budget changes need pre-approval from relevant national/regional funding organizations.

At the pre-proposal stage, applicants should focus on presenting the scientific idea/hypothesis and supporting preliminary results, studies, or data, describing the project from an unmet need to the expected endpoint. At the full proposal stage, 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.

Evaluation scores will be awarded according to Horizon Europe rules, using a common evaluation form. The scoring system is as follows:
0: Failure
1: Poor
2: Fair
3: Good
4: Very good
5: Excellent

The evaluation criteria are:
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)
f. Active and meaningful participation of Patients Organisations (PAOs) and patient partners in the project

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
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
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 at both the pre- and full proposal stages. Sub-criteria 1f, 2d, 3c, and 3d will only be assessed as part of the full proposal evaluation step.

The JCS will check all pre-proposals for formal criteria and compliance with country/regional eligibility rules. Pre-proposals that pass the eligibility check will be forwarded to the Scientific Evaluation Committee (SEC) members with biomedical expertise for evaluation. 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. Applicants will receive their individual consensus review report and corresponding SEC recommendations regarding full proposal submission.

Each criterion will be scored out of five. The weighting factor for the Excellence criterion will be two. 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 for each individual evaluation criterion and a minimum overall score of 15 points per expert vote.

Research teams from underrepresented or undersubscribed countries may join successful pre-proposals after approval by the relevant funding organizations. Applicants invited to submit a full proposal are strongly encouraged to use the ERDERA support services for FAIR data, translational mentoring, and regulatory advice.

The JCS will check the full proposals for formal criteria with the help of the CSC. The JCS will send full proposals to the SEC members, who will meet to discuss the proposals, assign their final scores, and create a ranking list with proposals recommended for funding. The final summary review report for each consortium will be prepared by the SEC members and sent to respective applicants.

Each full 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, with the Excellence criterion weighted double. 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. The patient reviewer will score according to subcriteria 1f, 2b, and 3b, with a maximum score of 15 points and a threshold of 10 points. The overall maximum score that can be given to a consortium is 75 points, and the minimum threshold to be considered for funding will be 55 points.

Full proposals recommended for funding by the SEC will be remotely evaluated by independent experts in ethics. Only proposals approved by both the scientific and ethical evaluations will be funded.

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 organization makes the final decision on its funding contribution. Prioritization will be based on the availability of national/regional funding, maximization of use of national/regional funding, proposals with participation of underrepresented or undersubscribed countries, and 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.

Regarding consortium composition, each consortium must involve four to six eligible principal investigator partners from at least four different participating countries. One of these partners must be an Early Career Researcher (ECR). In specific cases, the number of consortium partners can be increased to eight. No more than two eligible partners from the same country can be present in each consortium. Patient advocacy organizations (PAOs) requesting funding do not count toward the total.

There are at least 7000 distinct rare diseases, the great majority being of genetic origin, affecting at least 26-30 million people in Europe. Research on rare diseases is needed to provide knowledge for prevention, diagnosis, better care, and everyday life improvement for patients.

An information webinar will be held on December 17th, 2024, from 14:00 to 16:00 (CET).

The Joint Call Secretariat can be contacted at DLR Projektträger (DLR-PT, Germany) with Dr. Katarzyna Saedler, Dr. Michaela Fersch, and Dr. Ralph Schuster. The telephone number is +49228-38212453, and the email address is SelteneErkrankungen@dlr.de.

In summary, this ERDERA Joint Transnational Call 2025 is a funding opportunity focused on pre-clinical therapy studies for rare diseases, specifically using small molecules and biologicals for development and validation. It aims to foster transnational research collaborations to address the challenges in rare disease research, such as limited resources, scattered patient populations, and the need for interdisciplinary cooperation. The call involves a two-stage application process, with pre-proposals and full proposals, and emphasizes the importance of patient involvement, data management, and ethical considerations. The evaluation process includes scientific and ethical reviews, with specific criteria for excellence, impact, and quality of implementation. Successful projects will contribute to the development of therapies for rare diseases and improve the lives of patients and their families.

Eligible Applicant Types: The eligible applicant types are research consortia. The consortia must involve four to six eligible principal investigator partners from at least four different participating countries. One of these partners must be an Early Career Researcher (ECR). In specific cases, the number of consortium partners can be increased to eight. Patient advocacy organizations (PAOs) requesting funding do not count toward the total. The legal entities must be independent and from different EU Member States or Horizon Europe associated countries.

Funding Type: The funding type is a grant, specifically cascade funding.

Consortium Requirement: A consortium is required. Each consortium must involve four to six eligible principal investigator partners from at least four different participating countries. In specific cases, this number can be increased to eight partners.

Beneficiary Scope (Geographic Eligibility): The geographic eligibility includes EU Member States and Horizon Europe associated countries. The projects supported must be transnational, involving at least two independent legal entities from two different EU Member States or Horizon Europe associated countries.

Target Sector: The target sector is health, specifically research on rare diseases. The call focuses on pre-clinical therapy studies for rare diseases using small molecules and biologicals, including development and validation.

Mentioned Countries: Germany is explicitly mentioned as the location of the Joint Call Secretariat (DLR Projektträger, DLR-PT, Germany). The eligibility criteria also mention EU Member States and Horizon Europe associated countries.

Project Stage: The project stage is pre-clinical development and validation. The call specifically targets "Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation".

Funding Amount: The total funding available is 32,625,162.00 €. The funding range for individual projects is variable and depends on national/regional regulations and budget caps.

Application Type: The application type is a two-stage open call, involving a pre-proposal and a full proposal stage. Full proposals are only accepted from applicants explicitly invited by the JCS.

Nature of Support: Beneficiaries will receive money in the form of grants. ERDERA also provides support services for FAIR data, translational mentoring, and regulatory advice.

Application Stages: There are two application stages: a pre-proposal stage and a full proposal stage. Only applicants invited after the pre-proposal stage can submit a full proposal.

Success Rates: The success rates are not explicitly mentioned, but the process involves multiple evaluation steps, including eligibility checks, peer review by scientific experts, and ethical evaluation, suggesting a competitive process.

Co-funding Requirement: Co-funding requirements are not explicitly stated, but national/regional regulations and budget caps apply, implying that some level of co-funding or adherence to specific funding rules at the national/regional level may be necessary.

The European Rare Diseases Research Alliance (ERDERA) Joint Transnational Call (JTC) 2025 is a funding opportunity focused on supporting pre-clinical therapy studies for rare diseases. The call aims to facilitate transnational research projects that develop and validate therapies using small molecules and biologicals. Research consortia composed of 4 to 6 principal investigators from at least four different participating countries (with possible extension to 8 in specific cases), including an Early Career Researcher, are eligible to apply. Patient advocacy organizations can also be part of the consortium but do not count towards the total number of principal investigators. The call follows a two-stage submission process: a pre-proposal and a full proposal. The pre-proposal focuses on the scientific idea and preliminary data, while the full proposal requires a comprehensive plan including patient engagement, data management, statistical methods, and ethical considerations. Proposals are evaluated based on excellence, impact, and quality/efficiency of implementation, aligning with Horizon Europe rules. The total funding available is over 32 million euros. The call emphasizes transnational collaboration to address the challenges in rare disease research, such as the limited number of patients and the fragmentation of expertise. The ERDERA initiative aims to coordinate research efforts across Europe and associated countries, supporting the objectives of the International Rare Disease Research Consortium (IRDiRC). The Joint Call Secretariat (JCS) manages the call, and further information, including guidelines and templates, is available on the ERDERA website. An information webinar is scheduled to provide potential applicants with more details. The funding decision is based on a ranking list established by a Scientific Evaluation Committee (SEC), ethical evaluations, and the availability of funding, with final decisions made by each participating country's funding organization.