Clinical trials for advancing innovative interventions for neurodegenerative diseases

The scientific and clinical communities benefit from the exchange of data, knowledge and best practices, thereby strengthening their collaboration in the EU, the Associated Countries and beyond. The scientific and clinical communities make wide use of relevant databases and/or integrate them with existing infrastructures for storage and sharing of collected data according to FAIR [1] principles, thereby encouraging further use of the data. Policymakers, funders, scientific and clinical communities, patient organisations, regulators, and other relevant bodies are informed of the research advances made and the requirements for a widespread implementation of the innovative therapeutic interventions and complementary approaches. Patients and caregivers are constructively engaged with the research, ensuring that their needs are catered for, with the aim of tangibly benefitting from the interventions. Scope: Neurodegenerative diseases are a high burden for patients, caregivers, health systems and society. Given the limitations with current therapeutic solutions, including that they primarily address symptoms rather than underlying causes and can have serious side effects, together with the increasing prevalence of neurodegenerative diseases in an aging population, there is a huge need to develop more innovative, safer and more effective therapeutic solutions for these diseases. To further enhance their safety and effectiveness, the therapeutic solution based on an active substance should be combined/complemented with another multidisciplinary approach (e.g. lifestyle changes, cognitive training, rehabilitation therapies). Together this innovative intervention should lead to an improved quality of life and reduce the societal impact of these diseases. Rare neurodegenerative diseases are excluded [2] . Proposals should address most of the following aspects: Perform rigorous early-stage [3] clinical trials into the safety and efficacy of the innovative interventions and their mode of administration, ensuring adequate cohorts/sample sizes with adequate representation of the patient population, including in terms of age, sex and ethnicity. Through the clinical trials and to the extent possible of additional studies, gain further insight into the potentially novel mechanism(s) of action of the innovative therapies and complementary approaches. This could entail analyses of imaging (e.g. MRI, ultrasound, nuclear imaging), as well as physiological, molecular, biochemical or omics signatures revealing potential perturbations prior to the intervention and recovery/improvement thereafter, and it could lead to the development of surrogate endpoints. This insight should open the path to more personalised interventions and approaches. Use and/or develop technologies, including digital ones (e.g. (generative) Artificial Intelligence - AI [4] , wearable technologies) to help implement and monitor the long-term efficacy of the intervention(s), as well as manage the disorder and/or monitor their progression (e.g. with unobtrusive technologies suitable for patient monitoring at home and in real-world conditions), whilst also ensuring they are bias-free, inclusive, and ethically sound. Utilise existing data, biobanks, registries and/or cohorts, together with the generation of new data that should be managed in line with the FAIR principles. Engage all relevant stakeholders (especially patients and patients’ representatives for the disease, caregivers, clinicians, counsellors, regulators, etc.) to design end-user optimised interventions, applying gender-sensitive and intersectional approaches. Advance research by leveraging already existing and emerging state-of-the-art research infrastructures (e.g. EuroBioImaging [5] , European Genomic Data Infrastructure [6] , ECRIN [7] , EATRIS [8] , EBRAINS [9] , BBMRI [10] , etc.), as well as results stemming from EU-supported research projects, where applicable [11] . Engage with national public health authorities and regulators to ensure a robust development pathway and further uptake of the intervention. The participation of start-ups, micro, small and medium-sized enterprises (SMEs) [12] is encouraged with the aim of strengthening their scientific and technological foundations, enhancing their innovation potential, and exploring possibilities for commercial exploitation. Funded projects should liaise with the co-funded European Partnership for Brain Health [13] , covered by topic HORIZON-HLTH-2025-02-DISEASE-01: “European Partnership for Brain Health”. The topic requires the effective contribution of social sciences and humanities (SSH) disciplines and the involvement of SSH experts, institutions as well as the inclusion of relevant SSH expertise, in order to produce meaningful and significant effects enhancing the societal impact of the related research activities. All projects funded under this topic are strongly encouraged to participate in networking and joint activities, as appropriate. Therefore, proposals should include a budget for the attendance to regular joint meetings and may consider covering the costs of any other potential joint activities without the prerequisite to detail concrete joint activities at this stage. The details of these joint activities will be defined during the grant agreement preparation phase. Applicants should provide details of their clinical studies [14] in the dedicated annex using the template provided in the submission system. As proposals under this topic are expected to include clinical studies, the use of the template is strongly encouraged. [1] See definition of FAIR data in the introduction to this Work Programme part. [2] Rare diseases, as defined by the European Union Regulation on Orphan Medicinal Products (1999), being a disease that affects not more than 1 person per 2000 in the European population ( https://www.orpha.net/ ). [3] For pharmacological-based interventions: phase 1 and/or phase 2 clinical trials. [4] Generative AI is a type of AI technology that can generate various forms of new content such as text, images, sounds, and even code, such as for programming or gene sequencing ( https://ec.europa.eu/newsroom/dae/redirection/document/101621 ). [5] https://www.eurobioimaging.eu [6] https://gdi.onemilliongenomes.eu [7] https://ecrin.org [8] https://eatris.eu [9] https://www.ebrains.eu [10] https://www.bbmri-eric.eu [11] Consult databases e.g. CORDIS ( https://cordis.europa.eu ) & the JPND Research Database ( https://neurodegenerationresearch.eu/search-our-database ). [12] https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32003H0361 [13] https://cordis.europa.eu/project/id/101259512 , https://www.brainhealth-partnership.eu [14] Please note that the definition of clinical studies (see introduction to this Work Programme part) is broad and it is recommended that you review it thoroughly before submitting your application.

Eligibility and submission conditions

"> General conditions 1. Admissibility Conditions, proposal page limit and layout Applicants submitting a proposal for a blind evaluation (see General Annex F) must not disclose their organisation names, acronyms, logos nor names of personnel in the proposal abstract and Part B of their first-stage application (see General Annex E). Admissibility conditions are described in Annex A and Annex E of the Horizon Europe Work Programme General Annexes. Proposal page limits and layout are described in Part B of the Application Form available in the Submission System. 2. Eligible Countries Eligible countries are described in Annex B of the Work Programme General Annexes. A number of non-EU/non-Associated Countries that are not automatically eligible for funding have made specific provisions for making funding available for their participants in Horizon Europe projects. See the information in the Horizon Europe Programme Guide . 3. Other Eligibility Conditions In recognition of the opening of the US National Institutes of Health’s programmes to European researchers, any legal entity established in the United States of America is eligible to receive Union funding. If projects use satellite-based earth observation, positioning, navigation and/or related timing data and services, beneficiaries must make use of Copernicus and/or Galileo/EGNOS (other data and services may additionally be used). Subject to restrictions for the protection of European communication networks. Other eligibility conditions are described in Annex B of the Work Programme General Annexes. 4. Financial and operational capacity and exclusion Financial and operational capacity and exclusion are described in Annex C of the Work Programme General Annexes. 5a. Evaluation and award: Award criteria, scoring and thresholds The first-stage proposals of this topic will be evaluated blindly. Award criteria, scoring and thresholds are described in Annex D of the Work Programme General Annexes. 5b. Evaluation and award: Submission and evaluation processes For the first stage, the thresholds for each criterion will be 4 (Excellence) and 4 (Impact). The overall threshold applying to the sum of the two individual scores will be set at a level that ensures the total requested budget of proposals admitted to stage 2 is as close as possible to four times the available budget, and not less than three and a half times the available budget. For the second stage, the thresholds for each criterion will be 4 (Excellence), 4 (Impact) and 4 (Implementation). The cumulative threshold will be 12. Submission and evaluation processes are described in Annex F of the Work Programme General Annexes and the Online Manual . 5c. Evaluation and award: Indicative timeline for evaluation and grant agreement Indicative timeline for evaluation and grant agreement are described in Annex F of the Work Programme General Annexes. 6. Legal and financial set-up of the grants The granting authority may, up to 4 years after the end of the action, object to a transfer of ownership or to the exclusive licensing of results, as set out in the specific provision of Annex 5. Legal and financial set-up of the grants are described in Annex G of the Work Programme General Annexes. Specific conditions Specific conditions are described in the specific topic of the Work Programme. Application and evaluation forms and additional documents: Application and evaluation form templates Standard application form (HE RIA IA Stage 1 BLIND) - the application form specific to this call is available in the Submission System Standard application form (HE RIA, IA) - the application form specific to this call is available in the Submission System Standard evaluation form (HE RIA, IA and CSA Stage 1 BLIND) - will be used with the necessary adaptations Standard evaluation form (HE RIA, IA) - will be used with the necessary adaptations Guidance HE Programme Guide Model Grant Agreement (MGA) HE MGA Call-specific instructions Information on clinical studies (HE) Additional documents HE Main Work Programme 2026-2027 – 1. General Introduction HE Main Work Programme 2026-2027 – 4. Health HE Main Work Programme 2026-2027 – 15. General Annexes HE Programme Guide HE Framework Programme 2021/695 HE Specific Programme Decision 2021/764 EU Financial Regulation 2024/2509 Decision authorising the use of lump sum contributions under the Horizon Europe Programme Rules for Legal Entity Validation, LEAR Appointment and Financial Capacity Assessment EU Grants AGA — Annotated Model Grant Agreement Funding & Tenders Portal Online Manual Funding & Tenders Portal Terms and Conditions Funding & Tenders Portal Privacy Statement

Hivatkozott dokumentumok

Annex A Horizon Europe Programme Guide Online Manual Standard application form (HE RIA IA Stage 1 BLIND) Standard application form (HE RIA, IA) Standard evaluation form (HE RIA, IA and CSA Stage 1 BLIND) Standard evaluation form (HE RIA, IA) HE MGA Information on clinical studies (HE) HE Main Work Programme 2026-2027 – 1. General Introduction HE Main Work Programme 2026-2027 – 4. Health Decision authorising the use of lump sum contributions under the Horizon Europe Programme Rules for Legal Entity Validation, LEAR Appointment and Financial Capacity Assessment EU Grants AGA — Annotated Model Grant Agreement Funding & Tenders Portal Terms and Conditions