Clinical trials for advancing innovative interventions for neurodegenerative diseases

The Horizon Europe opportunity, designated as HORIZON-HLTH, invites proposals focused on advancing innovative interventions for neurodegenerative diseases through early-stage clinical trials. The funding amounts are set at €10 million per grant, with a total budget of €39.3 million for approximately four expected grants. The call encourages participation from diverse organizations, including start-ups, SMEs, universities, and other legal entities from EU Member States, Associated Countries, and the United States. The application process comprises two stages. The first stage requires a brief proposal evaluated based on scientific excellence and impact, while the second stage invites selected applicants to submit comprehensive proposals assessed on additional criteria, including implementation. Deadlines for Stage 1 and Stage 2 submissions are April 13, 2027, and September 22, 2027, respectively. The focus is specifically on neurodegenerative diseases, excluding rare conditions, emphasizing clinical trials for pharmacological-based interventions and innovative combinations with lifestyle modifications and cognitive training. Projects should leverage advanced technologies, such as digital health tools and AI, ensuring adherence to FAIR data principles and stakeholder engagement. Success rates are competitive, reflecting the high demand and limited funding. The call emphasizes a multidisciplinary approach, collaboration across different sectors, and the importance of social sciences in addressing the societal impacts of neurodegenerative diseases. Participants are required to ensure diverse representation in clinical studies and engage with relevant health authorities and stakeholders throughout the research process.

This is a Horizon Europe call for proposals, specifically targeting clinical trials for advancing innovative interventions for neurodegenerative diseases. The call falls under the "Cluster 1 - Health" of the Horizon Europe program and is structured as a two-stage submission process. The overarching goal is to support activities that contribute to tackling diseases and reducing the overall disease burden, with a particular focus on neurodegenerative conditions.

The expected outcomes of funded projects are multi-faceted:

1. Enhanced utilization of state-of-the-art knowledge, data, technologies, tools, methods, best practices, and trainings by the scientific and clinical communities to develop more effective treatments for neurodegenerative diseases.

2. Improved collaboration and exchange of data, knowledge, and best practices among scientific and clinical communities within the EU, associated countries, and beyond.

3. Increased adoption of FAIR data principles through the wide use and integration of relevant databases and infrastructures for data storage and sharing.

4. Better informed policymakers, funders, scientific and clinical communities, patient organizations, regulators, and other relevant bodies regarding research advances and the requirements for widespread implementation of innovative therapeutic interventions and complementary approaches.

5. Constructive engagement of patients and caregivers in the research process, ensuring their needs are addressed and that they tangibly benefit from the interventions.

The scope of the call focuses on neurodegenerative diseases, recognizing their significant burden on patients, caregivers, health systems, and society. The call acknowledges the limitations of current therapeutic solutions, which often address symptoms rather than underlying causes and can have serious side effects. Given the increasing prevalence of these diseases in an aging population, the call emphasizes the need for more innovative, safer, and more effective therapeutic solutions. A key aspect is the combination of active substance-based therapies with multidisciplinary approaches such as lifestyle changes, cognitive training, and rehabilitation therapies to improve the quality of life and reduce the societal impact of these diseases. It is important to note that rare neurodegenerative diseases are explicitly excluded from this call.

Proposals submitted under this call should address most of the following aspects:

1. Conducting rigorous early-stage (phase 1 and/or phase 2) clinical trials to assess the safety and efficacy of innovative interventions, ensuring adequate cohort sizes and representation of the patient population in terms of age, sex, and ethnicity.

2. Gaining further insight into the mechanisms of action of innovative therapies and complementary approaches through clinical trials and additional studies. This includes analyses of imaging data (MRI, ultrasound, nuclear imaging) and physiological, molecular, biochemical, or omics signatures to identify potential perturbations and recovery patterns. The aim is to develop surrogate endpoints and pave the way for more personalized interventions.

3. Utilizing and/or developing technologies, including digital technologies like generative AI and wearable technologies, to implement and monitor the long-term efficacy of interventions, manage disorders, and monitor disease progression. These technologies should be bias-free, inclusive, and ethically sound, with a focus on unobtrusive technologies for patient monitoring at home and in real-world conditions.

4. Leveraging existing data, biobanks, registries, and cohorts, while also generating new data that adheres to FAIR principles.

5. Engaging all relevant stakeholders, including patients, caregivers, clinicians, counselors, and regulators, to design end-user optimized interventions that apply gender-sensitive and intersectional approaches.

6. Advancing research by utilizing existing and emerging state-of-the-art research infrastructures such as EuroBioImaging, European Genomic Data Infrastructure, ECRIN, EATRIS, EBRAINS, and BBMRI, as well as results from EU-supported research projects.

7. Engaging with national public health authorities and regulators to ensure a robust development pathway and further uptake of the intervention.

The call encourages the participation of start-ups, micro, small, and medium-sized enterprises (SMEs) to strengthen their scientific and technological foundations, enhance their innovation potential, and explore commercial exploitation opportunities. Funded projects are expected to liaise with the future co-funded European Partnership for Brain Health. The call also emphasizes the importance of social sciences and humanities (SSH) disciplines and the involvement of SSH experts to enhance the societal impact of the research activities. Projects are strongly encouraged to participate in networking and joint activities, with a budget allocated for attendance at regular joint meetings and potential joint activities. Applicants are required to provide details of their clinical studies in a dedicated annex using a provided template.

The admissibility conditions, proposal page limits, layout, eligible countries, and other eligibility conditions are described in the General Annexes of the Horizon Europe Work Programme and the Horizon Europe Programme Guide. Notably, any legal entity established in the United States of America is eligible to receive Union funding. The financial and operational capacity and exclusion criteria are also detailed in the General Annexes. First-stage proposals will be evaluated blindly based on award criteria, scoring, and thresholds described in the General Annexes. The submission and evaluation processes are outlined in the Work Programme General Annexes and the Online Manual. The indicative timeline for evaluation and grant agreement is also described in the General Annexes. The granting authority retains the right to object to a transfer of ownership or exclusive licensing of results for up to 4 years after the end of the action. Legal and financial aspects of the grants are described in the General Annexes, with specific conditions detailed in the specific topic of the Work Programme.

Application and evaluation form templates, guidance documents, and call-specific instructions are available in the Submission System and through links provided in the call documentation. These include the HE Programme Guide, Model Grant Agreement (MGA), information on clinical studies, and additional documents related to the Horizon Europe program and EU financial regulations.

The call provides a budget overview for several topics in 2027, all structured as two-stage HORIZON Research and Innovation Actions (HORIZON-RIA) or Innovation Actions (HORIZON-IA):

1. HORIZON-HLTH-2027-02-DISEASE-01-two-stage:With a budget of €63,800,000, contributions ranging from €7,000,000 to €8,000,000, and an indicative number of 8 grants.

2. HORIZON-HLTH:With a budget of €39,300,000, contributions around €10,000,000, and an indicative number of 4 grants.

3. HORIZON-HLTH-2027-02-IND-02-two-stage:With a budget of €39,300,000, contributions ranging from €5,000,000 to €7,000,000, and an indicative number of 6 grants.

4. HORIZON-HLTH-2027-02-TOOL-01-two-stage:With a budget of €44,200,000, contributions ranging from €6,000,000 to €8,000,000, and an indicative number of 6 grants.

The call encourages partner searches and provides links to partner search announcements. The submission system is planned to open on February 10, 2027, with deadlines for the first stage on April 13, 2027, and for the second stage on September 22, 2027.

In summary, this Horizon Europe call is a significant funding opportunity for research and innovation projects focused on developing and testing innovative interventions for neurodegenerative diseases. It emphasizes a multidisciplinary approach, the integration of advanced technologies, the engagement of stakeholders, and adherence to FAIR data principles. The call aims to improve the lives of patients and caregivers affected by these debilitating conditions and reduce the societal burden of neurodegenerative diseases.

Eligible Applicant Types:The eligible applicant types include start-ups, micro, small and medium-sized enterprises (SMEs), and any legal entity established in the United States of America. The participation of social sciences and humanities (SSH) experts and institutions is also required. The call is open to a wide range of stakeholders, including patients and patients’ representatives, caregivers, clinicians, counsellors, regulators, and other relevant bodies.

Funding Type:The funding type is a grant, specifically a HORIZON Research and Innovation Action (HORIZON-RIA) and HORIZON Innovation Actions (HORIZON-IA) under the Horizon Europe program. The grants are budget-based.

Consortium Requirement:While not explicitly stated, the encouragement of networking and joint activities, along with the need for multidisciplinary approaches and engagement with various stakeholders, suggests that a consortium is preferred or even necessary for a successful application.

Beneficiary Scope (Geographic Eligibility):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. Any legal entity established in the United States of America is eligible to receive Union funding.

Target Sector:The target sector is health, specifically focusing on neurodegenerative diseases. The program also targets the use of various technologies, including digital technologies like generative AI and wearable technologies, as well as state-of-the-art research infrastructures such as EuroBioImaging, European Genomic Data Infrastructure, ECRIN, EATRIS, EBRAINS, and BBMRI.

Mentioned Countries:United States of America, EU, Associated Countries.

Project Stage:The expected maturity of the project is early-stage clinical trials, specifically phase 1 and/or phase 2 clinical trials for pharmacological-based interventions. The call also supports activities that underpin and complement the development of innovative interventions.

Funding Amount:The funding amounts vary depending on the specific topic.

HORIZON-HLTH-2027-02-DISEASE-01-two-stage:€7,000,000 to €8,000,000

HORIZON-HLTH:around €10,000,000

HORIZON-HLTH-2027-02-IND-02-two-stage:€5,000,000 to €7,000,000

HORIZON-HLTH-2027-02-TOOL-01-two-stage:€6,000,000 to €8,000,000

Application Type:The application type is a two-stage open call.

Nature of Support:Beneficiaries will receive money in the form of a grant to support their research and innovation activities.

Application Stages:The application process involves two stages. In the first stage, proposals are evaluated blindly based on Excellence and Impact. In the second stage, proposals are evaluated based on Excellence, Impact, and Implementation.

Success Rates:The success rates are not explicitly mentioned, but the evaluation process indicates that the total requested budget of proposals admitted to stage 2 should be as close as possible to four times the available budget, and not less than three and a half times the available budget. This suggests that only a fraction of the initial proposals will make it to the second stage.

Co-funding Requirement:The information does not explicitly state whether co-funding is required.

Summary:This Horizon Europe call focuses on advancing innovative interventions for neurodegenerative diseases through clinical trials and related research activities. It aims to improve the quality of life for patients and caregivers while reducing the societal burden of these diseases. The call encourages the participation of SMEs, the use of advanced technologies and data management practices, and the engagement of various stakeholders, including patients, clinicians, and policymakers. The application process is a two-stage process, and funding is provided in the form of grants. The call is open to entities from eligible countries, including the United States. The projects should focus on early-stage clinical trials, mechanism of action studies, technology development for monitoring and management, data utilization following FAIR principles, stakeholder engagement, and leveraging existing research infrastructures. The call also emphasizes the importance of social sciences and humanities in enhancing the societal impact of the research.