Closed

European Partnership on Rare Diseases

HORIZON Programme Cofund Actions

Basic Information

Identifier
HORIZON-HLTH-2023-DISEASE-07-01
Programme
Partnerships in Health (2023)
Programme Period
2021 - 2027
Status
Closed (31094503)
Opening Date
January 12, 2023
Deadline
September 19, 2023
Deadline Model
single-stage
Budget
€50,000,000
Min Grant Amount
€50,000,000
Max Grant Amount
€50,000,000
Expected Number of Grants
1
Keywords
HORIZON-HLTH-2023-DISEASE-07-01HORIZON-HLTH-2023-DISEASE-07AnaesthesiologyAnatomy and morphologyAndrologyBiobanksCardiac and Cardiovascular systemsClinical managementClinical medicineClinical neurologyClinical researchClinical trialsCritical care medicine and Emergency medicineDentistry, oral surgery and medicineDermatology and venereal diseasesEndocrinology and metabolism (including diabetes, hormones)EpidemiologyEthics in medical sciencesGastroenterology and hepatologyGeneral and internal medicineGeriatrics and gerontologyHealth care sciences and services (including hospital administration, health care financing)Health policy and servicesHealth sciencesHealth services, health care researchHealthcare systemHematologyHuman geneticsImmunologyMedical ethicsMedical statisticsNeuro-ethicsNeurosciences (including psychophysiology)Non-communicable diseases (except for neural/psychiatric and immunity-related)Obstetrics and gynaecologyOncologyOphthalmologyOrthopaedicsOther clinical medicine subjectsOtorhinolaryngologyPaediatricsPathologyPeripheral vascular diseasePersonalised medicinePharmacoeconomicsPharmacology and pharmacyPhysiology (including cytology)Privacy policiesPublic health policiesRare diseasesResearch integrity and research misconductRespiratory systemsSurgeryTransplantationUrology and nephrology

Description

Expected Outcome:

This topic aims at supporting activities that are enabling or contributing to several expected impacts of destination 3 “Tackling diseases and reducing disease burden”. To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to all of the following expected outcomes:

  • The EU is reinforced as an internationally recognised driver of research and innovation in rare diseases (RD) and thereby substantially contributing to the achievement of the Sustainable Development Goals related to rare diseases;
  • Research funders align, adopt and implement their RD research policies allowing for the optimal generation and translation of knowledge into meaningful health products and interventions responding to the needs of people living with a rare disease across Europe and globally.
  • The RD research community at large benefit from and use an improved comprehensive knowledge framework integrating the EU, national/regional data and information infrastructures to improve translational research.
  • People living with a rare disease benefit from a more timely, equitable access to innovative, sustainable and high-quality healthcare, taking stock of highly integrated research and healthcare systems.
  • Researchers, innovators - as well as people living with a rare disease and their advocates (as co-creators) - effectively constitute and operate into an integrated research and innovation ecosystem to deliver cost-effective diagnosis and treatments.
  • Public and private actors, including civil society (e.g. NGOs, charities), establish coordinated and efficient multi-stakeholder collaborations at EU and national (including regional) levels, allowing for more effective clinical research, for example aiming at improved success rates of therapeutic development.
Scope:

The Partnership should contribute to priorities of the “Communication on effective, accessible and resilient health systems” (COM(2014) 215 final), the “Communication on enabling the digital transformation of health and care in the Digital Single Market; empowering citizens and building a healthier society” (COM(2018) 233 final) and support the objectives of the new EU4Health Programme (COM(2020) 405 final, Regulation (EU) 2021/522[1]).

This partnership should also contribute to achieving the objectives of the Pharmaceutical Strategy for Europe[2], in terms of fulfilling unmet medical needs (e.g. for rare diseases with so called “orphan medicinal products”[3]) and ensuring that the benefits of innovation reach patients in the EU.

Thanks to its capacity to bring together different stakeholders (e.g. research funders, health authorities, healthcare institutions, innovators, policymakers), the Partnership will create a critical mass of resources and to implement a long-term Strategic Research and Innovation Agenda (SRIA).

The co-funded European Partnership on rare diseases should be implemented based on the priorities identified in the SRIA and through a joint programme of activities ranging from coordinating and funding transnational research to highly integrative and community-driven ‘in-house’ activities such as innovation strategies for the efficient exploitation of research results, EU clinical trial preparedness activities, optimisation of research infrastructures and resources, including networking, training and dissemination activities. It should be structured along the following main objectives:

  • Launch joint transnational calls for RD research and innovation priorities as defined in the SRIA, resulting in financial support to third parties, based on the annual work plans;
  • Develop a European Clinical Research Network to accelerate the clinical trial readiness of the RD research community in Europe, to improve the research and innovation potential of RD stakeholders and facilitate the cost-effective clinical development of new therapies;
  • Develop and consolidate the capacity building of the RD data ecosystem by supporting the federated access/sharing of FAIR[4] research data, information resources to ensure the effective and fast translation of the research results to safe and effective health innovations;
  • Integrate basic, pre-clinical and clinical research to reduce the burden for people living with a rare disease.
  • Support research in relevant medical fields and intervention areas (prevention, diagnosis, treatment), while improving the utilisation of existing health technologies in clinical practice;
  • Support the scientific work of the International Rare Disease Research Consortium.

The Partnership is open to all EU Member States, as well as to countries associated to Horizon Europe and will remain open to third countries wishing to join. The Partnership should include or engage with the following actors:

  • Ministries in charge of R&I policy, as well as national and regional R&I and technology funding agencies and foundations;
  • Ministries in charge of health and care policy, as well as national and regional healthcare authorities, organisations and providers (including providers members of the European Reference Networks);
  • Research infrastructures;
  • Patients organisations;
  • Industry;
  • Charities.

The Partnership may also encourage engagement with other relevant Ministries and research funders. It should involve other key actors from civil society and end-users, research and innovation community, innovation owners, health and care systems owners/organisers and health and care agencies.

The Partnership’s governance structure should enable an upfront strategic steering, effective management and coordination, daily implementation of activities and ensure the use and uptake of the results. Importantly, the EU Member States, as public funders should have a leading role in the governance and strategic steering of the whole Partnership, including in the co-design and the strategic orientations of the ‘in-house’ activities, such as consolidating the research & innovation ecosystem, clinical trial preparedness for the community, contribution to ERA, training activities etc.). Moreover, the management structure should allow the coordinated input of key stakeholders, including but not limited to the research and innovation community, patients and citizens, health and care professionals, formal and informal care organisations, and innovation owners.

To ensure coherence and complementarity of activities and leverage knowledge and investment possibilities, the Partnership is expected to establish relevant collaborations with other Horizon Europe partnerships (institutionalised and co-funded) and missions as set out in the working document on ‘Coherence and Synergies of candidate European Partnerships under Horizon Europe’[5] as well as to explore collaborations with other relevant activities at EU and international level. The proposal should also consider synergies with EU programmes, including but not limited to EU4Health, the Digital Europe Programme (DIGITAL), the European Social Fund Plus (ESF+), the European Regional Development Fund (ERDF)[6], InvestEU, the Recovery and Resilience Facility (RRF) and the Technical Support Instrument (TSI).

Cooperation with international organisations, and non-European institutions and experts may be considered. Participation of third countries is encouraged. Their commitments to the Partnership would not be eligible for the calculation of EU funding. Applicants should describe in their proposal the methodology for their collaboration and the aims they want to achieve with this kind of collaboration.

Proposals should pool the necessary financial resources from the participating national (or regional) research programmes with a view to implementing joint calls for transnational proposals resulting in grants to third parties. Financial support provided by the participants to third parties is one of the activities of this action in order to be able to achieve its objectives.

Collaboration with the EU agency involved in authorising orphan medicinal products, the European Medicines Agency (EMA), should be considered to enhance the sharing of knowledge and data regarding orphan medicinal products and rare diseases, while national agencies producing knowledge on orphan medicinal products and rare diseases may also join the Partnership, e.g. as beneficiaries.

When defining calls for proposals, this Partnership needs to consider 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.

Collaboration with the European Commission's Joint Research Centre (JRC) must be considered to materialise the sharing of (meta)data regarding registries for rare diseases, exchanging data for clinical studies and research based on a unified pseudonymisation tool provided by the European Platform on Rare Disease Registration (EU RD Platform) and related tools and services, as well as in other areas of mutual interest, such as training and capacity building.

The total indicative budget for the partnership is up to EUR 150 million and subject to the effective implementation of the commitments made by the members of the consortium. The Commission envisages to include new actions in its future work programmes to provide continued support to the partnership for the duration of Horizon Europe.

The expected duration of the partnership is seven to ten years.

[1] https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=uriserv:OJ.L_.2021.107.01.0001.01.ENG

[2] COM(2020) 761 final, https://ec.europa.eu/health/medicinal-products/pharmaceutical-strategy-europe_en

[3] https://ec.europa.eu/health/medicinal-products/orphan-medicinal-products_en

[4] See definition of FAIR data in the introduction to this work programme part.

[5] Directorate-General for Research and Innovation, A4 Partnership Sector, October 2020: https://ec.europa.eu/info/sites/default/files/research_and_innovation/funding/documents/ec_rtd_coherence-synergies-of-ep-under-he_annex.pdf

[6] “Synergies between Horizon Europe and ERDF programmes (Draft Commission Notice)” https://research-and-innovation.ec.europa.eu/news/all-research-and-innovation-news/synergies-guidance-out-2022-07-06_en

Destination & Scope

Calls for proposals under this destination are directed towards the Key Strategic Orientation KSO-D ‘Creating a more resilient, inclusive and democratic European society’ of Horizon Europe’s Strategic Plan 2021-2024. Research and innovation supported under this destination should contribute to the impact area ‘Good health and high-quality accessible healthcare’ and in particular to the following expected impact, set out in the Strategic Plan for the health cluster: ‘health care providers are able to better tackle and manage diseases (infectious diseases, including poverty-related and neglected diseases, non-communicable and rare diseases) and reduce the disease burden on patients effectively thanks to better understanding and treatment of diseases, more effective and innovative health technologies, better ability and preparedness to manage epidemic outbreaks and improved patient safety’. In addition, research and innovation supported under this destination could also contribute to the following impact areas: ‘A resilient EU prepared for emerging threats’, ‘Climate change mitigation and adaptation’, and ‘High quality digital services for all’.

Communicable and non-communicable diseases cause the greatest amounts of premature death and disability in the EU and worldwide. They pose a major health, societal and economic threat and burden. Many people are still suffering from these diseases and too often dying prematurely. Non-communicable diseases, including mental illnesses and neurodegenerative diseases, are responsible for up to 80% of EU health care costs[1]. These costs are spent on the treatment of such diseases that to a large extent are preventable. Furthermore, only around 3% of the health care budgets are currently spent on preventive measures although there is a huge potential for prevention. Infectious diseases, including emerging infectious diseases and infections resistant to antimicrobials, remain a major threat to public health in the EU but also to global health security. Deaths caused by antimicrobial resistance (AMR) could exceed 10 million per year worldwide according to some predictions[2].

To further advance, there is an urgent need for research and innovation to develop new preparedness and prevention measures, public health interventions, diagnostics, vaccines, therapies, alternatives to antimicrobials, as well as to improve existing preparedness and prevention strategies to create tangible impacts, taking into account sex/gender-related issues. This will require international cooperation to pool the best expertise and know-how available worldwide, to access world-class research infrastructures and to leverage critical scales of investments on priority needs through a better alignment with other funders of international cooperation in health research and innovation. The continuation of international partnerships and cooperation with international organisations is particularly needed to combat infectious diseases, to address antimicrobial resistances, to respond to major unmet medical needs for global health security, including the global burden of non-communicable diseases, and to strengthen patient safety.

In this work programme, destination 3 will focus on major societal challenges linked to the Commission’s political priorities such as the fight against cancer and other non-communicable diseases, better diagnosis and treatment of rare diseases, preparedness and response to and surveillance of health threats and epidemics, reduction of the number of antimicrobial-resistant infections, improving vaccination rates, demographic change, mental health and digital empowerment in health literacy. In particular, the topics under this destination will support activities aiming at: i) better understanding of diseases, their drivers and consequences, including pain and the causative links between health determinants and diseases, and better evidence-base for policymaking; ii) better methodologies and diagnostics that allow timely and accurate diagnosis, identification of personalised treatment options and assessment of health outcomes, including for patients with a rare disease; iii) development and validation of effective intervention for better surveillance, prevention, detection, treatment and crisis management of infectious disease threats; iv) innovative health technologies developed and tested in clinical practice, including personalised medicine approaches and use of digital tools to optimise clinical workflows; v) new and advanced therapies for non-communicable diseases, including rare diseases developed in particular for those without approved options, supported by strategies to make them affordable for the public payer; and vi) scientific evidence for improved/tailored policies and legal frameworks and to inform major policy initiatives at global level (e.g. WHO Framework Convention on Tobacco Control; UNEA Pollution Implementation Plan).

In view of increasing the impact of EU investments under Horizon Europe, the European Commission welcomes and supports cooperation between EU-funded projects to enable cross-fertilisation and other synergies. This could range from networking to joint activities such as the participation in joint workshops, the exchange of knowledge, the development and adoption of best practices, or joint communication activities. Opportunities for potential synergies exist between projects funded under the same topic but also between other projects funded under another topic, cluster or pillar of Horizon Europe (but also with ongoing projects funded under Horizon 2020). In particular, this could involve projects related to European health research infrastructures (under pillar I of Horizon Europe), the EIC strategic challenges on health and EIT-KIC Health (under pillar III of Horizon Europe), or in areas cutting across the health and other clusters (under pillar II of Horizon Europe). For instance, with cluster 3 “Civil security for society” such as on health security/emergencies (preparedness and response, medical countermeasures, epidemic outbreaks/pandemics, natural disasters and technological incidents, bioterrorism); with cluster 4 “Digital, Industry and Space” such as on decision-support systems or on geo-observation and monitoring (e.g. of disease vectors, epidemics); or with cluster 6 “Food, bioeconomy, natural resources, agriculture and environment” such as on health security and AMR (one-health: human/animal/plant/soil/water health). In addition, while focusing on civilian applications, there may be there may be synergies with actions conducted under the European Defence Fund, notably in the field of defence medical countermeasures.

Based on needs that emerged during the management of COVID-19, some research and innovation actions under Destination 3 should support the mission of the European Health Emergency and Response Authority (HERA) to strengthen Europe’s ability to prevent, detect, and rapidly respond to cross-border health emergencies by ensuring the availability and access to key medical countermeasures. Other actions should deliver relevant complementary inputs to the “Europe’s Beating Cancer Plan”[3] in order to cover the entire cancer care pathway, including prevention, early detection, diagnosis, treatment, cancer data monitoring, as well as quality of life of cancer patients and survivors. Furthermore, synergies and complementarities will be sought between Destination 3 and the implementation of the EU4Health Programme (2021-2027)[4]. These synergies and complementarities could be achieved, notably through mechanisms based on feedback loops, enabling on the one hand to identify policy needs that should be prioritised in research and innovation actions and facilitating on the other hand the implementation of research results into policy actions and clinical practice, thereby providing an integrated response across sectors and policy fields.

Expected impacts:

Proposals for topics under this destination should set out a credible pathway to contributing to tackling diseases and reducing disease burden, and more specifically to several of the following impacts:

  • Health burden of diseases in the EU and worldwide is reduced through effective disease management, including through the development and integration of innovative diagnostic and therapeutic approaches, personalised medicine approaches, digital and other people-centred solutions for health care. In particular, patients are diagnosed early and accurately and receive effective, cost-efficient and affordable treatment, including patients with a rare disease, due to effective translation of research results into new diagnostic tools and therapies.
  • Premature mortality from non-communicable diseases is reduced by one third (by 2030), mental health and well-being is promoted, and the voluntary targets of the WHO Global Action Plan for the Prevention and Control of NCDs 2013-2020 are attained (by 2025), with an immediate impact on the related disease burden (DALYs)[5],[6], [7].
  • Health care systems benefit from strengthened research and innovation expertise, human capacities and know-how for combatting communicable and non-communicable diseases, including through international cooperation. In particular, they are better prepared to respond rapidly and effectively to health emergencies and are able to prevent and manage communicable diseases transmissions epidemics, including within healthcare settings.
  • Citizens benefit from reduced (cross-border) health threat of epidemics and AMR pathogens, in the EU and worldwide[8], [9].
  • Patients and citizens are knowledgeable of disease threats, involved and empowered to make and shape decisions for their health, and better adhere to knowledge-based disease management strategies and policies (especially for controlling outbreaks and emergencies).

The EU benefits from high visibility, leadership and standing in international fora on global health and global health security.

[1] Currently, around 50 million people in the EU are estimated to suffer from two or more chronic conditions, and most of these people are over 65. Every day, 22 500 people die in Europe from those diseases, counting of 87% of all deaths. They account for 550 000 premature deaths of people of working age with an estimated €115 billion economic loss per year (0.8% of GDP).

[2] AMR is estimated to be responsible for 25 000 deaths per year in the EU alone and 700 000 deaths per year globally. It has been estimated that AMR might cause more deaths than cancer by 2050.

[3] https://ec.europa.eu/info/law/better-regulation/have-your-say/initiatives/12154-Europe-s-Beating-Cancer-Plan

[4] https://ec.europa.eu/health/funding/eu4health_en

[5] WHO Global Action Plan for the Prevention and Control of NCDs 2013-2020 (resolution WHA66.10), https://www.who.int/publications/i/item/9789241506236

[6] Including for instance the following voluntary targets (against the 2010 baseline): A 25% relative reduction in the overall mortality from cardiovascular diseases, cancer, diabetes, or chronic respiratory diseases; Halt the rise in diabetes and obesity; An 80% availability of the affordable basic technologies and essential medicines, including generics, required to treat major non-communicable diseases in both public and private facilities.

[7] Disability-adjusted life year (DALY) is a quantitative indicator of overall disease burden, expressed as the number of years lost due to ill-health, disability or early death.

[8] WHO global action plan on antimicrobial resistance, 2015

[9] EU One Health Action Plan against AMR, 2017

Eligibility & Conditions

General conditions

General conditions

1. Admissibility conditions: described in Annex A and Annex E of the Horizon Europe Work Programme General Annexes.

Proposal page limits and layout: described in Part B of the Application Form available in the Submission System.

 

2. Eligible countries: 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.

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. Because the US contribution will be considered for the calculation of the EU contribution to the partnership, the concerned consortium of research funders from eligible EU Members States and Associated Countries must expressly agree to this participation.

The Joint Research Centre (JRC) may participate as member of the consortium selected for funding.

 

3. Other eligibility conditions: described in Annex B of the Work Programme General Annexes.

 

4. Financial and operational capacity and exclusion: described in Annex C of the Work Programme General Annexes.

Award criteria, scoring and thresholds are described in Annex D of the Work Programme General Annexes.

The thresholds for each criterion will be 4 (Excellence), 4 (Impact) and 3 (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.

Indicative timeline for evaluation and grant agreement: described in Annex F of the Work Programme General Annexes.

Beneficiaries may provide financial support to third parties. The support to third parties can only be provided in the form of grants. Financial support provided by the participants to third parties is one of the primary activities of the action in order to be able to achieve its objectives. Given the type of action and its level of ambition, the maximum amount to be granted to each third party is EUR 10.00 million.

The funding rate is 50% of the eligible costs. This is justified by the pooling of proposers' in-kind contributions and in-house activities and by the nature of activities to be performed: in addition of joint calls, highly integrative activities (EU clinical trial preparedness, training, patients’ empowerment activities etc.) contributing to enhance the rare disease research and innovation ecosystem in Europe and beyond.

 

6. Legal and financial set-up of the grants: described in Annex G of the Work Programme General Annexes.

 

Specific conditions

7. Specific conditions: described in the specific topic of the Work Programme.

Support & Resources

Online Manual is your guide on the procedures from proposal submission to managing your grant.

Horizon Europe Programme Guide contains the detailed guidance to the structure, budget and political priorities of Horizon Europe.

Funding & Tenders Portal FAQ – find the answers to most frequently asked questions on submission of proposals, evaluation and grant management.

Research Enquiry Service – ask questions about any aspect of European research in general and the EU Research Framework Programmes in particular.

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The European Charter for Researchers and the Code of Conduct for their recruitment consult the general principles and requirements specifying the roles, responsibilities and entitlements of researchers, employers and funders of researchers.

Partner Search Services help you find a partner organisation for your proposal.

Latest Updates

Last Changed: January 29, 2024

The results of the evaluation are as follows:
- Number of proposals submitted (including proposals transferred from or to other calls): 1
- Number of inadmissible proposals: 0
- Number of ineligible proposals: 0
- Number of above-threshold proposals:1
- Total budget requested for above-threshold proposals: EUR 56 929 273




We recently informed the applicants about the evaluation results for their proposals.

For questions, please contact the Research Enquiry Service.

Last Changed: September 20, 2023

Call HORIZON-HLTH-2023-DISEASE-07 closed on 19 September 2023. 1 proposal was submitted. The breakdown per topic is:

  • HORIZON-HLTH-2023-DISEASE-07-01: 1 proposal

Evaluation results are expected to be communicated on 24 January 2024 at the earliest.

 

Last Changed: September 20, 2023

Call HORIZON-HLTH-2023-DISEASE-07 closed on 19 September 2023. 1 proposal was submitted. The breakdown per topic is:

  • HORIZON-HLTH-2023-DISEASE-07-01: 1 proposal

Evaluation results are expected to be communicated on 24 January 2024 at the earliest.

 

Last Changed: January 12, 2023
The submission session is now available for: HORIZON-HLTH-2023-DISEASE-07-01(HORIZON-COFUND)
European Partnership on Rare Diseases | Grantalist