Upcoming and open funding callsWe're continuously updating this page with calls relevant to biomedical imaging
Below are the full call texts for open and forthcoming calls relevant to biomedical imaging. This includes information on the budget and prospective estimates on how many projects will be funded. You can scroll down, or use the quick menu on the right side of the screen to navigate.
A confidential copy of the draft 2023-2024 Horizon Europe Health work programme and the draft 2023-2024 Cancer Mission calls is available at the EIBIR office. Members are invited to get in touch for more information.
Calls for Funding
- DP-g-23-31-02: Call for proposals to support stakeholders on the prevention of NCDs in the area of chronic respiratory diseases
- DP-g-23-34: Call for proposals for operating grants to NGOs: financial contribution to the functioning of health non-governmental bodies implementing one or more specific objectives of Regulation (EU) 2021/522
- CR-g-23-44-01: Call for proposals to support the implementation of the strategic agenda for medical ionising radiation applications (SAMIRA95) – organisation of clinical audit campaigns as a tool to improve the quality and safety of medical applications of ionising radiation
- CR-p-23-41: Development of EU guidelines and quality assurance scheme for lung, prostate and gastric cancer screening
- CR-p-23-44-02: To support the implementation of the strategic agenda for medical ionising radiation applications (SAMIRA) – study on the implementation of the EURATOM and the Union legal bases with respect to medical devices used in medical applications of ionising radiation
- EU4H-2022-PJ-11: Call for proposals on NCDs – cardiovascular diseases and diabetes
- EU4H-2022-PJ-12: Call for proposals on cancer and other NCDs prevention – action on health determinants
- EU4H-2022-PJ-14: Call for proposals to support the roll-out of the second cohort of the inter-speciality cancer training programme
- EU4H-2022-PJ-15: Call for proposals to support structured dialogue at national or regional level on public procurement in the health sector – HERA
- IHI-03-01: Screening platform and biomarkers for prediction and prevention of diseases of unmet public health need
- IHI-03-02: Patient generated evidence to improve outcomes, support decision making, and accelerate innovation
- IHI-03-03: Combining hospital interventional approaches to improve patient outcomes and increase hospital efficiencies
- IHI-03-04: Strengthening the European ecosystem for Advanced Therapy Medicinal Products (ATMPs) and other innovative
therapeutic modalities for rare diseases
- IHI-03-05: Digital health technologies for the prevention and personalised management of mental disorders and their long-term
- IHI-04-01: Expanding translational knowledge in minipigs: a path to reduce and replace non-human primates in non-clinical drug
- IHI-04-02: Patient-centric blood sample collection to enable decentralised clinical trials and improve access to healthcare
- IHI-02-01: Cardiovascular diseases – improved prediction, prevention, diagnosis, and monitoring
Call for proposals to support stakeholders on the prevention of NCDs in the area of chronic respiratory diseases
Indicative budget: € 1 million
Opening: Q2-Q3 2023
Keywords: noncommunicable diseases; chronic respiratory diseases; public health; Beating Cancer Plan
The action will implement projects on health promotion and disease prevention, and is expected to support the Member States’ efforts to reduce the burden of NCDs (approximately 80% of the disease burden in Europe), in particular that related to CRDs, and to reach the Sustainable Development Goal 3, in particular target 3.4.
The expected results will include initiatives to complement the Member States’ efforts in the design, planning and implementation of best practices, such as for the development of public health guidelines, patient pathways, and support for the preparation and roll-out of new policy approaches, participation in the pilot testing of innovative practices, development of support actions such as training and improving health awareness and health literacy.
The aim of this action is to complement the implementation of the joint action “DP-g-23-31-01 ‘Healthier Together’ EU NCD Initiative – Chronic respiratory diseases” led by the Member States, thus helping to reduce the burden of CRDs in the Union, both at personal and
population level, targeting or addressing the related risk factors and their determinants, as necessary.
The activities will cover the prevention and management of CRDs and will complement the joint action. Activities will include implementing projects involving civil society organisations to support the Member States’ authorities in the implementation of comprehensive public health policies, the development and transfer of best practices, the development of public health guidelines, the preparation and roll-out of innovative approaches, projects supporting patient pathways and the launching of projects expected to have a significant public health impact and which benefit citizens directly. These may include projects to support the Member States in meeting the objectives of the Zero Pollution Action Plan82, the Chemicals Strategy for Sustainability83 and of the Europe’s Beating Cancer Plan.
Activities should also include an equity dimension and aim at reducing health inequalities.
Call for proposals for operating grants to NGOs: financial contribution to the functioning of health non-governmental bodies implementing one or more specific objectives of Regulation (EU) 2021/522
Indicative budget: € 9 million
Opening: Q1 2023
Keywords: non-governmental organisations; health literacy; capacity building; communication and dissemination
Through their core operational activities the health NGOs will deliver on increased health literacy and health promotion, capacity building and networking contributing to the optimisation of healthcare activities and practices, by providing feedback from and facilitating
communication with patients.
The beneficiaries are expected to further demonstrate in their proposals the EU added value of their activities and commit to deliver concrete results such as: online materials, webpages, manuals and tools on case studies promoting health in schools, factsheets and relevant literature, materials for teachers on health literacy, and assistance and promotion of twinning with other schools in the EU capacity-building and training activities to reduce the impact of risk factors for non-communicable diseases; new approaches to promote healthy and sustainable diets; expert guidance and peer-to-peer connections; and collaborate in shared areas of activity.
Some of the beneficiaries’ activities are expected to contribute to the implementation of non-legislative policy initiatives and/or the implementation of relevant Union health legislation. The beneficiaries will facilitate the exchange of knowledge, capacity building related to their expertise and should cooperate with other civil society organisations and international organisations (e.g., WHO and other organisations).
The objective is to ensure the participation of health NGOs in activities that are necessary to implement one or more specific objectives of the EU4Health Programme. Hence, operating grants should provide support to the functioning of certain NGOs during 2023 for activities
including awareness raising on various health aspects, communication and dissemination, capacity building and training, expert collaboration and networking.
Call for proposals to support the implementation of the strategic agenda for medical ionising radiation applications (SAMIRA95) – organisation of clinical audit campaigns as a tool to improve the quality and safety of medical applications of ionising radiation
Indicative budget: € 1.5 million
Opening: Q2-Q3 2023
Keywords: clinical audit; medical radiation; accreditation and certification; Basic Safety Standards Directive; radiology; radiotherapy; nuclear medicine
The action will contribute to a better implementation of the BSS Directive’s requirement with regard to clinical audit taking into account the differing challenges across Member States. It can serve as a reference action to establish a permanent clinical audit mechanism in some Member States.
It will improve the overall quality and safety of radiological medical procedures in order to bring their full benefits to patients. It will contribute to the development of the professional skills of the auditors and of the audited professionals and foster inter-disciplinary and multi-professional relationships. It should contribute to the development of leadership in this area.
This action could also strengthen structures involved in hospital accreditation or individuals involved in professional healthcare certification schemes. The pilot outcomes should be relevant to health systems as a whole and be designed in a way that their outcomes can be scaled up into the broader health system practice of the Member State(s).
The objective of this action is to pilot clinical audit campaigns in Member States in diagnostic and interventional radiology, radiotherapy and nuclear medicine by identifying and bringing together relevant actors and resources. It should take into account the specificities of the national health systems. It will include coordination, planning, recruiting, training, auditing and reporting activities. It should build on the results of the QuADRANT and the EU-JUST-CT project.
Up to four proposals of different sizes will be accepted, ranging from organising pilot audits in a single (large) department or hospital, a hospital trust, a region or a single Member State to coordinated audits in several Member States and should be implemented in coordination with the appropriate health authorities. A priority will be given to proposals covering several types of medical practice in several Member States and also to different practices within different regions of a Member State. Proposals should include considerations and activities to scale up pilot outcomes into the broader health system practice of Member State(s).
In particular, the clinical audit action should seek to improve justification of radiological imaging, in line with the 2015 Council conclusions on this topic98, and the implementation of the optimisation principle. The action will be implemented in close cooperation with other SAMIRA activities on quality and safety of medical applications of ionising radiation and include a reporting on the pilots carried out in Member States to the SGQS.
Development of EU guidelines and quality assurance scheme for lung, prostate and gastric cancer screening
Indicative budget: € 7.5 million
Keywords: cancer screening; lung, prostate, gastric cancer; quality assurance; health guidelines
On 20 September 2022, the Commission adopted a proposal for a Council Recommendation on strengthening prevention ‘A new EU approach on cancer screening’ replacing Council Recommendation of 2 December 2003 on cancer screening 2003/878/EC119. In addition to the cancer screening programmes for breast, colorectal and cervical as recommended under the 2003 Council Recommendation, the Commission proposal recommends screening for lung, prostate, and under certain conditions, gastric cancer. Through the Commission initiatives on Breast and Colorectal Cancer, a system and methodology for the development of EU guidelines for cancer screening and treatment including also a Quality Assurance Scheme, has already been developed. Based on this existing methodology, guidelines for the screening of lung, prostate and gastric cancers will be developed as indicated in the Commission proposal for the Council Recommendation. The guidelines will be complemented by quality assurance manuals and tools to help the implementation and monitoring of their use in the Member States to support the further design, planning, and implementation of population-based and targeted cancer screenings, diagnosis and treatment.
This action supports the implementation of Europe’s Beating Cancer Plan and implements the EU4Health Programme’s general objective of improving and fostering health in the Union (Article 3, point (a) of Regulation (EU) 2021/522) through the specific objectives defined in
Article 4, points (a) and (i), of Regulation (EU) 2021/522.
To support the implementation of the strategic agenda for medical ionising radiation applications (SAMIRA) – study on the implementation of the EURATOM and the Union legal bases with respect to medical devices used in medical applications of ionising radiation
Indicative budget: € 300,000
Keywords: medical devices; radiation protection; legal implementation
A variety of nuclear and radiation technologies play a key role in the fight against cancer. Mammography, computed tomography and other forms of radiological imaging are indispensable technologies for all stages of cancer management. Radiotherapy is among the most effective, efficient and widely used cancer treatments available to patients and physicians. Nuclear medicine is routinely used for cancer diagnosis and follow-up, and increasingly available for cancer treatment. Medical applications of ionising radiation are constantly evolving in a complex regulatory environment and there is scope to improve coordination in implementing the different regulatory frameworks. This is the case with regard to medical devices used in medical applications of ionizing radiation that are subject to the EU medical devices and the Euratom radiation protection legislations, both setting requirements for installation and acceptance testing, reporting of adverse events, and other indicators. The results of the work will underpin further efforts to improve the coordination between the two legal bases and support their efficient implementation for the benefit of patients. This action supports the implementation of Europe’s Beating Cancer Plan and implements the EU4Health Programme’s general objective of improving and fostering health in the Union (Article 3, point (a) of Regulation (EU) 2021/522) through the specific objectives defined in Article 4, points (a) and (h), of Regulation (EU) 2021/522.
Call for proposals on prevention of NCDs – cardiovascular diseases, diabetes and other NCDs
Indicative budget: € 5 million
Opening: 15 September 2022
Deadline(s): 28 February 2023
Keywords: noncommunicable disease; EUNCD; best practices; public health guidelines; innovation
The action will contribute to the implementation of projects on disease prevention and health promotion, which are expected to reduce the burden of NCDs, namely diabetes and cardiovascular diseases in Member States.
The expected results will include initiatives to complement the Member States’ efforts in the design, planning and implementation of best practices, such as support for the development of public health guidelines, support for the preparation and roll out of new policy approaches; participation in the pilot testing of innovative practices; development of support actions such as training and twinning, health communication or health literacy; and implementation of best practices in health promotion and disease prevention. The short-term impact will be an increased number of public health interventions being scaled up in all Member States and improvements in disease prevention and health promotion, and management policies relatedto NCDs.
The aim of this action is to reduce the burden of NCDs and related risk factors, targeting:
a) Cardiovascular diseases and diabetes, both at an individual and societal level, namely by supporting the NCDs policies and corresponding actions led by the Member States;
b) NCDs other than the five action strands of the initiative ‘Healthier Together – EUNCD Initiative’ and cancer; such as chronic kidney diseases and liver diseases, auto-immune diseases, musculo-skeletal conditions, etc.
This action will complement the Member States’ joint action on ‘Prevention of NCDs -Cardiovascular diseases and diabetes’ (DP-g-22-06.03) that aims to contribute to reducing the burden of NCDs, i.e. cardiovascular diseases and diabetes, and related risk factors, both at apersonal and societal level, including the health inequalities dimension.
The civil society organisations will implement targeted projects and activities, complementing the Member States’ efforts in the design, planning and implementation of best practices, including support to the definition of public health guidelines, to the preparation and roll out of new policy approaches, to the pilot test of innovative practices, and to support actions such as training and twinning, health communication or health literacy. Activities should also include an equity dimension and aim at reducing health inequalities.
Call for proposals on cancer and other NCDs prevention – action on health determinants
Indicative budget: € 11 million
Opening: 15 September 2022
Deadline(s): 28 February 2023
Keywords: Beating Cancer Plan; Farm to Fork Strategy; HealthyLifestyle4All; best practices; public health guidelines; health inequalities
The action will implement projects on disease prevention and health promotion, which isexpected to reduce the burden of cancer and other NCDs in the Member States.
The expected results will include initiatives to complement the Member States’ efforts in the design, planning and implementation of best practices, such as support for the development of public health guidelines and, support for the preparation and roll out of new policy approaches; participation in the pilot testing of innovative practices; development of support actions such as training and twinning, health communication or health literacy; and implementation of best practices in health promotion and disease prevention.
The short-term impact will be an increased number of public health interventions being scaled up in all Member States and improvements in disease prevention and health promotion, and management policies related to cancer and other NCDs.
The aim of this action is to complement the implementation of the joint action on ‘Cancer andother NCDs prevention – action on health determinants’ led by the Member States, thus helping to reduce the burden of cancer and other NCDs, and related risk factors, both at a personal and societal level, namely by supporting the Europe’s Beating Cancer Plan andpolicy initiatives on NCDs. It may also support other Union initiatives that aim to improve public health such as the Farm to Fork Strategy and the HealthyLifestyle4All initiative in so far as it shares the objectives of promoting sustainable food consumption and facilitating the shift to healthy, sustainable diets and promoting a healthy lifestyle.
Activities will run in parallel to the joint action, include implementing targeted projects involving civil society organisations complementing the Member States’ efforts in the design, planning and implementation of best practices, the production of public health guidelines, patients’ and caregivers’ consultations, or other actions that can benefit citizens directly, the preparation and roll out of innovative practices (pilot test), and support actions such as training and twinning, health communication or health literacy. The activities include provision of input via the Health Policy Platform, namely as regards their role inimplementation.
Activities should also include an equity dimension and aim at reducing health inequalities.
Call for proposals to support the roll-out of the second cohort of the inter-speciality cancer training programme
Indicative budget: € 7 million
Opening: 15 September 2022
Deadline(s): 28 February 2023
Keywords: trustworthy AI tools, risk prediction, chronic non-communicable diseases, evidence-based recommendations and guidelines
The action will increase the coverage of the inter-speciality cancer training programme across the Union, and will result in the upskilling and re-skilling of healthcare professionals in the areas of clinical oncology, surgery, radiology and radiation technology, and nursing and other specialised services, with an increase in the number of training centres engaged in the initiative, as well as trainees and trainers. This action will help Member States to improve cooperation among their cancer services, by addressing skill gaps and better equipping the health workforce with personnel trained in cancer care.
The aim of this action is to extend the implementation coverage of the first cohort of the inter-speciality cancer training programme through enrolling new cancer centres, as well asadditional trainees and trainers.
The action will roll-out the second cohort of trainees and, in addition, will organise a dissemination event to present the outcomes of the training. The action is expected to be complemented by the organisation of events to share the experiences developed during the training, which will also strengthen the networking of the trainees and trainers across the Union.
Specific activities will include a selection process for the trainees, trainers, and cancer centres which will participate in the inter-speciality cancer training programme. The programme is expected to train medical doctors and nurses and other specialised staff (such as thoseinvolved in medical imaging and radiation oncology including radiation technologists, medical physicists, radiobiologists, etc…) working in cancer centres in the three specialties of clinical oncology, surgery, radiology and radiation oncology with the aim of optimising the inter-speciality approach and cooperation. The training will be based on curricula previously developed ad hoc for the action and will include technology-based learning, simulations, and on-the-job training. The training programme will follow a coaching and mentoring approach with regular reporting and assessment of skills and tasks developed; case studies will be part of the training programme.
Call for proposals to support structured dialogue at national or regional level on public procurement in the health sector – HERA
Indicative budget: € 5 million
Opening: 15 September 2022
Deadline(s): 28 February 2023
Keywords: public procurement; supply chain; healthcare resilience; cross-border threats
The action is expected to result in the development of new or improved national and regional strategies on public procurement that will make current practices more resilient and efficient all over the Union. This will increase preparedness for future health crises.
The involvement of Member States at national, regional and/or local level as well as of all relevant stakeholders by using collective intelligence methods will also increase coordination and more and better cooperation for the future
Specific mandatory deliverables and/or milestones
A high coverage of MS is encouraged in the proposal, noting that the total budget of the call was established to conduct such work ideally in around 12 Member States.
Minimum activities that should be covered by the proposal:
Mapping of the public procurement environment in the health sector in the Member States covered by the project including:
– Mapping of purchases made by central purchasing bodies or hospitals, bound by EU public procurement rules. Concretely, a mapping should be made of who is buying what, from whom, and how? This will allow to understand not only the concrete needs public buyers have, but also to set concrete objectives, such as ensuring the security of supply chain, ensuring more innovative and sustainable purchases, etc.
-Mapping of all relevant stakeholders involved.
– Mapping to be done in a format that can be easily used for statistics, data gathering, graphics.
Listing of relevant thematic topics to organise the work (preliminary topics should be mentioned in the proposal) and facilitate exchanges, gather opinions and draw lessons learned, on the basis of the above mentioned mapping.
Organisation of brainstorming sessions, dialogues or additional work arrangement on the different thematic topics identified by way of collective intelligence with the involvement of all stakeholders and decision makers. Reports on the assessment of the outcomes of those brainstorming working sessions on each of the thematic topics discussed including a first draft of recommendations and which will form the basis for an overall strategy on public procurement for/by hospitals who are bound by public procurement rules.
New strategies for public procurement in health sector tailored to the needs of the Member State or region concerned as well as an implementation plan based notably on:
– Discussion of all recommendations in order to develop up a first draft national/regional strateg(y)ies with the involvement of all stakeholders and decision makers,
– Exchange between the consortia from different awarded projects in order to draw and share lessons learned in and within Member States,
– Discussion and finalisation of draft strategy(ies) at national and/or regional level as well as an implementation plan at national and/or regional level.
Dissemination of the results of the project in a final conference with a European dimension. While preparing the conference, results of the activities carried out under the same call for proposals shall be covered to cross-fertilise results among projects.
During the entire project, selected consortia are encouraged to cooperate with one another. Thereto, it is expected they will include in their proposals the possibility to organize common meetings, for example a kick-off that could be organised by the Commission for all the awarded projects.
Specific action-level indicators for reporting purposes
– Number of stakeholders included in the mapping (i.e. outreach of the activity)
– Number of information products (e.g. data, statistics, graphical representations etc) stemming from the mapping
– Number of key stakeholders identified by topic (i.e. stakeholders’ mapping)
– Number of events (e.g. brainstorming sessions, dialogues etc.) organized by topic
– Number of participants to the events by topic and by country
– Number of stakeholders involved in the reflection on public procurement strategies, by topic, by type (Member States, beneficiaries, health care entities etc) and by country
– Number of stakeholders endorsing the recommendations/strategies produced, by topic, by type (Member States, beneficiaries, health care entities etc) and by country
– Number of participants to the project conference by type and by country
Screening platform and biomarkers for prediction and prevention of diseases of unmet public health need
Indicative budget: € 10 million
Keywords: biomarkers; unmet public health needs; cost-effective interventions; advanced analytics; artificial intelligence
Proposals under this topic should aim to deliver results that contribute to all of the following expected outcomes for disease(s) of high unmet public health need selected by the applicants:
- Patients will receive earlier or more timely interventions (prevention, early treatment to avoid complications, etc) to reduce morbidity and mortality from major diseases, improving lives of citizens;
- Health care professionals have access to a screening platform and clinically validated biomarkers for identifying people at risk of disease to facilitate the selection of the most appropriate preventative action;
- Researchers have new biomarkers for prediction and prevention to allow for the development of safer and more effective personalised interventions tailored to the individual’s characteristics;
- Healthcare systems will benefit from reliable evidence to target effective, preventative therapeutic interventions to those citizens who will benefit most from them.
As the population of the EU ages, the rising burden of disease is an important challenge to the sustainability and resilience of healthcare systems. The identification of individuals at risk of developing an illness so that they can receive an appropriate treatment before the disease develops is an important factor to address this problem. However, for many health conditions, we lack full understanding of the underlying mechanisms, including the predisposition to disease and how environmental and genetic factors affect the occurrence of
Projects funded under this topic should address this challenge by developing an open platform for screening individuals who are not yet diagnosed with a disease with the aim of identifying people at risk. Applicants should clearly identify disease(s) of unmet public health need,1 and specify the initial biomarkers to identify people at risk that will be used within the project (e.g. genetic, metabolic, digital and imaging biomarkers, lifestyle, family inherited disease) and explain their choices with empirical evidence where possible. By the end of the project, the screening platform should be able to be used for population screening and decisionmaking including selection of the most appropriate intervention(s) and new technology development.
In particular, for the selected disease(s), the project(s) funded under this topic are expected to:
- Set up a comprehensive interdisciplinary collaboration of the clinical research, industrial, public health and health technologies communities to develop the screening platform and generate the evidence base for general population screening. This platform should be open to devices from different vendors and build on clearly identified existing initiatives where relevant;
- Clinically validate and assess the utility of the screening platform and biomarkers to identify people at risk by designing and implementing a large-scale general population cohort screening study in several European countries;
- Design and clinically validate innovative assay technologies for disease risk identification, including digital technologies with data capture/analysis;
- Deliver digital tools for more effective and efficient management and execution of screening programmes
and improved disease prevention. AI tools should be explainable where relevant;
- Publish the relevant methods, standard operating procedures (SOPs), algorithms, standards and guidelines to allow the platform to be used more broadly and for diagnostics and therapies to be developed;
- Develop a plan/roadmap to facilitate the eventual regulatory qualification of the biomarkers used within the project;
- Develop and optimize relevant clinical practice guidelines through systematic evidence and outcome review;
- Raise awareness of disease prevention and provide training and education to relevant health care professionals, patients and family members. These training materials should be made available for use after the project ends.
A key objective is to facilitate changing healthcare practice, so applicants will need to demonstrate that their outputs can be taken up by healthcare systems and take steps to facilitate this.
Applicants should also reserve resources to synergise with other relevant initiatives, including other projects funded under this topic.
Patient generated evidence to improve outcomes, support decision making, and accelerate innovation
Indicative budget: € 8–10 million
Keywords: patient-reported outcome measures; patient preference information; policy dialogue; awareness raising; patient-reported experience measures; integrated healthcare; m-health; e-health
Proposals under this topic should aim to deliver results that contribute to all of the following expected outcomes for the use cases selected:
- Decision makers have new methods for the integration of PROMs, PPI, and PREMs and other people generated information into regulatory and health technology assessment evaluation processes for integrated healthcare solutions;
- Patients of all ages have access to novel integrated healthcare solutions that are developed using structured patient input and better respond to their needs and preferences;
- Researchers have new methodological approaches to elicit and integrate patient preferences into the conception, development, and implementation of integrated healthcare solutions;
- Researchers have wider access to interoperable, quality patient input and patient generated data, respecting the FAIR (findable, accessible, interoperable, reusable) principles, facilitating research and development of integrated healthcare solutions;
- Researchers are provided with new outcomes, outcome measures and the time horizon over which value should be assessed to develop appropriate tools and methods for collection and analysis of PROMs, PPI, and PREMs.
The amount of health data generated by citizens themselves is rapidly increasing. Such data includes patient-reported outcome measures (PROMs), patient preference information (PPI), and patient-reported experience measures (PREMs) as well as other digital health data/digital biomarkers. While the potential for these data to be harnessed to improve individual healthcare is enormous, these data are often fragmented among multiple providers, so that neither the citizen, nor the healthcare ecosystem have a comprehensive
overview, and therefore it is very challenging to fully use these data to provide reliable evidence for decisionmakers, and to improve health outcomes.
Projects funded under this topic will aim to address this challenge by:
- Developing a framework to integrate patient input (PROMs, PPI, PREMs) and patient-generated data (digital health data/digital biomarkers) for use in decision making (regulatory, health economic evaluation, reimbursement, healthcare programme design, tailored prescription of therapies, and technology development), building on existing frameworks where appropriate. This framework should allow for the benefit-risk evaluation and value assessment of integrated care solutions;
- Implement several use cases to support and demonstrate the use of the framework, focussing on using patient input and patient generated evidence to address challenges that are not adequately addressed by other initiatives. These use cases should demonstrate the value of using patient input (PROMs, PPI, PREMs) and patient generated data (digital health data/digital biomarkers) across the healthcare continuum including showcasing improvements to data interoperability, healthcare workflows and processes, disease prevention, and care, including home-based care. These use cases should also act as examples of best practice for future use of the framework;
- Facilitating multi-stakeholder access to patient inputs and patient generated health data such that actionable harmonised data can be used for quality decision making;
- Comparing/contrasting the properties of the three types of patient input (PROMs, PPI, PREMs), identify differences and opportunities for integrated/complementary use;
- Developing an approach or approaches to integrating PROMs, PPI, and PREMs data into the design of core outcomes sets, end-to-end patient treatment pathways, clinical decision support systems, and treatment guidelines. The core outcome sets used within the project should be made available more widely where possible.
Applicants should also reserve resources to synergise with other relevant initiatives including the European Health Data Space.
Combining hospital interventional approaches to improve patient outcomes and increase hospital efficiencies
Indicative budget: € 8–10 million
Keywords: fragmentation; innovative interventional approaches; evaluation methodologies; advanced analytics; artificial intelligence
Proposals under this topic should aim to deliver results that contribute to all of the following expected outcomes:
- Patients will be offered improved, evidence based innovative hospital treatment combinations that lead to better outcomes;
- Healthcare professionals will have access to improved clinical decision support systems that will recommend personalised treatments using patient-specific datasets collected in the hospital setting;
- Healthcare systems will have better evidence on cost-effective combinations of interventions and how these combinations can address hospital staffing challenges;
- Researchers will have improved information on treatment combinations to facilitate the development of improved interventions.
Patients admitted to hospital to undergo elective or non-elective procedures typically require recovery and rehabilitation to get back to normal life. New innovative treatment approaches such as minimally invasive surgical approaches, locoregional interventions, imaging, diagnostics, monitoring, clinical decision support systems, and robotics have the potential to reduce complications after surgery, facilitate faster recovery, and help reduce hospital staffing challenges. However, due to limitations in interoperability, reliable evidence and suitable guidelines, these innovative approaches, treatment options and clinical decision support systems are not being optimally combined to provide the best patient care.
Projects funded under this topic should address this challenge by showcasing how existing hospital interventions, treatment approaches and technologies can be optimally combined to improve patient outcomes, enhance patient pathways, help to lower costs, generate efficiency gains, reduce hospital staffing challenges, and decrease societal burden.
In particular, projects should:
- Access & integrate clinical data routinely generated using existing technologies during the patient journey (e.g., medical history profile of patients, diagnosis achieved, for example, by medical imaging and in-vitro diagnostic (IVD) tests, digital information generated during the surgery, vital signs and anaesthesia management, clinical outcome records in electronic healthcare record systems (EHRs), drug prescriptions such as analgesics). Suitable, secure IT infrastructure to support edge and cloud computing in compliance with the general data protection regulation (GDPR) and other data privacy policies at national and local levels should be utilised;
- Train and clinically validate AI algorithms to support the development of training programs, surgical planning and intraoperative assistance solutions, including clinical decision support systems;
- Demonstrate, via use-cases using these data & algorithms, how combinations of and/or synergies between the above-mentioned tools, technologies, and therapeutic approaches can be harnessed to improve patient care. This should include comparing the combination of innovative interventional approaches and clinical decision support systems (CDSS) versus limited or no systematic combination of these innovative interventional approaches and CDSS;
- Implement tools to confirm successful treatment during or after the procedure and monitor therapy response and disease regression;
- Demonstrate the medico-economic value of the solutions for all stakeholders as a use case for future procurement tenders;
- Encourage the uptake of the results of the project through a strong communication and outreach plan, including the publication of a gap assessment in order to guide future research in this field..
Applicants should also reserve resources to synergise with other relevant initiatives, including any projects resulting from Horizon Europe Cluster 1 Health topics.
Strengthening the European ecosystem for Advanced Therapy Medicinal Products (ATMPs) and other innovative therapeutic modalities for rare diseases
Indicative budget: € 20–30 million
Keywords: rare diseases; advanced therapy medicinal products; centres of excellence; clinical networks
Research and innovation actions to be supported under this topic must contribute to all the following outcomes:
- A sustainable network of interconnected centres of excellence with all relevant stakeholders engaged, that should:
- Be accessible by all involved in the development of ATMPs and other innovative therapeutic modalities, including the research community, academia, clinics, small to medium-sized enterprises (SMEs), healthcare, biotech, medical technology and pharmaceutical companies, patient organisations;
- Represent the most promising, impactful, translatable, quality-controlled technologies that facilitate the development of ATMPs and other innovative therapeutic modalities such as use of nucleic acids and nanoparticle (NPs) delivery for gene editing;
- Agreed standards that support the early generation of ATMPs and other innovative therapeutic modalities;
- A streamlined and more transparent regulatory pathway to optimise and speed up the development and delivery of ATMPs and other innovative therapeutic modalities for rare diseases for the benefit of patients, carers, healthcare systems and the society;
- Improved technologies/processes, analytic tools, methods, assays useful for the development and support of all ATMPs and other innovative therapeutic modalities, beyond those targeting underserved rare diseases.
There are over 7000 rare diseases resulting in 30 million patients in Europe with a rare disease. Globally more than 300 million patients are affected. In Europe, less than 10% of rare disease patients receive treatment and only 1% are managed using an approved treatment. There are many challenges in the development of treatments for rare diseases, notably ATMPs and other innovative therapeutic modalities, such as gene and cell therapies. They have shown promise yet rely on complex technologies with largely underdeveloped manufacturing processes.
The aim of this topic is to optimise and streamline the development of ATMPs and other innovative therapeutic modalities for rare diseases by strengthening the ecosystem that facilitates the transition of early research to development. This will streamline and improve the value chain of advanced therapies from bench to market.
To fulfil this aim, the selected projects should:
- Establish a network of interconnected and complimentary scientific and technical centres of excellence (de novo and/or existing laboratories/institutions) in defined types of ATMPs or other innovative therapeutic modalities to enable translational research. This covers therapeutic approaches relevant to the future treatment of genetically defined diseases. The scientific and technical centres are expected to provide access and advance translatable, quality-controlled technologies. These centres should develop technologies and share data, and to provide an opportunity to define key characteristics of ATMPs, and quality standards that are critical to later stage development. Relevant therapeutic modalities include recombinant adeno-associated virus (rAAV), and innovative modalities such as messenger RNA (mRNA) and nanoparticles (NPs) for therapeutics. Technology areas of interest include targeted delivery, stability, transgene expression, advanced redosing technology approaches/reduced immunogenicity of gene delivery platforms, and other underlying biology relevant to the specific therapeutic modality, enabling accelerated translation to clinical development and manufacturing;
- Develop standardised analytical assays, methods and technological platforms, and design strategies for:
- Reducing the timeframe and costs and improve the robust assessment of therapeutic modalities and/or;
- Optimising of advanced manufacturing processes to assure product quality, ensuring broad accessibility of critical manufacturing materials and demonstrating the economy of scale for ATMPs or other innovative therapeutic modalities;
- Demonstrate the translatability, scalability, and robustness of technologies suitable for the development of subsequent ATMPs and other innovative therapeutic modalities. This may include process development, mRNA and NPs scale up and stability, vector production, improvement of the throughput capability for the systematic assessment of the biological and mechanistic features and product characterisation, and ensuring broad accessibility of critical manufacturing materials such as cell lines and producer plasmids;
- Implement use cases in defined types of ATMPs, representing groups of rare diseases with commonalities, with the aim of demonstrating the performance of the methods and technologies developed for addressing the bottlenecks in the development and manufacturing cycles of ATMPs and other therapeutic modalities;
- Assess the methods and technological platforms developed for their regulatory validity/utility. Define a regulatory pathway to support the fit-for purpose development of ATMPs, taking into account an evolving regulatory environment and the interplay between all applicable legislation. Engage early with the regulators to ensure that the methods and data generated support regulatory needs;
- Contribute to strengthening the European rare disease ecosystem by ensuring the engagement of all relevant stakeholders, especially patients and patients’ representatives for rare diseases, carers, clinicians, regulators;
- Define metrics to measure the use of centres of excellence by relevant stakeholders for the development of their assets or novel technological solutions/therapies.
This topic focuses on the scientific and technological barriers that are limiting the rapid and cost-effective development of ATMPs and other innovative therapeutic modalities for underserved rare disease patients. To test the functionality of the centres of excellence, the utility of the processes developed and whether they are fit-for purpose, ATMPs may be used for proof of concept. Nonetheless the development of ATMPs and/or clinical trials are out of scope for this topic.
Applicant consortia should take stock of the state-of-the-art methods and technologies delivered by other inter-connecting EU and global initiatives (e.g., Bespoke, IMI project ARDAT, or other EU funded consortia) and their proposals should reflect potential synergies and collaborations to ensure complementarity while avoiding duplication.
Digital health technologies for the prevention and personalised management of mental disorders and their long-term health consequences
Indicative budget: € 8–10 million
Keywords: digital health technologies; mental healthcare; early prevention; precise diagnosis; pathway management; European Health Data Space; long-term impact
R&I actions to be supported under this topic shall contribute to all of the following outcomes:
- Robust evidence on the feasibility, adherence, and personal satisfaction with digital health technologies (DHT) in people with mental disorders. This includes pathways to maximise motivation and engagement of the relevant patient population and their families/caregivers, as well as of social workers and teachers where relevant. This includes patient-centric selection of potential application features, measurement technologies and digital endpoints. Proper attention should be given to the issue of vulnerability and stigma. Consideration should be given to cultural, gender and age-specific (e.g. adolescents) needs and preferences to ensure continued use of the DHT, and to the fostering of engagement of end-users and healthcare actors about DHT;
- A flexible, interoperable, and reusable digital platform that can be used across numerous conditions and scenarios (various mental disorders, comorbidities, long-term health consequences and other disease areas) to collect and analyse and integrate diverse multimodal clinical and patient data with an emphasis on those generated by DHT. Variability across countries should be addressed, as digital infrastructure and availability of digital tools may differ. Relevant consideration on ethical, social, and legal aspects and of the FAIR (findable, accessible, interoperable, reusable) principles must be addressed;
- Effective and agreed guidelines for development and implementation of DHT in clinical research and as a part of everyday health and care, enabling the development of more patient-centric treatments, optimised health and care interventions and better disease prevention. Relevant organisation and work processes, policy and regulatory aspects should be addressed to foster sustained integration of DHT in real world practice;
- Robust knowledge for better understanding of mental disorders, their change over time and how all this relates to clinical outcomes including remission, relapse, and recurrence of the conditions, long-term health conditions and mortality. Socioeconomic outcomes and family burden should be addressed;
- A robust body of data to enable the development of digital engagement tools for patients, caregivers and other relevant actors (healthcare professionals, social workers etc.) adapted to the needs of the patient population and age-specific needs, tackling the issue of stigma, vulnerability, lack of treatment seeking and overall poor adherence to treatment (including lifestyle related). Consideration should be given to provide intuitive equipment and user interfaces and easy troubleshooting;
- Enhanced and more reliable tools and methods (e.g., analytical tools and algorithms) able to provide (near) real time feedback on the DHT, including on the usability, efficacy/effectiveness, and long-term safety. Together, these enable healthcare professionals and providers to make more inclusive and efficient patient-centred decisions in collaboration with the patients and their families;
- Robust evidence of how DHT may influence patient treatment or behaviour. The inclusion of school/social workers in the evidence generation should be considered where relevant.
Mental health disorders represent an area of severe unmet public health need. This has been further negatively impacted by the COVID-19 pandemic, with a substantial increase in the number and severity of people affected for example by anxiety and depression, which creates substantial pressures on already strained mental health care systems. People with mental disorders have a reduced life expectancy compared to the general population, which is linked to a greater risk of developing a range of chronic physical conditions. The long-standing separation of psychiatry from other branches of medicine and the lack of specific training on this issue further contribute to the poor attention dedicated to management of physical comorbidities of mental health disorders.
Digital health technologies (DHT) applied via electronic devices such as wearable sensors, implanted equipment, and handheld instruments and smartphones have already shown significant promise for the prevention and disease management of chronic conditions (e.g. cardiovascular disease, diabetes, obesity). DHT, by making it possible to virtually perform medical activities that have traditionally been conducted in person, have also the potential to decrease the pressure on healthcare systems and their personnel. Thus, DHT might have the potential to address some of the challenges in the prevention, prediction, monitoring and personalised management of mental disorders and their long-term health consequences, as well as to tackle some of the organisational issues in providing mental health care.
The scope of this topic is to investigate how DHT might positively impact the healthcare pathway for people with mental disorders.
Applicants should demonstrate how DHT may enable:
- Better prevention and prediction of disorder onset or relapse;
- Better disease management;
- Tackling comorbidities;
- Addressing long-term health consequences (such as cardiovascular disease or diabetes.
The choice of the specific mental disorder should be justified based on unmet public health need and feasibility and preliminary evidence available on the use and value of DHT.
To contribute to breaking the silos between psychiatry and other medical branches and better address the impact of physical co-morbidities in people with mental disorders, applicants should consider a relevant comorbidity (and/or long-term physical consequence) disease area where DHT data, learnings and technologies are already available and can be further applied to mental disorders. The choice of this area must be justified accordingly.
Ways of decreasing the burden on caregivers and families should be considered, and applicants should actively engage these actors as well as the patients in addressing critical issues and research questions, including about (sustained) engagement with DHT. Consortia should propose ways to foster the future integration of digital and clinical mental healthcare, as well as how DHT might enhance the outcomes of interventions by social and healthcare professionals while decreasing the burden on the healthcare system.
Resources, and learnings from previous initiatives at European and national level (Innovative Medicines Initiative funded among others) should be taken into consideration.
Applicants should aim to deliver robust evidence on how DHT may be:
- Made easy to adopt and continue using for both patients, their families/caregivers and health and care providers;
- Effectively incorporated into clinical research and in clinician workflows.
Early engagement with regulators should be sought to ensure future acceptance and usability of the results.
Applicants are expected to implement activities to achieve all expected outcomes.
Projects selected under this topic are strongly encouraged to participate in joint activities as appropriate. These joint activities could, for example, involve joint coordination and dissemination activities such as participation in joint workshops, the exchange of knowledge, the development and adoption of best practices and adoption strategies on regional, national, and European level and synergies with other projects generated by this call as well as any other relevant initiatives, also at global level. Applicants should plan a necessary budget to cover these activities even if details of these joint activities will be defined by the successful applicants together with the IHI office during the grant preparation phase.
Expanding translational knowledge in minipigs: a path to reduce and replace non-human primates in non-clinical drug safety assessment
Indicative budget: In-kind contributions
Keywords: non-human primates; new research pathways; non-invasive digital technologies; non-animal models; knowledge sharing
- Obtain and share biological knowledge of minipigs, thereby facilitating the development of innovative solutions by improving the translational understanding between minipigs versus NHPs and humans, including further understanding of the minipig immune system, with the overall aim to replace, reduce and refine the use of animals in drug safety assessment;
- A regulatory pathway for drug safety assessment of biologicals and other novel therapeutic modalities in minipigs with the potential to impact regulatory strategies;
- Publicly available databases and software for physiologic, genomic, transcriptomic, metabolomic, proteomic and epigenetic minipig data to understand underlying mechanisms of disease/toxicities and find new mode of actions for pharmaceutical intervention;
- Characterized and validated genetically modified minipig models:
- Genetically modified minipig models based on the CRISPR/CAS inducible gene-editing technology;
- Minipigs with “humanized” immune system components and effectors for biologicals’ testing;
- Small–sized micropig for efficacy/safety assessment to facilitate compound availability in pharmaceutical R&D;
- Assessment of the utility of the minipig as a relevant toxicology species for immunosafety testing using drugs, which have been tested preclinically and clinically. Assisting and synergizing the already existing translational and regulatory efforts related to immunological safety evaluation. Developing validated antibodies and in vitro immunoassays to characterize the immune system and assess immuno-safety of drugs in minipigs;
- Minipig-specific technology for automated study data: validated medical devices, biosensors, algorithms, software, and digital animal housing. Machine learning and Artificial Intelligence (AI)-based tools to monitor abnormalities in behaviour and physiological systems in undisturbed animals.
To ensure long term sustainability, all the obtained interdisciplinary science-based knowledge generated in this proposal will be shared, integrated, digitalised, and published in peer-reviewed journals encouraging industry and academia to develop innovative medical science solutions and technologies, such as scientifically and ethically sound animal models, assays, biomarkers, monitoring devices, biosensors for normal physiological behaviour and algorithms. Based on the close collaboration with regulatory bodies, the generated knowledge in this proposal is further expected to impact regulatory guideline strategies. All outputs will require long term sustainability and maintenance to fulfil the scope of the proposal.
- Increasing need to find alternatives to testing in NHPs in line with EU legislation;
- Lack of knowledge about minipigs in safety assessment to allow de-selection of NHP as second nonrodent species across diverse drug modalities with strong scientific confidence;
- Almost no precedence in minipig use for safety testing of biologicals and novel therapeutic modalities [e.g., oligonucleotides, small interfering RNAs (SiRNAs), crystallizable fragments (Fcs), antigen-binding fragments (Fabs), single-chain variable fragments (scFvs), monoclonal antibodies (mAbs), vaccines, gene-editing and cell-based therapies];
- Lack of public minipig reference “omics” with good quality annotation: Full genome sequencing, in parallel with baseline transcriptomics, proteomics, metabolomics and epigenetic information;
- Lack of “humanized” and genetically modified models available for drug efficacy/safety testing, including genetically modified smaller micropigs to address cases of limited substance supply;
- Significant knowledge gap on the minipig immune system and reduced number of laboratory tools and reagents when compared to other toxicology species (rodent and non-rodent);
- Lack of widespread use of biosensors, medical devices, “intelligent” animal housing for automated data collection and analysis in minipig studies.
The overall objective of this proposal is to characterize the minipig for use in R&D of medical technology, device, and pharmaceutical development. The knowledge generated in this proposal may facilitate innovative health solutions, improve disease understanding and human predictions. The goal is to advance biomedical R&D by generating background scientific data to evaluate if the minipigs could be a viable and feasible alternative to NHP in key therapeutic areas, with a special focus on translatability from minipigs to humans.
- Safety assessment and regulatory aspects of novel therapeutic modalities: Compile and publish historical data in minipig biomedical R&D;
- Evaluate the translatability of minipigs in human risk assessment following treatment with biologicals and novel therapeutic modalities and discuss future perspectives of the minipigs with regulatory agencies;
- Minipigs multi-omics: Generate omics reference data (genomics, transcriptomics, proteomics, metabolomics, and epigenetic information) to enable translational research in minipigs;
- Genetically modified pig models including the micro-pig: Characterise and validate humanized and genetically modified minipig models, including the micropig to generate translatable animal models in drug safety assessment.
- iPig: Digital technologies, clinical data collection and AI: Create, validate, qualify, and benchmark digital solutions that can objectively measure clinically relevant and functional biomarkers in minipigs for use in preclinical toxicity studies in line with the regulatory agencies’ requirements;
- Minipig immune system: validate reagents, assays, and biomarkers for immunologic investigations: Conduct investigative studies in minipigs to support their translational significance in immuno-safety assessments and validate reagents/assays;
- Project management: Compile, digitalise, publish existing and newly produced data.
Patient-centric blood sample collection to enable decentralised clinical trials and improve access to healthcare
Indicative budget: In-kind contributions
Keywords: obesity, biological pathways and pre-obesity markers, environmental, socio-economic and lifestyle factors, prevention, evidence-based guidelines, recommendations
The results of the project generated by this topic will enable innovations in healthcare delivery and research by generating the infrastructure and logistics for blood collection at home, that is simple, minimally invasive, less painful, convenient, and feasible.
Importantly, the project will also set the stage to answer research questions by creating an unprecedented data set that will enable multiple secondary research options for years to come. Notably:
- It will create insights into the public acceptability for microsampling home: are patients comfortable with a new kind of medical technology? What training is necessary?
- Are we able to advance the transition of care from the hospital to the home? Does the care quality improve?
- How do we utilize the higher frequency of data, including its integration with electronic medical records and using advanced analytics methodology?
- Do doctor’s practices and decisions change with the increased frequency of biomarker data, and does it lead to better outcomes for the patient?
While integrating existing components for microsample collection and central lab analysis, quality standards for the new infrastructure and logistics will be rigorously and transparently validated and established in Europe and harmonised with parallel ongoing efforts in the USA. This will be undertaken in ways that are acceptable for patients and their caregivers, health care professionals, regulatory agencies, policy makers, Health Technology Assessment (HTA) experts, payers, and advocacy groups.
The overall aim of the project generated from this topic is to create and validate the infrastructure and logistics for blood collection by the patient and/or caregiver at home as a healthcare tool and an alternative to the current gold standard venous blood for routine clinical assays. This project will employ only commercially available CE-marked microsampling devices, according to their intended use. Development of new devices for blood sampling or of new clinical assays / analytes is not the focus of this project, and no new clinical assays will be evaluated. Similarly, given their current maturity, home sample analysis is out of scope.
Training materials, customised for patients and caregivers as well as for medical personnel will be developed, ensuring the acceptability of the new approach to these groups. Interactions with regulatory authorities, the European Medicines Agency (EMA), local European agencies as well as the USA Food and Drug Administration (FDA) will be sought to advance regulatory acceptability of the logistics model and harmonization across the EU, the UK and USA. Further, key stakeholders (e.g. policy makers, HTA experts, payers, patient advocacy groups) will be encouraged to implement the infrastructure and logistics throughout Europe. Lastly, the best ways to integrate, transmit, and analyse (including AI) the generated data will be explored. Results will be shared broadly through peer-reviewed publications or other mechanisms.
To be noted – home blood microsampling has been used in geographically restricted pilot projects. With the project generated from this topic , it is expected to generalise them, and leverage the learnings from the pilot projects, to enable broad adoption. Importantly, it is known that patients greatly appreciated this experience compared to the traditional blood sampling methods currently in use.
Applicants should in their proposal entail the following:
1. Demonstration of concordance between patient-centric microsampling techniques and venipuncture
This requires delivery of a framework across Europe for establishing concordance between capillary blood as collected by microsampling devices outside of traditional collection setting by the patient and/or caregiver, versus the gold standard venous blood, for routine clinical assays.
- To generate an umbrella / master protocol that is acceptable for regulatory authorities in Europe and UK and can be easily adopted for future applications (e.g. in additional patient populations, countries, by any vendor or organization). The umbrella / master protocol must include:
- Sites in at least 3 EU member states, and may include additional sites in third countries associated to Horizon Europe or other European countries (e.g. UK); at least one of the countries must be in Central or Eastern Europe;
- At least two different types (e.g., finger stick, upper arm capillary) of commercially available CE-marked microsampling devices;
- Routine clinical assays: i.e., blood chemistry, liver and lipid panels;
- Collection of at least 50% of microsamples by the caretaker or patient;
- Collection of least 50% of microsamples at home, and may include collections in other locations (e.g. hospitals, general practitioners, specialists’ offices) for concordance testing and establishing microsampling of capillary blood versus venous blood for routine assays;
- To design, adapt, and translate patient-facing materials, obtain ethics board approvals, recruit healthy human volunteers and expand to a patient population which should be agreed upon in a project committee, collect biological samples and conduct bioanalysis according to the study protocol;
- To investigate potential errors related to the mishandling of samples and design ways to mitigate them, as well as the potentially harmful downstream effects for the individual;
- To conduct concordance analyses according to existing regulatory guidance for routine clinical assays, and define sample quality criteria (if applicable).
2. Validation of the logistics of sample collection and shipping, standardising central lab analysis.
This requires identification of an optimum workflow for device ordering, fulfilment, shipping, at-home collection and return to central labs and a seamless integration of microsampling into current central lab processes; accessioning, analysis and reporting.
- To select at least two different types of CE-marked microsampling devices, and identify and audit device vendors with ordering (portal) and fulfilment capabilities; to work with device vendors on ordering devices;
- To define appropriate shippers/processing/temperature based on the devices and assay requirements, and confirm requisition requirements;
- To identify strategic partners in terms of Logistical expertise, e.g. global couriers;
- To identify countries to test devices in and confirm regulatory requirements for self-collections or collections by caretaker and shipping of devices;
- To define needed support for use of devices and train participants on devices; to identify telehealth partners e.g. for identification verification;
- To identify the best ways to integrate the new data with existing electronic medical records and medical decision frameworks;
- To investigate the “green dimension” of logistics: microsampling has the potential to reduce the green footprint of office visits and transportation required (fuel, costs, carbon emissions);
- To confirm accessioning process needed, reporting requirements, and data management model.
3. Education and Medical & Patient Acceptability
- To deliver training materials for patients, caregivers and clinical trial sites, ensuring smooth behindthe-scenes shipment logistics and support;
- To develop guidelines for compiling training materials to meet expectations from different training recipients, such as clinical sites, patients, caregivers, telehealth and home health providers, leveraging previous feedback collected from users (patients, caregivers, principal investigators (PIs) and medical personnel), including to develop training by telehealth;
- To develop a plan to collect patient, caregiver and medical personnel (site staff, PIs, trial coordinator) experience and feedback:
- To develop a well-designed questionnaire that will be used either electronically or in paper format, develop tool(s) to collect feedback and store the information, pilot the use, refine the questionnaire and data base as needed;
- To implement the questionnaire to collect feedback from different groups (patients and caregivers, medical personnel, regulators, device manufactures);
- To maintain a database of information collected and perform data analysis to obtain patient acceptability scores.
- To publish survey results to validate the training and feedback with other patient advocacy groups.
4. Regulatory acceptability and implementation into clinical practice in the EU, UK and USA
- To prepare an overview of the regulatory landscape of microsampling at home per country in the EU, third countries associated to Horizon Europe, and other European countries (e.g. UK), and to conduct an in-depth exploration in those countries that might be suitable for the “Microsampling logistics modelling;”
- To establish early and continuous dialogue with the European Medicine Agency (EMA) / Innovation Task Force, in addition to local regulatory agencies of the EU, the USA-FDA and other relevant authorities, e.g. the UK Medicines and Healthcare products Regulatory Agency (UKMHRA):
- To assess acceptability with regulators and seek (informal) prospective input on the umbrella / master protocol, choice of countries and approach to validate the logistics.
- To discuss the best strategy/timing for qualification and/or integration of project outputs into regulatory practices, prepare relevant documents (e.g. briefing books, EMA guidance document) to share project results, request scientific and qualification advice, and seek a harmonization with the USA-FDA and other authorities, e.g. UK-MHRA.
- To establish early and continuo
Cardiovascular diseases – improved prediction, prevention, diagnosis, and monitoring
Indicative budget: € 22 million
Opening: 28 June 2022
Deadlines: 20 September 2022 (First Stage), 28 February 2023 (Second Stage)
Keywords: cardiovascular diseases; prediction; prevention; diagnosis; monitoring; harmonised methodology; access and integration of heterogeneous health data
The results of the selected project will provide the basis for better primary and secondary prevention of CVD. The goal is to identify existing comprehensive CVD and heart failure (HF) patient datasets (with contextual parameters e.g., behavioural, socioeconomic, gender, ethnicity) and integrate them with data from diagnostic tools (e.g. wearables, imaging devices, bio samples / biopsies) and routine clinical practice. This will provide the basis for independently validated prediction models for improving the stratification of patients, and reveal insights to achieve earlier intervention. Additionally, the project will leverage developed algorithms to define and validate care pathways that tailor therapy towards individual patient needs and compare it to the “one-size-fits-all” approach.
This project is expected to achieve all of the following outcomes:
- Identification of relevant data sets, for instance derived from classical diagnostic screening; in-vitro diagnostics; ‘multi-omic’ platforms (comprising genomic, transcriptomic, proteomic and multimodality imaging data, most preferably with multiple timepoint assessments to ascertain the directionality and dynamics of relevant changes); continuous glucose monitoring (CGM) data, continuous electrocardiogram (ECG) data from wearables. In addition HF and activity data, wearable devices, digital health applications and routine clinical practice;
- Leverage data in currently available federated databases with ‘open access’ generated during, for example, IMI1/IMI2 projects in compliance with GDPR (General Data Protection Regulation), such as results/data/biomarkers/electronic health records provided by project participants, adding to the knowledge base;
- Demonstration of the utility of biomarker combinations including data from different modalities e.g., wearables, smart (acute or chronic) care setting devices, imaging/screening for the diseases and comorbidities;
- Based on existing biomarker combinations, determination of whether new biomarkers are needed for detecting patients at risk;
- Developed and/or evaluated artificial intelligence (AI) models that, using data from various sources, can identify patient subgroups who require and respond differently to the prevention and/or treatment of atherosclerotic cardiovascular disease (ASCVD) and HF in clinical practice;
- Identification of previously undiagnosed subgroups of ASCVD and HF patients, for instance people with insulin resistance, diabetes, and obesity, into clinically meaningful subgroups;
- Documentation and analysis of patient preferences regarding information, diagnosis and treatment of CVD, as well as requirements and preferences of individuals to share their data;
- Integration of patient data (e.g. via a federated database concept) to enable a holistic overview of specific patient groups to enable more effective and efficient disease management and execution of screening programmes and individual treatment tailoring;
- Inclusion of validated patient reported outcome and experience measure (PROMs and PREMs) data including biophysical, mental and psychosocial parameters with the aim of using it in a clinical setting. This may include, but is not limited to, measures on quality of life, sleep quality, physical activity, emotional stress, satisfaction with treatment, healthcare service experience;
- Leveraging developed algorithms/decision trees to define and validate care pathways that tailor therapy towards individual patient needs and compare them to the “one-size-fits-all” approach;
- Sustainability of relevant results and data repositories;
- Identification of incentives that reward positive health behaviour and motivate consistent and continuous data generation especially when health status has changed;
- Utilisation of the knowledge gained from the project to facilitate and guide better prevention, considering the patient perspective;
- Data collection in the patient population with type 1 diabetes that historically has been excluded from clinical trials. Identifying the highest-risk individuals (in the paediatric, adolescent and adult populations, among others) to aim for more intensive contemporary CVD risk lowering agents (such as glucose, lipid and blood pressure lowering), and other, ideally personalised, cardioprotective adjunct therapies could help reduce the burden of CVD and contribute to improving outcomes in type 1 diabetes;
- Data collection in patient populations with other (genetically defined) predispositions to CVD and HF, that historically have been excluded from clinical trials. Identifying the highest-risk individuals could contribute to improving the outcomes in people with obesity, type 2 diabetes or (genetic) predisposition to CVD/HF.
The overall aim of the project is to provide tools for the earlier diagnosis of atherosclerosis and heart failure as well as earlier identification of patients at risk. This includes biomarker or predictive algorithms to assess changes in risk and stratify patients according to individual responses to therapeutic intervention. Currently, patient data from various sources such as devices, intake forms, and diagnostic and exploratory tests are not integrated or monitored to give a complete understanding of the patient’s disease state. Integration of these data sets, e.g. by a federated database, and its accessibility to healthcare providers and researchers will provide better understanding to help detect, monitor, and treat ASCVD and HF. The selected project should clearly outline their approach for data capture, storage and sharing, for instance data federation, or an open, centralised database architecture. The proposed data management strategy should be sustainable, seek synergies with other relevant projects, and align with the FAIR principles1. To fulfil this aim, the selected project should:
- Increase our understanding of the initial hallmarks of disease, which will allow for a better identification of individuals at risk for ASCVD and HF at a young age, and the creation of a clinical risk profile based on a multi-omic approach (e.g. genetic markers, transcriptomics, proteomics, and in depth multimodality imaging data) in adolescents who have either genetic and/or enrichment of specific endpoint associated risk factors (obesity, chronic kidney disease, type 1 diabetes, type 2 diabetes, genetic preponderance for HF and increased atherosclerosis);
- Generate and validate a risk model better than currently used risk engines such as SCORE, by evaluating whether and to which extent risk factors identified in large prospective CVD primary prevention cohorts are predictive in a secondary prevention setting. The data from surrogate markers such as imaging, electronic health records (EHR), and predictive markers (plasma based multi- omics), as well as data from wearables, will generate a more refined risk engine;
- Outline the extent to which social, ethical, and regulatory implications can be considered and quantified in the new risk models and gauge the potential additive value of data generated by wearable devices in current healthcare systems. Outline the extent to which regional and legal issues have an impact, and what models and methodologies can be used to examine this. Moreover, as the risk-benefit of wearable derived data will be ascertained in individuals who are likely to be frontrunners in the adoption (i.e. people with type 1 diabetes and people with a (genetic) risk for premature atherosclerosis and/or HF), the project should include behavioural elements to be analysed to provide suggestions to increase adoption in other populations;
- Model short- and long-term economic and public health morbidity and mortality benefit/risk assessments of therapeutic intervention in people at risk with the new risk models to prevent or delay onset of CVDs;
- Develop a decision tool that will allow a physician to select the intervention to best address ASCVD and HF in an individual patient. The tool will provide a risk-benefit profile, helping the physician and the patient in a decision-making process, integrating also patient reported outcome and experience measure (PROMs and PREMs) data;
- Explore possibilities for novel methods of clinical development and trial execution. Based on learnings about risk prediction and pathophysiological modelling, novel surrogate endpoints may be considered for a risk-based cardiovascular outcome trial approach. The project generated from this topic could provide an exploratory and interactive platform to discuss the validity of novel methods of evidence generation, such as the use of data from wearable devices. The project should pave the way to transform the rather static phase 3 clinical trial approach into a more agile (more inclusive/enriched patient population, faster, cost-effective etc.) and sustainable part of clinical development. Specifically, the project should engage in the Regulatory Science Research Needs initiative, launched by the European Medicines Agency (EMA), assessing the utility of real-world healthcare data to improve the quality of randomised controlled trial simulations (H2.3.3). During the COVID-19 pandemic, the world has experienced a transition to virtual and remote care as more and more patients connect with their health care teams online. This presents an enormous opportunity and benefits for patients. A pathway forward could be to through use of real-world evidence (RWE) data to address sex, ethnicity and race disparities in cardiovascular outcome trials and better promote CV management.