Loading…
Strategic Analysis
This call seeks to accelerate the development of orphan and paediatric medicines by advancing and validating computational modelling and simulation (M&S) tools. A winning proposal must demonstrate a robust, multidisciplinary approach to developing, calibrating, and benchmarking these in-silico models, ensuring their fitness for regulatory purposes and their capability to enhance clinical trial efficiency and patient safety in small populations. The focus should be on practical application, regulatory acceptance, and clear pathways to impact for patients, industry, and public health authorities.
TRL 2 → 6
Based on programme defaults
Establish a multidisciplinary approach for assessing the utility of mature computational models, as tools for supporting the optimal design of innovative clinical trials for small populations and as fit-for-purpose solutions for enabling the regulatory scientific advice and marketing authorisation assessment of orphan and/or paediatric medicines, including their pharmacovigilance follow-up.
Calibrate and optimise mature computational models for enhancing their clinical performance, by using relevant sources of patient data (e.g. natural history and observational clinical studies, medical records, registries, pharmacovigilance and longitudinal studies etc.). The models should include a variety of modelling methods and in particular hybrid solutions linking quantitative mechanistic modelling with advanced statistical modelling (e.g. quantitative systems pharmacology, disease mechanistic models, physiology-based pharmacodynamic/pharmacokinetic models, Bayesian modelling, artificial intelligence algorithms etc.).
Assess validated in-silico models for their capability to increase the statistical robustness, improve the risk/benefit assessment in small population clinical trials, and for their accuracy to predict and extrapolate the therapeutic and dose effects, taking into account the patient’s genotypes/phenotypes, disease characteristics/stage variables and/or clinical/surrogate endpoints for delivering robust evidence of safety and efficacy of the orphan and paediatric medicines under study. The assessment of the in-silico models should be demonstrated in use cases representing well-justified group(s) of rare and/or paediatric diseases with commonalities, such as shared molecular denominators/disease pathways within the same and/or across different medical areas, excluding cancer and infectious diseases.
Benchmark of diverse computational models by showcasing their simulation performance in virtual patient cohorts and by demonstrating that the models’ synthetic data estimates match to actual clinical trial data. This should lead to an assessment of the performance and credibility of a model simulation in the context of their specific use for regulatory purposes. Benchmark studies should be performed in the use cases mentioned above. Availability of clinical trials data and other relevant data is an indispensable requirement that must be demonstrated at the proposal submission.
Set-up the criteria for the performance and credibility assessment of any relevant computational models for small population clinical trials to progress on their regulatory qualification and acceptability. Further develop and disseminate standards for the design, performance assessment and reporting of modelling and simulation tools with an emphasis on those of high regulatory value for accelerating the clinical development of orphan and paediatric medicinal products.
Developers and regulators have access to robust modelling and simulation tools to accelerate the effective development of orphan and/or paediatric medicinal products.
Clinical researchers, developers and regulators use accurate computational models to improve the statistical robustness in clinical trials intended for small populations and guide cost-effective clinical trial designs.
Clinical researchers and regulators have access to accurate in-silico tools for assessing the actionable use of real-world data and for successfully estimating the risk-benefit effects in clinical trials for small populations.
Regulators develop guidance for the use of validated computational models to support a robust extrapolation framework and facilitate the safety and efficacy assessment in the process of regulatory appraisal of orphan and/or paediatric medicinal products.
Facilitating the production of pharmaceuticals in compliance with the objectives of the European Green Deal.
Developing methodologies, guidelines and standards, assessment studies, and structuring activities adapted to digital solutions and interventions for GDPR compliant translation into health care practice, including inter-operability, cyber-security and data confidentiality.
Supporting public authorities with better methodologies and interdisciplinary approaches to assess and value new health technologies and interventions.
Health industry in the EU is more competitive and sustainable, assuring European leadership in breakthrough health technologies and open strategic autonomy in essential medical supplies and digital technologies, contributing to job creation and economic growth, in particular with small- and medium-sized enterprises (SMEs).
Health industry is working more efficiently along the value chain from the identification of needs to the scale-up and take-up of solutions at national, regional or local level, including through early engagement with patients, health care providers, health authorities and regulators ensuring suitability and acceptance of solutions.
European standards, including for operations involving health data, ensure patient safety and quality of healthcare services as well as effectiveness and interoperability of health innovation and productivity of innovators.
Citizens, health care providers and health systems benefit from a swift uptake of innovative health technologies and services offering significant improvements in health outcomes, while health industry in the EU benefits from decreased time-to-market.
Health security in the EU benefits from reliable access to key manufacturing capacity, including timely provision of essential medical supplies of particularly complex or critical supply and distribution chains, such as regards vaccines or medical radioisotopes.
European Green Deal
lowThe European Green Deal is the EU's overarching growth strategy to make Europe climate-neutral by 2050. It encompasses a wide range of policy initiatives across various sectors, including energy, industry, transport, and agriculture, aiming for a just and inclusive transition. It sets ambitious targets for emissions reduction, renewable energy, and energy efficiency.
Proposals must clearly articulate how they contribute to the overarching goals of the European Green Deal, such as achieving climate neutrality, enhancing energy efficiency, promoting sustainable industrial practices, or fostering the deployment of clean energy technologies. Demonstrating a positive environmental impact and alignment with the 'do no significant harm' principle is crucial.
Specific requirements
US entities eligible for Union funding
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.
">
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.
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.
6. Legal and financial set-up of the grants: described in Annex G of the Work Programme General Annexes.
7. Specific conditions: described in the specific topic of the Work Programme.
Call documents:
Standard application form (HE RIA, IA) - call-specific application form is available in the Submission System Standard evaluation form (HE RIA, IA) HE General MGA v1.0 Information on clinical studies (HE)
HE Main Work Programme 2023–2024 – 1. General Introduction HE Main Work Programme 2023–2024 – 4. Health HE Main Work Programme 2023–2024 – 13. General Annexes HE Programme Guide HE Framework Programme and Rules for Participation Regulation 2021/695 HE Specific Programme Decision 2021/764 EU Financial Regulation Rules for Legal Entity Validation, LEAR Appointment and Financial Capacity Assessment EU Grants AGA — Annotated Model Grant Agreement Funding & Tenders Portal Online Manual Funding & Tenders Portal Terms and Conditions Funding & Tenders Portal Privacy Statement
Evaluators will prioritize:
Everything the call asks for, seen from the call's point of view. Each line shows what answers it, and which partner carries it.
This matrix lists everything the call asks for: outcomes, impacts, scope, the requirements buried in the call text, and policy alignment. Sign up free and GrantForge tracks each line against the concept you build.
| Requirement | Covered by | Carried | Status |
|---|---|---|---|
| Scope activities | |||
| SC1Establish a multidisciplinary approach for assessing the utility of mature computational models, as tools for supporting the optimal design of innovative clinical trials for small populations and as fit-for-purpose solutions for enabling the regulatory scientific advice and marketing authorisation assessment of orphan and/or paediatric medicines, including their pharmacovigilance follow-up. | · | · | Sign up to track |
| SC2Calibrate and optimise mature computational models for enhancing their clinical performance, by using relevant sources of patient data (e.g. natural history and observational clinical studies, medical records, registries, pharmacovigilance and longitudinal studies etc.). The models should include a variety of modelling methods and in particular hybrid solutions linking quantitative mechanistic modelling with advanced statistical modelling (e.g. quantitative systems pharmacology, disease mechanistic models, physiology-based pharmacodynamic/pharmacokinetic models, Bayesian modelling, artificial intelligence algorithms etc.). | · | · | Sign up to track |
| SC3Assess validated in-silico models for their capability to increase the statistical robustness, improve the risk/benefit assessment in small population clinical trials, and for their accuracy to predict and extrapolate the therapeutic and dose effects, taking into account the patient’s genotypes/phenotypes, disease characteristics/stage variables and/or clinical/surrogate endpoints for delivering robust evidence of safety and efficacy of the orphan and paediatric medicines under study. The assessment of the in-silico models should be demonstrated in use cases representing well-justified group(s) of rare and/or paediatric diseases with commonalities, such as shared molecular denominators/disease pathways within the same and/or across different medical areas, excluding cancer and infectious diseases. | · | · | Sign up to track |
| SC4Benchmark of diverse computational models by showcasing their simulation performance in virtual patient cohorts and by demonstrating that the models’ synthetic data estimates match to actual clinical trial data. This should lead to an assessment of the performance and credibility of a model simulation in the context of their specific use for regulatory purposes. Benchmark studies should be performed in the use cases mentioned above. Availability of clinical trials data and other relevant data is an indispensable requirement that must be demonstrated at the proposal submission. | · | · | Sign up to track |
| SC5Set-up the criteria for the performance and credibility assessment of any relevant computational models for small population clinical trials to progress on their regulatory qualification and acceptability. Further develop and disseminate standards for the design, performance assessment and reporting of modelling and simulation tools with an emphasis on those of high regulatory value for accelerating the clinical development of orphan and paediatric medicinal products. | · | · | Sign up to track |
| Expected outcomes | |||
| EO1Developers and regulators have access to robust modelling and simulation tools to accelerate the effective development of orphan and/or paediatric medicinal products. | · | · | Sign up to track |
| EO2Clinical researchers, developers and regulators use accurate computational models to improve the statistical robustness in clinical trials intended for small populations and guide cost-effective clinical trial designs. | · | · | Sign up to track |
| EO3Clinical researchers and regulators have access to accurate in-silico tools for assessing the actionable use of real-world data and for successfully estimating the risk-benefit effects in clinical trials for small populations. | · | · | Sign up to track |
| EO4Regulators develop guidance for the use of validated computational models to support a robust extrapolation framework and facilitate the safety and efficacy assessment in the process of regulatory appraisal of orphan and/or paediatric medicinal products. | · | · | Sign up to track |
| Other requirements | |||
| REQ1Involvement of regulatory bodies and the European Medicines AgencyProposals should involve national healthcare product regulatory bodies and the European Medicines Agency (EMA) to catalyse effective collaboration between researchers and regulators. | · | · | Sign up to track |
| REQ2Active involvement of patient representatives across all phasesThe active involvement of patient representatives is required in all phases of the research and innovation activities. | · | · | Sign up to track |
| REQ3Participation of small and medium-sized enterprises (SMEs)SME participation is encouraged with the aim to strengthen their scientific and technological basis. | · | · | Sign up to track |
| REQ4Demonstration of relevant data availability at submissionAvailability of relevant patient data and clinical trials data to address the topic requirements is an indispensable condition that must be demonstrated at proposal submission. | · | · | Sign up to track |
| REQ5Adherence to FAIR data principles and research infrastructure standardsProposals should adhere to FAIR data principles, adopt data quality standards, data integration operating procedures, and GDPR-compliant data sharing/access practices developed by European research infrastructures. | · | · | Sign up to track |
| REQ6Adoption of recommendations for in-silico model construction and validationProposals are invited to consider adopting recommendations for in-silico models construction and validation, including relevant ISO guidelines under development. | · | · | Sign up to track |
| REQ7Synergies with the European Platform on Rare Disease RegistrationData-intensive proposals, particularly those using registry data, should utilise tools and services of the EU RD Platform, with retrospective registry data expected to be made accessible via the platform where reasonably feasible. | · | · | Sign up to track |
| REQ8Networking and joint activities across funded projectsProjects are strongly encouraged to participate in networking and joint activities (workshops, best-practice sharing, communication), allocating a dedicated budget for regular joint meetings. | · | · | Sign up to track |
| REQ9Clinical studies dedicated annex requirementApplicants envisaging clinical studies must provide details using the dedicated annex template provided in the submission system. | · | · | Sign up to track |
| REQ10Eligibility of United States entities for EU fundingIn recognition of the opening of US NIH programmes to European researchers, legal entities established in the United States of America are eligible to receive Union funding. | · | · | Sign up to track |
| Expected impacts | |||
| EI1Facilitating the production of pharmaceuticals in compliance with the objectives of the European Green Deal. | · | · | Sign up to track |
| EI2Developing methodologies, guidelines and standards, assessment studies, and structuring activities adapted to digital solutions and interventions for GDPR compliant translation into health care practice, including inter-operability, cyber-security and data confidentiality. | · | · | Sign up to track |
| EI3Supporting public authorities with better methodologies and interdisciplinary approaches to assess and value new health technologies and interventions. | · | · | Sign up to track |
| EI4Health industry in the EU is more competitive and sustainable, assuring European leadership in breakthrough health technologies and open strategic autonomy in essential medical supplies and digital technologies, contributing to job creation and economic growth, in particular with small- and medium-sized enterprises (SMEs). | · | · | Sign up to track |
| EI5Health industry is working more efficiently along the value chain from the identification of needs to the scale-up and take-up of solutions at national, regional or local level, including through early engagement with patients, health care providers, health authorities and regulators ensuring suitability and acceptance of solutions. | · | · | Sign up to track |
| EI6European standards, including for operations involving health data, ensure patient safety and quality of healthcare services as well as effectiveness and interoperability of health innovation and productivity of innovators. | · | · | Sign up to track |
| EI7Citizens, health care providers and health systems benefit from a swift uptake of innovative health technologies and services offering significant improvements in health outcomes, while health industry in the EU benefits from decreased time-to-market. | · | · | Sign up to track |
| EI8Health security in the EU benefits from reliable access to key manufacturing capacity, including timely provision of essential medical supplies of particularly complex or critical supply and distribution chains, such as regards vaccines or medical radioisotopes. | · | · | Sign up to track |
| Underlying policies | |||
| No underlying policies in this call. | |||
The binding rules of this call. Items marked auto are verified by GrantForge from the call and the template. The others are yours to confirm.
US entities eligible for Union funding
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.
Nothing to show for this call yet.