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Horizon Europe
1 phase
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.
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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:
The AI has drafted potential core elements based on the call analysis. To start building your project proposal structure, select the elements that resonate with your consortium's concept. You can refine and rewrite them fully once your project workspace is created.
Developing medicines for small patient populations is inherently challenging, leading to prolonged development cycles and high costs due to difficulties in recruiting sufficient patients for traditional clinical trials.
Conducting extensive clinical trials in paediatric and rare disease populations raises significant ethical concerns and practical difficulties, necessitating alternative, less invasive, yet robust methods for evidence generation.
Despite the potential of modelling and simulation, there is a current gap in widely accepted, validated, and standardised computational models and clear regulatory guidelines for their integration into drug development and approval processes.
The vast potential of real-world data to inform drug development and regulatory decisions for small populations remains largely untapped due to challenges in data integration, analysis, and the lack of validated in-silico tools to assess its actionable use.
Companies involved in the research, development, and manufacturing of medicines for rare diseases and paediatric populations, seeking to improve efficiency and regulatory success.
Bodies responsible for the scientific advice, marketing authorisation, and pharmacovigilance of medicinal products, requiring robust tools and guidance for assessment.
Academics and industry professionals designing and conducting clinical trials, particularly those in small and vulnerable patient populations, seeking to improve trial design and statistical robustness.
Experts in quantitative systems pharmacology, disease mechanistic models, PBPK, Bayesian modelling, and AI algorithms, contributing to the development and validation of in-silico tools.
Groups representing patients with rare and/or paediatric diseases, whose involvement ensures that new solutions address real-world needs and improve access to effective treatments.
Individuals who will ultimately benefit from faster, safer, and more effective development and availability of medicines tailored to their specific needs.
Professionals and systems that will integrate and deliver new, approved orphan and paediatric medicines, benefiting from improved treatment options and health outcomes.
Small and medium-sized enterprises that can leverage or contribute to the developed modelling and simulation tools, fostering innovation and competitiveness in the EU health sector.
To develop, calibrate, and optimise mature computational models, including hybrid solutions, for assessing their utility in designing innovative clinical trials for small populations and supporting regulatory processes for orphan and paediatric medicines. This includes ensuring statistical robustness and accurate prediction of therapeutic and dose effects, considering patient-specific factors.
To rigorously benchmark diverse computational models by showcasing their simulation performance in virtual patient cohorts and demonstrating alignment with actual clinical trial data. This objective aims to assess the models' performance and credibility for specific regulatory purposes, using well-justified rare and/or paediatric disease use cases (excluding cancer and infectious diseases).
To define clear criteria for the performance and credibility assessment of computational models relevant to small population clinical trials, thereby progressing their regulatory qualification and acceptability. This objective also involves developing and disseminating standards for the design, assessment, and reporting of high regulatory value M&S tools to accelerate clinical development.
By enabling more efficient clinical trial designs and reducing the need for extensive physical trials, the project will indirectly contribute to a lower environmental impact associated with pharmaceutical research and development, aligning with the European Green Deal.
The development of robust, GDPR-compliant methodologies and standards for modelling and simulation will build confidence in digital health solutions, facilitating their translation into healthcare practice while ensuring interoperability, cybersecurity, and data confidentiality.
Public authorities will benefit from better methodologies and interdisciplinary approaches to assess and value new health technologies, particularly innovative orphan and paediatric medicines, leading to more informed and timely regulatory decisions.
By accelerating the development and market access of orphan and paediatric medicines, the project will enhance the competitiveness and sustainability of the EU health industry, fostering European leadership in breakthrough health technologies and contributing to job creation, especially for SMEs.
The integration of advanced modelling and simulation tools, coupled with early engagement with patients, healthcare providers, and regulators, will streamline the health industry value chain for orphan and paediatric medicines, from needs identification to solution uptake.
The establishment and dissemination of European standards for modelling and simulation in orphan and paediatric drug development will ensure higher patient safety and quality of healthcare services, promoting effective and interoperable health innovation.
Citizens, healthcare providers, and health systems will benefit from a swift uptake of innovative orphan and paediatric medicines, leading to significant improvements in health outcomes, while the EU health industry benefits from decreased time-to-market.
By accelerating the development and regulatory processes for orphan and paediatric medicines, the project contributes to ensuring more reliable and timely access to these often critical and complex medical supplies, thereby strengthening EU health security.