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Modelling and simulation to address regulatory needs in the development of orphan and paediatric medicines
|Funding Program||Horizon Europe - Cluster 1 - Destination 6: Maintaining an innovative, sustainable and globally competitive health industry|
|date - 2nd stage||Opening|
|Call budget||€ 25,000,000.00|
|Estimated EU contribution per project||between € 4,000,000.00 and € 6,000,000.00|
|Link to the call||ec.europa.eu|
|Link to the submission||ec.europa.eu|
In its "Regulatory Science Strategy to 2025", the European Medicines Agency included specific recommendations to optimise the capabilities of modelling and simulation in the medicines development process and in particular to benefit special populations and neglected patient populations.
Orphan drug development faces numerous challenges, including low disease prevalence, patient population heterogeneity and strong presence of paediatric patient populations. Consequently, clinical trials for orphan and/or paediatric medicines are often smaller than traditional large-scale randomised ones and they require the development of efficient trial designs relevant to small.
Model-based approaches are significantly advantageous in small populations, as extrapolation tools for rationalising and increasing the statistical robustness in clinical trial designs and pharmacometric studies.
The topic will support research and innovation activities focusing on the development of diverse modelling and simulation methods, as tools for addressing some of the regulatory needs in the clinical development cycle of new orphan and paediatric medicinal products. The topic is not intended to implement new preclinical/clinical studies but to use the existing knowledge/data for assessing and optimising the performance of mature in-silico models in the regulatory context with the goal of improving the clinical trial designs for small populations. Availability of the relevant data to address the requirements of the topic is an indispensable condition that must be demonstrated at the proposal submission.
Proposals should involve national healthcare product regulatory bodies and the European Medicines Agency (EMA) in order to catalyse an effective collaboration between the researchers and the regulators. The active involvement of patient representatives is required in all phases of the research and innovation activities. Furthermore, SME(s) participation is encouraged with the aim to strengthen their scientific and technological basis.
The proposals should address all of the following activities:
The proposals should adhere to the FAIR data principles, adopt data quality standards, data integration operating procedures and GDPR-compliant data sharing/access good practices developed by the European research infrastructures, where relevant. Proposals are invited to consider adopting recommendations for in-silico models construction and validation. Data-intensive proposals, particularly those using data from patient registries, should take stock of the tools and services provided by the European Platform on Rare Disease Registration (EU RD Platform). For example, retrospective registry data are expected to be made accessible via EU RD platform, if reasonably feasible.
All projects funded under this topic are strongly encouraged to participate in networking and joint activities. These networking and joint activities could, for example, involve the participation in joint workshops, the exchange of knowledge, the development and adoption of best practices, or joint communication activities. This could also involve networking and joint activities with projects funded under other clusters and pillars of Horizon Europe, or other EU programmes. Therefore, proposals are expected to include a budget for the attendance to regular joint meetings and may consider covering the costs of any other potential joint activities without the prerequisite to detail concrete joint activities at this stage. The details of these joint activities will be defined during the grant agreement preparation phase. In this regard, the Commission may take on the role of facilitator for networking and exchanges, including with relevant stakeholders.
Applicants envisaging to include clinical studies should provide details of their clinical studies in the dedicated annex using the template provided in the submission system. See definition of clinical studies in the introduction to this work programme part.
|Expected effects and impacts|
|Regions / countries for funding||EU Member States, Overseas Countries and Territories (OCT)|
Moldova (Moldova), Albania (Shqipëria), Armenia (Հայաստան), Bosnia and Herzegovina (Bosna i Hercegovina / Босна и Херцеговина), Faeroes (Føroyar / Færøerne), Georgia (საქართველო), Island (Ísland), Israel (ישראל / إِسْرَائِيل), Kosovo (Kosova/Kosovë / Косово), Montenegro (Црна Гора), Morocco (المغرب), North Macedonia (Северна Македонија), Norway (Norge), Serbia (Srbija/Сpбија), Tunisia (تونس /Tūnis), Türkiye, Ukraine (Україна), United Kingdom
|eligible entities||EU Body, Education and training institution, International organization, Natural Person, Non-Profit Organisation (NPO) / Non-Governmental Organisation (NGO), Other, Private institution, incl. private company (private for profit), Public Body (national, regional and local; incl. EGTCs), Research Institution incl. University, Small and medium-sized enterprise (SME)|
To be eligible for funding, applicants must be established in one of the following countries:
Applications may be submitted by one or more legal entities, which may be established in a Member State, Associated Country or, in exceptional cases and if provided for in the specific call conditions, in another third country.
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.
Any legal entity, regardless of its place of establishment, including legal entities from non-associated third countries or international organisations (including international European research organisations) is eligible to participate (whether it is eligible for funding or not), provided that the conditions laid down in the Horizon Europe Regulation have been met, along with any other conditions laid down in the specific call topic.
A ‘legal entity’ means any natural or legal person created and recognised as such under national law, EU law or international law, which has legal personality and which may, acting in its own name, exercise rights and be subject to obligations, or an entity without legal personality.
Digitalisation, Digital Society, ICT,
Health, Social Services, Sports
|Relevance for EU Macro-Region||EUSAIR - EU Strategy for the Adriatic and Ionian Region, EUSALP - EU Strategy for the Alpine Space, EUSBSR - EU Strategy for the Baltic Sea Region, EUSDR - EU Strategy for the Danube Region|
|UN Sustainable Development Goals (UN-SDGs)|
All proposals must be submitted electronically via the Funders & Tenders Portal electronic submission system (accessible via the topic page in the Search Funding & Tenders section). Paper submissions are NOT possible.
Proposals must be complete and contain all parts and mandatory annexes and supporting documents, e.g. plan for the exploitation and dissemination of the results including communication activities, etc.
The application form will have two parts:
Annexes and supporting documents will be directly available in the submission system and must be uploaded as PDF files (or other formats allowed by the system).
The limit for a full application (Part B) is 45 pages.
The award criteria are described in General Annex D. The following exceptions apply: The thresholds for each criterion will be 4 (Excellence), 4 (Impact) and 3 (Implementation). The cumulative threshold will be 12.
The rules for the legal and financial set-up of the grant agreements are described in General Annex G. The following exceptions apply: In order to optimise synergies and increase the impact of the projects, all projects selected for funding from this topic will form a cluster and be required to participate in common networking and joint activities (and in determining modalities for their implementation and the specific responsibilities of projects). Depending on the scope of proposals selected for funding, these activities may include:
|Call documents|| HE-Work Programme 2023-2024, Cluster 1, Destination 6 (452kB)
|Contact||National Contact Points for Horizon Europe |
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