Project description
Innovating drug development for paediatric and orphan diseases
Limited patient populations and ethical constraints delay access to life-saving treatments for paediatric and orphan diseases. Traditional methods struggle to provide reliable assessments of drug efficacy and safety. In this context, the EU-funded ERAMET project integrates modelling and simulation methods to establish credibility within regulatory procedures. Its transparent ecosystem connects questions, data, and methods, facilitating adoption of advanced techniques like AI and hybrid approaches. Three pillars underpin ERAMET: a versatile repository, high-quality standards for data and methods, and an AI-driven platform for automated analysis and credibility assessment. Five use-cases, spanning rare diseases like ataxia and neuromuscular disorders, demonstrate ERAMET’s potential, aiming for regulatory approval of validated tools. Training initiatives will ensure stakeholders embrace this transformative paradigm.
Objective
ERAMET will provide an integrated approach for developers and regulators’ decision-making for paediatric and orphan drugs, centred on the drug development questions. This will constitute a transparent ecosystem for drug development and assessment, that will facilitate the adoption of modelling and simulation (M&S) methods and related data types (including real word data such as registries and electronic healthcare data).
The overall objective of ERAMET is to provide and implement a framework for establishing the credibility of M&S methods and related results as sources of evidence within regulatory procedures.
The ecosystem proposed by ERAMET will be based on three pillars:
(1) A repository connecting questions, data and methods.
(2) The development and validation of high-quality standards for data and analytical methods (including M&S and hybrid approaches). These will cover computational M&S, digital twins, AI, hybrid approaches, standard statistics and pharmacometrics, as analytical methods and alternative data types and sources such as RWD, eHealth data, registries, historical regulatory submissions, scientific and (non)clinical trials).
(3) An AI-based platform that will automate and optimise the data collection, formatting and modelling and simulation analysis and implement the credibility assessment.
As part of ERAMET, the ecosystem will be applied to five use-cases including paediatric extrapolation and characterisation of drug benefit/risk in 4 groups of rare diseases, namely ataxia, transfusion dependent haemoglobinopathies, bronchopulmonary dysplasia, and paediatric tuberculosis.
Each of the use-case is planned to lead to submission and regulatory approval of at least one validated M&S tool via the EMA qualification procedure.
Training will be proposed to familiarise regulatory assessors, drug developers and clinical researchers with this new approach.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- medical and health sciences health sciences health care services eHealth
- medical and health sciences clinical medicine pneumology tuberculosis
- natural sciences biological sciences ecology ecosystems
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.2.1 - Health
MAIN PROGRAMME
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HORIZON.2.1.3 - Non-Communicable and Rare Diseases
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HORIZON.2.1.5 - Tools, Technologies and Digital Solutions for Health and Care, including personalised medicine
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-RIA - HORIZON Research and Innovation Actions
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) HORIZON-HLTH-2023-IND-06
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
5000 NAMUR
Belgium
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.