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Ecosystem for Rapid Adoption of Modelling and Simulation Methods in Orphan and Paediatric Drug Development

ERAMET aims to create an integrated approach for decision-making in the development of paediatric and orphan drugs. This ecosystem will facilitate the adoption of modelling and simulation (M&S) methods, incorporating real-world data such as registries and electronic healthcare data, to aid in drug development and regulatory assessment. The project will establish a framework to validate M&S methods and their results as credible sources of evidence in regulatory procedures. ERAMET's ecosystem is based on three pillars: a repository connecting questions, data, and methods; high-quality standards for data and analytical methods; and an AI-based platform to automate data collection and analysis. The ecosystem will be tested through five use-cases involving paediatric extrapolation and drug benefit/risk characterization in four rare diseases. Training will be provided to familiarize regulatory assessors, drug developers, and clinical researchers with this new approach.

Feedback Overview:

The ERAMET project is highly innovative and addresses a critical need in the pharmaceutical industry for paediatric and orphan drug development. To enhance its market fit, the project could benefit from partnerships with regulatory bodies and pharmaceutical companies to ensure wide adoption. Additionally, integrating patient and clinician feedback could further validate the practical applications of the ecosystem.

Market Competitors:

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Chief Scientific Officer

Expert in drug development, clinical trials, and regulatory affairs.

How can the credibility of M&S methods be ensured in regulatory procedures?

By establishing rigorous validation protocols and collaborating with regulatory bodies to align the methods with existing standards and guidelines.

What are the potential challenges in integrating real-world data into the ecosystem?

Challenges include data quality, standardization, and the need for robust data privacy and security measures.

How can the ecosystem be scaled to accommodate different types of drugs and diseases?

By creating a flexible and modular platform that can be easily adapted to various drug development scenarios and incorporating feedback from diverse stakeholders.

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