FUTURE WEBINAR - 2025, Tuesday, April 8th - Prof Ine Rusten: "ERAMET – building a transparent ecosystem for orphan and paediatric drug development"
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Abstract:
The EU-funded ERAMET project is setting a new standard for the development of orphan and paediatric medicines. With an innovative approach that blends advanced modelling and simulation techniques with real-world data, ERAMET aims to create a transparent ecosystem to benefit both medicine developers and regulatory authorities. This will enable faster and more effective decision-making processes, ultimately speeding up the development of essential medicines for vulnerable patient groups.
ERAMET will provide an integrated approach for developers and regulators’ decision-making for paediatric and orphan drugs, centred on drug development questions. 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
(3) A knowledge-enhanced AI System that will support data collection, formatting, M&S analysis and implementation of 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 selected groups of rare diseases, namely ataxia, transfusion dependent haemoglobinopathies, bronchopulmonary dysplasia. Each of the use-cases is planned to lead to the submission and regulatory approval of at least one validated M&S tool via the EMA qualification procedure. Training will be offered in order to familiarise regulatory assessors, drug developers and clinical researchers with these new approaches.
Bio - Ine Skottheim Rusten:
It has been a few years now since Dr Ine Rusten left her government positionto pursue a more research and development oriented path. Don’t worry, shesays,she is still working within the domain and trying to solve the same old, interesting problems, just working at it from another angle as the founder of Systems Resource Lab AS (SRLAB).
SRLAB is a startup that focuses on developing and implementing tools and practices that foster learning across systems. But what do we mean when we talk about systems?
Systems are just a condensed way of saying that a whole consists of parts and that these parts are related. And that one whole can be a part of another whole. Oh my, how fantastic - this goes on forever! As for learning, it is all about discovering. You learn by discovering and when you share what you discover with another person - in your team, in your organisation, in another team in another organisation and so on, you are fostering learning across systems yourself. SRLAB is all about developing, testing and sharing ways of working, talking and nice tools to help you in your daily work.
So, a little bit more on the background: Dr Ine Rusten has worked for the regulatory authorities for 20 years with a main focus on regulatory strategies for model-informed drug development. She has contributed to implementing the use of pharmacometrics and extrapolation in paediatric developments both as a member of the European Medicines Agency´s (EMAs) Paediatric Committee and as the former chair of the EMAs Modeling and Simulation Working Party. Moreover, she has published several papers on regulatory perspectives on Model Informed Drug Development (MIDD) and model credibility and have the last years also managed initiatives on organizational learning to improve the robustness of the regulatory system.
She is now a proud partner in the ERAMET project aiming to create a transparent ecosystem for knowledge work in the domain of drug development to advance the development of essential medicines for vulnerable patient groups.