A question-centric approach to understanding the role of modelling and simulation in medicine development

The development of monoclonal antibodies for moderate-to-severe asthma has introduced new opportunities for more targeted treatments, while also raising complex questions around evidence generation and regulatory assessment. As these therapies are increasingly developed for diverse patient populations, including children, innovative methodologies are needed to support the integration of available data and optimise decision-making throughout development.

Against this background, the publication “Use of Modeling and Simulation to Inform the Development of Monoclonal Antibodies to Treat Moderate-to-Severe Asthma: A Retrospective Review of EMA Centralized Procedure From 2014 to 2024” examines how modelling and simulation have been used in the development and regulatory assessment of monoclonal antibodies for asthma. More specifically, the study reviews the marketing authorisation applications of five approved monoclonal antibodies and evaluates the role of modelling and simulation in addressing key regulatory questions related to pharmacokinetics, efficacy, and safety.  

Using a question-centric approach, the authors have developed a repository of regulatory questions, data, and methods used across the five marketing authorisation applications. The analysis identifies 190 regulatory questions, with modelling and simulation methods used to address 25% of them, mainly in relation to pharmacokinetic characterisation and, to a lesser extent, efficacy assessment. The study also applies credibility assessments based on the ICH M15 framework to evaluate the use of model-informed evidence and benchmark these approaches against traditional methods.

Strengthening the assessment of model-informed evidence is essential to supporting the wider adoption of innovative methodologies in medicine development. The approach developed in this study contributes to this objective by creating a repository that links regulatory questions with the data and analytical methods used to address them, promoting greater transparency and consistency in the evaluation of modelling and simulation evidence. This perspective aligns with ERAMET’s work to advance frameworks that support the assessment, benchmarking, and responsible adoption of innovative methodologies in paediatric and orphan drug development. By bringing together regulatory questions, evidence sources, and analytical approaches, ERAMET aims to facilitate a more structured evaluation of model-informed evidence and support collaboration between researchers, regulators, and stakeholders involved in medicine development.

Learn more about the approach here!