Mechanism-of-Action Workshop 2025

Innovative Approaches to Paediatric Research: Implementing Mechanism-of-Action Based Development Plans

Under the current paediatric framework, a Paediatric Investigation Plan (PIP) must receive approval from the European Medicines Agency (EMA) for each medicine in development, unless a waiver is granted. Waivers may be granted if the disease targeted by the medicine does not occur in children. The PIP outlines the company's strategy for product development, specifying how and when data will be generated for the use of the medicinal product in children.

The revised Paediatric Regulation is expected to restrict these waivers. Developers will need to conduct a paediatric development plan based on the product’s mechanism of action, with a potential impact on various paediatric diseases if supported by scientific evidence.

A science-based system is essential to ensure that this requirement promotes meaningful research for children in clinical trials and the broader paediatric population. Research should not impose undue burdens on developers with excessive studies to validate disease mechanisms, molecular targets or specific clinical endpoints in children. Instead, developers should target areas where new treatments for children are most needed, backed by compelling scientific evidence from the research ecosystem.

This workshop aims to gather experts to discuss how we can assist developers in creating these science-based plans and identify the necessary measures to ensure a balanced and efficient process that addresses children's needs while generating the required data within the research community.