For as long as I can remember in my pharma career, there have always been challenges in enabling development of paediatric medicines. However, significant steps (as part of the Paediatric Action Plan) have been made by European Union (EU) regulators in recent years to improve the situation, which we shall now explore.


Enhancing the efficiency of regulatory processes: to support companies in forming their Paediatric Investigation Plans (PIPs) a “stepwise PIP” framework has been created (pilot phase launched 6th February 2023). This agreement may allow acceptance of a partial development programme, pending the creation of a final PIP once data becomes available over time. In certain cases, it will be possible to accept PIPs for innovative medicines where vital information is not yet in existence.


Increasing attention on the areas of unmet clinical need: the last few years have seen much effort put into better-identifying and raising awareness of the areas where medicines for children are especially required. For instance, a full spectrum of involved parties, ranging from physicians to paediatric patients and from regulators to developers and other stakeholders, has been included in policy discussions to agree the needs of children with inflammatory bowel disease, and of children with cancer. Key knowledge from these discussions is incorporated by the European Medicines Agency (EMA) when reviewing PIPs for novel treatments.


Better alignment of data requirements between decision-makers: EMA is working ever more closely with global partners to enable the compatibility of paediatric requirements between regulators.


I look forward with hope in anticipation of seeing the benefits that these positive steps bring to the development of children’s medicines in the future.