A study published in Frontiers in Medicine examined the development and approval of paediatric in the European Union (EU) and the United States (US) over the period 2007- 2024. Among the authors are our researchers Viviana Giannuzzi and Adriana Ceci.

The study shows that in recent years, particularly after 2018, the number of approved treatments for childhood cancers has increased. These are mainly molecular targeted therapies and immunotherapies, which are gradually replacing traditional chemotherapy. However, the pace of innovation has not been the same in both regions: the United States has achieved faster results, while progress in Europe has been slower (35 new oncology drugs and 49 new paediatric indications approved in the EU, compared to 53 drugs and 79 new indications in the US).

Interestingly, the analysis shows   in approved indications in the EU for very rare paediatric cancers, like melanoma and thyroid cancer.

The article also explores whether paediatric oncology medicines were developed from an existing adult indication (adult-driven) or to address a paediatric-specific oncology indication: the percentage of non-adult-driven indications increases significantly in the EU after 2018, along with the number of PIPs approved to develop paediatric- only indications.

The analysis highlights that regulatory frameworks play a decisive role in shaping paediatric oncology drugs development: in the US, obligations and incentives have accelerated paediatric research & development programmes, mainly coming from the RACE Act enacted in 2017.

In the EU specific obligations and incentives for paediatric oncology developments are not currently in place. In this context, the ongoing revision of the EU pharma legislation , has proposed some measures, like the obligation to develop paediatric medicines on the basis of the mechanism of action of the drugs.

In this scenario, the study reveals that a gap between the EU and the US remains evident, and therefore provides an important contribution to the debate on research policies and strategies aimed at ensuring that all children with cancer benefit from well-studied  innovative and effective therapies.

This paper represents a successful model for evaluating to which extent regulatory frameworks improved the availability of new medicines

Read the full article here.