Supporting medicines for children in the EU: Clinical trials and RWE
31 Aug 2018

The European Medicines Agency (EMA) recently released a report into the differences of medications and medicines on children and infants. The Paediatric Regulation, which was introduced in 2007 and requires manufacturers to consider children’s therapeutic needs when researching new medicines. The regulation has raised awareness of the need to study medicines in children, and paediatric research is now standard practice in the development of medicines. The responsible organisation for this is the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA).

The organisation’s main aim is to facilitate studies focusing on children and medicines, over the past ten years the organisation has proven to be effective in this, as more clinical trials for children have occurred. Between 2007 and 2017, more than 200 medicines for children were authorised. This accounts for 25% of all new medicines authorised for marketing in the EU. The most popular therapeutic areas were in infectious diseases and rheumatology, some areas lacking behind in clinical trials were oncology and neonatology. The EMA has stated that it will take steps to improve the situation in these areas.

The FDA, EMA’s sister organisation, has begun encouraging the use of Real-World Evidence (RWE) in the assessments for drugs and medicines, it would be interesting if the EMA would also do this. Taking the area of children’s health, there are a number of rich Real-World Data (RWD) sources that could be used to further this field in the European setting. This is especially true for the area of Childhood oncology, with a number of these RWDs being of high-quality and collecting variables covering different treatment areas that would be of interest to EMA.

We have highlighted some of these childhood RWDs in a previous blog post about Norway. To read the full  EMA report, click here