EMA-Meeting on Pediatric Research

Written by Miguel R. Molina, member of OIFE’s Medical Advisory Board

Miguel R. Molina from our Medical Advisory Board represented OIFE at the meeting of the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) in Amsterdam on November 23rd 2023.

Miguel Molina in front of EMA buildingThe European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Enpr-EMA enables networking and collaboration with members from within and outside the European Union (EU), including academia and the pharmaceutical industry. It acts as a platform for sharing good practices as well as a pan-European voice for promoting research into medicines for children. At this present moment there are 21 Networks working in the Enpr-EMA with 15 European countries represented in national networks also including Japan, Switzerland and the United States. November 23rd the network hosted a multistakeholder methodology workshop in Amsterdam.

Here are some of the take-away messages from Miguel:

Off-label is not always off-evidence. Half of all paediatric medicine prescriptions in the EU are off-label. Today legislation mandates clinical trials for those medicines that are still under patent protection. The proposal of this working group is trying to include more paediatric data. Steps from off-label to effective and safe therapies can be:
– Step 1: A decision model for evidence generation.
– Step 2: How to prioritize international consensus?
– Step 3: Research agenda by subspeciality
– Step 4: Generate the evidence
– Step 5: Implement in practice
– Step 6: Market authorization

Other topics covered by the Enpr-EMA working groups:
• Survey of paediatric clinical trial site requirements
• Survey done on EU Research Nurses
• Paediatric clinical trial sites incl. workshop co-organised with Conect4children (C4C)
• Language discrimination and cross-border access to paediatric clinical trials

Another topic was the decentralised elements in clinical trials a paediatric perspective. We need to take advantage of technological progress to make clinical trials more accessible and participation more convenient for trial participants. This includes trials performed more regionally or locally, more use of wearables, home nursing, patient reported outcomes, new forms of digital consent, administration of medicinal products at home etc.
Decentralisation has huge potential for minimizing logistical hurdles affecting families. Indirect costs are significantly higher for families with children enrolled in a trial compared to adults. Because “A sick child is a sick family”.

Graphic showing key political objectives

This second part of the session was about the Good Clinical Practice – Scientific Guideline ICH E6 and the Accelerating Clinical Trials Project. Then there was a talk and discussion about the new EU pharmaceutical legislation and its impact on paediatric and orphan medicine development. The consultation about the new regulation will probably be in the EU parliament in 2025. And probably the new legislation could be implemented in 2027, but this is not for certain.

The third session was about highlights in 2022-2023 of Enpr-EMA and its member networksGraphic showing a ladder and steps about participation and non-participation incl. the Conect4Children journey. The C4C academy has shown to be very useful for students and researchers. You can find more information about this here.

Session four was about patient involvement which included the following topics:

• Patient recruitment and retention.
• Benefits of involving patients and young people (YPAGs) in paediatric clinical research. Through the EYPAGNET 14 countries and more than 30 groups are working with patient involvement of young people.
• Improvement of paediatric health, medicine research, and innovation by sharing children’s


This article was first published in OIFE Magazine 4-2023. 

An official report from the EMA workshop has now been published. You can download it here.

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