EFGCP Better Medicines for Children Conference 2025
Innovation in Paediatric Drug Development as a Driver for Change
The EFGCP “Better Medicines for Children” Conference is an annual flagship event providing a unique opportunity to share best practice, reflect on what has happened since we last met in October 2024, and discuss paediatric updates with a truly global outreach. Despite an ever changing and challenging environment, we believe we have been able to organise a dynamic, innovative, and highly engaging conference for advancing paediatric research and innovation across Europe and beyond.
This year will be a hybrid conference but we strongly encourage you to attend in-person in Amsterdam at the NH Amsterdam Schiphol Airport Hotel as this would be a great opportunity to connect with key stakeholders in paediatric drug development and create the all important relationships with other delegates.
The conference will focus on “Innovation in paediatric drug development as a driver for change”. At the heart of the Conference lies a resolute commitment to patient-centricity—placing the voices, needs, and lived experiences of children and their families at the forefront of paediatric research and innovation. The conference will bring together distinguished speakers from all around the world and relevant stakeholders and experts to resolve the most urgent and complex challenges in paediatric drug development.
Day One, will start with a Keynote speech from a parent on the impact of trial participation, experience and solutions for study success. As the conference continues there will be the opportunity to discuss novel approaches to drug development in paediatric chronic kidney disease as well as key opportunities in streamlining global clinical trials. We will continue to unpack the implications of evolving EU legislation with a discussion amongst all stakeholders affected by it. Finally, we will explore the integration of artificial intelligence (AI) and machine learning (ML) in paediatric data ecosystems.
Active engagement of conference delegates will be welcomed in Breakout Sessions to share learnings, discuss challenges, and identify solutions on what could be done to optimise children’s access to new medicines, with a focus on these important topics:
1. Pediatric Extrapolation.
2. Innovative approaches in clinical trials for small populations.
3. Paediatric medical devices.
At the end of Day 1, there will be the opportunity for an EMA PDCO member to share their views and perspective on paediatric drug development and also on the EMA Paediatric Committee (PCDO) work.
Day two will start with the feedback and key deliverables from the Breakout Sessions. Then it will be the opportunity to engage on the status of Europe as a competitive/innovative region for paediatric research with a panel of experts including regulators from various regions, such as Europe, the UK, Canada and Japan. Further to this session, there will be two sessions that will focus on Optimising the Clinical Development Path of Orphan Medicines and also on Drug Repurposing, highlighting the outcomes of several projects and real-life examples that enhance delegates expertise in these fields. These sessions will be then complemented by Global Regulatory Alignment and the critical role of these partnerships in derisking drug development. Finally, as a conference first we are proud to present New voices in Paediatric Research with a focus on real world examples of successful paediatric drug development.
The Programme Committee looks forward to welcoming you all at the conference for a global educational and networking event that is a unique opportunity to learn from one another, to meet and interact with experienced colleagues and experts from all over of the world.
Early Bird Fees available until June 30, 2025!
Programme
09:00 |
Registration and welcome coffee |
10:00 |
Introduction to the Conference
Begonya Nafria-Escalera, Barcelona Children’s Hospital & eYPAGnet
Martine Dehlinger-Kremer, ICON & EUCROF
|
10:15 |
Keynote – Patient |
10:45 |
Session 1: Challenges and opportunities for paediatric drug development in chronic kidney disease : Panel discussion
Franz Schaefer, Heidelberg University
Jennifer Mckenzie, Boehringer-Ingelheim
Sebastian Honoré, Cure Lowe Foundation
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11:45 |
Session 2: Actions to streamline and facilitate paediatric clinical trial
Bethan Phillips, Cardiff and Vale UHB - Children and Young adult research unit
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12:45 |
Lunch |
13:45 |
Session 3: EU legislation updates
Gilles Vassal, Gustave Roussy Institute
Ralph Bax, EMA
|
14:45 |
Breakout sessions: |
1. Pediatric Extrapolation
Dominik Karres, EMA
Sabine Fuerst-Recktenwald, Novartis
|
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2. Innovative approaches in clinical trials for small populations Moderator(s): Donato Bonifazi, TEDDY Solange Corriol-Rohou, Consultant |
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3. Paediatric medical devices |
|
16:30 |
Coffee Break |
17:00 |
Session 4: The role of AI within the Paediatric Data Ecosystem
Alberto Tozzi, Ospedale Pediatrico Bambino Gesù
Jari Petaja, HUS for PHEMS Initiative
|
18:00 |
Reflection from the PDCO Chair |
18:30 |
Closing Remarks End of Day 1 |
19:00 |
Get-together and Networking |
08:00 |
Welcome Coffee |
08:30 |
Welcome and Debrief from Day 1 |
08:40 |
Feedback from breakout sessions |
09:15 |
Session 5: EU as a competitive/innovative region for paediatric research |
10:15 |
Coffee Break |
10:35 |
Session 6: Optimising the clinical development path of orphan medicine |
11:35 |
Session 7: Paediatric Drug repurposing |
12:00 |
Lunch |
13:30 |
Session 8: Global regulatory alignment
Fahimeda Ali, MHRA
Franca Ligas, EMA
Ming Su, Health Canada
|
14:30 |
Session 9: New voices in paediatric research |
15:30 |
End of Day 2 - Conclusion & Farewell |
15:45 |
END OF CONFERENCE |