EFGCP Better Medicines for Children Conference 2025
Innovation in Paediatric Drug Development as a Driver for Change
| Event | Conference |
| Type | Hybrid |
| Language | English |
| Date(s) | 21 & 22 October |
| Day(s) | 2 Day(s) |
| Duration | 2 Days |
Patronage
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. Paediatric 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 the EMA PDCO chair 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, EUCROF
|
| 10:15 |
Keynote – Patient Perspective
Maria Baselga, Patient Representative
|
| 10:45 |
Session 1: Challenges and opportunities for paediatric drug development in chronic kidney disease : Panel discussion Moderator(s): Angeliki Siapkara, AstraZeneca Mark Turner, conect4children Stichting
Franz Schaefer, Heidelberg University
Gunter Egger, EMA
Jennifer Mckenzie, Boehringer-Ingelheim
Sara Galluzzo, PDCO Representative
Sebastian Honoré, Cure Lowe Foundation
|
| 11:45 |
Session 2: Actions to streamline and facilitate paediatric clinical trial Moderator(s): Emmanuella Amoako, Paediatric Oncologist and Yemaachi Biotech Rhian Thomas-Turner, Noah’s Ark Children’s Hospital for Wales
Bethan Phillips, Cardiff and Vale UHB - Children and Young adult research unit
Jo Dewhurst, ICON
Kathleen Scott, Novartis
Michelle Haber, FAHMS
|
| 12:45 |
Lunch |
| 13:45 |
Session 3: EU legislation updates Moderator(s): Fahimeda Ali, MHRA Mark Turner, conect4children Stichting
Gilles Vassal, Gustave Roussy Institute
Jyothsna Krishnan, EFPIA
|
| 14:45 |
Breakout sessions: |
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1. Paediatric Extrapolation Moderator(s): Angelika Joos, MSD Sabine Fuerst-Recktenwald, Novartis
Dominik Karres, EMA
Flora Musuamba, AFMPS
Oscar Della Pasqua, GSK
Stephanie Läer, Heinrich-Heine-Universität Düsseldorf
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2. Innovative approaches in clinical trials for small populations Moderator(s): Donato Bonifazi, TEDDY Solange Corriol-Rohou, Consultant
Alexandre Mejat, AFM-Telethon
Emma Hansson, Pharmatheus
Kit Roes, EMA
Ralf-Dieter Hilgers, Sigmund Freud University
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3. Paediatric medical devices Moderator(s): Angeliki Siapkara, AstraZeneca Claudio Fracasso, Hippocrates Research Srl
Lada Boecherer, Novartis
Mark Grumbridge , MHRA
Miguel Antunes, EMA
Richard Holborow, Head of Clinical Compliance BSI NL Notified Body
Sandra Brasil, EPTRI
Tom Melvin, University of Galway
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| 16:30 |
Coffee Break |
| 17:00 |
Session 4: The role of AI within the Paediatric Data Ecosystem Moderator(s): Donato Bonifazi, TEDDY - European Network of Excellence for Paediatric research Thomas Severin, Novartis
Alberto Tozzi, Ospedale Pediatrico Bambino Gesù
Luca Emili, InSilico Trials Technologies
Pekka Kahri, PHEMS
|
| 18:00 |
Reflection from the PDCO Chair Moderator(s): Martine Dehlinger-Kremer, EUCROF
Sabine Scherer, EMA
|
| 18:30 |
Closing Remarks End of Day 1
Begonya Nafria-Escalera, Barcelona Children’s Hospital & eYPAGnet
Martine Dehlinger-Kremer, EUCROF
|
| 19:00 |
Get-together and Networking |
| 08:00 |
Welcome Coffee |
| 08:00 |
Welcome and Debrief from Day 1
Begonya Nafria-Escalera, Barcelona Children’s Hospital & eYPAGnet
Martine Dehlinger-Kremer, EUCROF
|
| 08:10 |
The Future of Neonatology Research
Dina Apele-Freimane, Pauls Stradins Clinical University Hospital, PDCO
Mark Turner, conect4children Stichting
|
| 08:40 |
Feedback from breakout sessions |
| 09:15 |
Session 5: EU as a competitive/innovative region for paediatric research Moderator(s): Angelika Joos, MSD Sabine Fuerst-Recktenwald, Novartis
Andrea Manfrin, MHRA
Helena Luning, EUCROF
Mark Turner, conect4children Stichting
Monique Al, MedEthics, CCMO
Thorsten Ruppert, VfA - German Association of research-based Pharmaceutical Companies
|
| 10:15 |
Coffee Break |
| 10:35 |
Session 6: Optimising the clinical development path of orphan medicines Moderator(s): Christina Kyriakopoulo, EU Commission Kristina An Haack, Sanofi
Dariusz Adamczewski, Children's Tumor Foundation Europe
Kristina An Haack, Sanofi
Michela Guglieri, Newcastle University
Rima Nabbout, AP-HP
|
| 11:35 |
Session 7: Paediatric Drug repurposing Moderator(s): Christina Kyriakopoulou, EU Commission Solange Corriol-Rohou, Consultant
Anna Tavridou, EMA
Anton Ussi, EATRIS
Harald Schmidt, REPO4EU
Wael Khazen, AFM-Telethon, Horizon Europe DREAMS Project
|
| 12:35 |
Lunch |
| 13:30 |
Session 8: Global regulatory alignment Moderator(s): Mette Due Theilade Thomsen, PIP Adviser ApS Yury Kiselev, EMA
Atsuko Oimura, PMDA
Fahimeda Ali, MHRA
Franca Ligas, EMA
Michiyo Sakiyama, PMDA
Ming Su, Health Canada
|
| 14:30 |
Session 9: New voices in paediatric research Moderator(s): Emmanuella Amoako, Paediatric Oncologist and Yemaachi Biotech Rhian Thomas-Turner, Noah's Ark Children's Hospital for Wales
Elena Godoy, University Hospital of Málaga
Lone Friis, C-Further
Meritxell Teixidó, Gate2Brain
Rob ter Heine, Radboud University
|
| 15:30 |
End of Day 2 - Conclusion & Farewell
Begonya Nafria-Escalera, Barcelona Children’s Hospital & eYPAGnet
Martine Dehlinger-Kremer, EUCROF
|
| 15:45 |
END OF CONFERENCE |
