EFGCP Better Medicines for Children Conference 2023

Global Paediatric Drug Development: the value of multi-stakeholders’ collaboration


Event Conference
Type Face To Face
Language English
Date(s) 17 & 18 Oct. 2023
Day(s) 2 Day(s)
Duration 2 Days


European Elections: 6-9 June 2024

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 2022, 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 to inform our delegates of the latest scientific and regulatory developments in the paediatric space.


This year will give us the opportunity to reconnect in person after 3 years of virtual conferences due to the pandemic. 


The conference will focus on Multi-stakeholders’ collaboration, its challenges and solutions, and in particular its value, with a focus on what has been achieved in Europe, in the US and globally, whether for example, through Public-Private Partnerships, e.g., the EU Innovative Health Initiative or the US Critical Path Institute. To this effect, the conference will bring together not only distinguished speakers from all around the world but also all the relevant stakeholders and experts involved in paediatric drug development.


Day One will be the opportunity to hear from the EU Commission where they are with the revision of Pharmaceutical Legislation and its impact of paediatric drug development. The stakeholders who have already used the Stepwise Paediatric Investigation Plan Pilot established by EMA in February 2023, will share their experience so far. Stakeholders from around the world will share their view on the value of multi-stakeholders’ collaboration, what can be learnt and what could be expected in the future to address unmet needs of underserved populations, such as neonates or those with rare diseases.


Active engagement of conference delegates will be then welcomed in Breakout Sessions to discuss challenges and identify solutions on what could be done to optimise children’s access to new medicines, with a focus on important topics:

1.    Inclusion of Adolescents in Adult Trials.

2.    Data collection and Data sharing.

3.    Paediatric Extrapolation: what progress has been made since 2022?


At the end of Day 1, a fireside chat will be the opportunity to hear from Brian Aylward (PDCO chair), Sylvie Benchetrit (PDCO vice-chair) and Angeliki Siapkara (former UK representative on PDCO) about their respective activities and priorities; and to hear their view and answers to questions raised ahead of the conference. 


Day two will be the opportunity to keep the momentum with some of the experts who joined the 2022 conference. Hot paediatric topics for which multi-stakeholders’ collaboration is more than ever valued to address gaps in scientific knowledge and find synergies to optimise patients’ access to new medicines, will explore innovative development avenues that could foster paediatric drug development. A panel committed to finding solutions for rare diseases, which present a unique public health challenge, will discuss the latest information on rare diseases. Similarly, new initiatives will be discussed, and their respective panels will explain their strategy to deliver tangible and innovative changes, whether for neonates, for the selection of investigational sites through quality criteria and standards for paediatric trials, or for the implementation of a platform for the use of Paediatric Oncology RWE in regulatory and clinical decision making.


The programme committee looks forward to welcoming you all to 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 from all parts of the world.


Fees Reductions can be applied for group registrations, see the Fees or contact events@efgcp.eu

Patients/Advocates can benefit from a Waiver to this conference, limited seats are available and subject to approval by EFGCP. Please make your request to events@efgcp.eu, including a short motivation paragraph.


Networking Event Conditions: A walking dinner will be organised on the 17th of October 2023 evening, at the venue. There are no additional costs to join, however, to ensure enough food is planned and avoid any wastes, registration to the networking event will take place separately, after participants have first confirmed their attendance to at least the Day 1 of this Conference, by registering on this website and settling their invoice. EFGCP will contact each participant after their registration to the conference is settled, to formally invite them to the Networking Event.


Thank you for your understanding.


IMPORTANT: By registering to this event, you also get a free access to the Virtual Pre-Conference on Pediatric Dose Optimization, held Virtualy on 12th of September 2023!

This waiver access it transferable to a colleague.

With the Support of



Tuesday 17 October 2023
9:00 Registration and Welcome Coffee
10:30 Opening of the Conference and Introduction
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
Begonya Nafria Escalera, Barcelona Children's Hospital & eYPAGnet
10:45 Revision of the Pharmaceutical Legislation - Impact on Paediatric & Orphan Drug Development
Moderator(s): Alexandru Costescu, European Commission
Fabio d'Atri, European Commission
Gilles Vassal, Gustave Roussy Institute
Victoria Kitcatt, Pfizer
Virginie Hivert, Eurordis
11:45 Panel Discussion: Stepwise Paediatric Investigation Plan
Moderator(s): Gilles Vassal, Gustave Roussy Institute Karl-Heinz Huemer, former PDCO member
Brian Aylward, PDCO Chair
Gesine Bejeuhr, Bayer
Giovanni Lesa, EMA
12:45 Lunch
14:00 Panel Discussion: The Value of Multistakeholder Collaboration for Paediatric Drug Development at the Global level
Moderator(s): Alexandru Costescu, European Commission Angeliki Siapkara, AstraZeneca
Andy Pearson, ACCELERATE
Begonya Nafria Escalera, Barcelona Children's Hospital & eYPAGnet
Cecile Ollivier, C-Path Institute
Heidrun Hildebrand, Bayer
Lynne Yao, FDA
Mark Turner, Conect4Children
Martina Penazzato, World Health Organization
Nathalie Seigneuret, IHI
Ralph Bax, EMA
15:30 Breakout Sessions
Inclusion of Adolescents in Adult Trials
Moderator(s): Rhian Thomas Turner, Noah's Ark Children's Hospital for Wales Solange Corriol-Rohou, AstraZeneca
Gyorgy Zorenyi, AstraZeneca
Lionel Tan, ViiV Healthcare
Max Williamson, ACCELERATE FAIR Work Group
Data Collection and Data Sharing
Moderator(s): Angeliki Siapkara, AstraZeneca David Sibbald, Aridhia
Alexandre Bétourné, C-Path Institute
Becca Leary, Conect4Children
Jeff Barrett, Aridhia
Paediatric Extrapolation - what progress has been made since 2022?
Moderator(s): Richard Vesely, Allucent Sabine Fuerst-Recktenwald, Roche
Andrew Thomson, EMA
Eric Zuckerman, Pediatric IBD Foundation,
Lynne Yao, FDA
17:00 Coffee Break
17:30 Fireside Chat
Moderator(s): Angeliki Siapkara , AstraZeneca MarkTurner, Conect4Children
Brian Aylward, PDCO Chair
Sylvie Benchetrit, PDCO Vice-Chair
18:30 End of Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
19:00 Networking Event: Walking Dinner (until 22.00)
Wednesday 18 October 2023
08:00 Welcome coffee
08:30 Welcome and Debrief from Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
Begonya Nafria Escalera, Barcelona Children's Hospital & eYPAGnet
08:45 Panel Discussion: the Enpr-EMA Initiative on Site Quality Criteria/ Standards for Paediatric Trials
Moderator(s): Gunter Egger, EMA Pirkko Lepola, FINPEMED
Begonya Nafria Escalera, Barcelona Children's Hospital & eYPAGnet
Ensio Norjavaara, AstraZeneca
Eva Degraeuwe, Ghent University
Kirsten Sherman Cervati, Icon
Ricardo Fernandes, STAND4Kids
09:30 Panel Session: The Value of Digital Health Technlogies in Paediatric Drug Development
Moderator(s): Angeliki Siapkara, AstraZeneca
Laurent Servais, Oxford University
Niina Kolehmainen, Newcastle University
Tim Buckinx, Epihunter
Yury Kiselev, EMA
10:15 Coffee break
10:45 Panel Session: The Rare Disease MOONSHOT Initiative: how to make rare diseases more common through drug development optimisation?
Moderator(s): Cecile Ollivier, C-Path Institute Solange Corriol-Rohou, AstraZeneca
Christina Kyriakopoulou, European Commission
Yanis Mimouni, EJP RD
11:45 Paediatric formulation development between patients’ needs, methodology and regulatory requirements
Moderator(s): Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc. Martina Penazzato, WHO
Daniel Marin, KIDS Barcelona
Sibylle Reidemeister, Novartis
Viviane Klingmann, Paediatric University Hospital Düsseldorf
12:45 Lunch
14:00 Neonatal Research - What progress has been made since the 2022 Paediatric Conference?
Moderator(s): Mark Turner, Conect4Children Solange Corriol-Rohou, AstraZeneca
Jeff Barrett, Aridhia
John van den Anker, Children's National Medical Center Washington
Ralph Bax, EMA
Susan McCune, PPD
15:00 Feedback from the Breakout Sessions
16:00 End of Day 2 - Conclusion and Farewell
Begonya Nafria Escalera, Barcelona Children's Hospital & eYAPGnet
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.