EFGCP Better Medicines for Children Conference 2022

Global Paediatric Drug Development: Progress made & always remaining challenges


Event Conference
Type Virtual Event
Language English
Date(s) 18 & 19 Oct. 2022
Day(s) 2 Day(s)
Duration 8 hours/ day


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 2021, and discuss paediatric updates with a truly global outreach. Despite an ever changing and challenging environment, and the need to meet virtually again, 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.


The conference will focus on Global Paediatric Development, its challenges and solutions, and in particular on what has been achieved during the pandemic, and the remaining opportunities and challenges. To this effect, the conference brings together not only distinguished speakers from all around the world but also all the stakeholders involved in paediatric drug development.


Day One, the new elected Chair of the Paediatric Committee (PDCO) at the EMA, will share with us the vision of the PDCO, and regulators and patient representatives will discuss the potential impact on R&D of the implementation of the Clinical Trial Regulation. Stakeholders from around the world while focusing on global alignment will share their view on what can be learnt from paediatric oncology 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. 


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.    How to improve Patient and parents’ involvement in Paediatric research?

2.    Paediatric Lay Summaries

3.    Is it time to prioritise?


At the end of Day 1, a fireside chat will be the opportunity to hear from Dr Koen Norga (former PDCO chair) and Dr Angeliki Siapkara (MHRA and former UK representative on PDCO) about their respective activities and priorities; and also to hear their view and answers to questions raised ahead of the conference. 


Day two will be the opportunity to keep the momentum with experts who joined the 2021 conference. Hot paediatric topics for which multi-stakeholders’ collaboration is more than ever valued to address gaps in scientific knowledge, will explore innovative development avenues that could foster paediatric drug development.


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 colleagues from all parts of the world.

We do hope that the 2022 conference will be the last one organised remotely, and 2023 will give us to go back to face-to-face meetings.


For those interested in paediatric drug development, a pre-conference Workshop on  the "Fundamentals of Paediatric Extrapolation" has been set up on October 17. This will be the opportunity to learn from the experts the value of paediatric extrapolation, a topic detailed in the ICH E11 guideline recently released for public consultation. For further details on this pre-conference Workshop, please visit the webpage

-> Exclusive Offer: Combine your registration to the pre-conference and to the conference (17, 18 & 19 October 2022), to benefit from exclusive registration rate on both events. Register via this page and select the "Combined Events" registration option.


Thank you for joining us!


Tuesday 18 October 2022
11:45 Opening of Conference with a video with the young people from eYPAGnet
12:00 Welcome and Introduction to the Conference
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON
12:10 PDCO Vision
Brian Aylward, Chair PDCO, EMA
12:40 Implementation of the CT Regulation 536/2014 - Impact on Paediatric trials
Moderator(s): Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON
Ann Marie Janson-Lang, Swedish MPA
Nicola Ruperto, PRINTO
13:30 Panel Discussion Global/transatlantic alignement for the development of paediatric products - what can be learnt from paediatric oncology drug development?
Moderator(s): Gilles Vassal, Gustave Roussy Institute
Angeliki Siapkara, MHRA
Dominik Karres, EMA
Helen Mao, Health Canada
Kaye Robertson, TGA
Martha Donoghue, FDA
14:30 Coffee Break
14:45 Panel Session Rare diseases - what is happening and can be expected?
Moderator(s): Angeliki Siapkara, MHRA
Christina Kyriakopoulou , European Commission
Dimitrios Athanasiou, EURORDIS
Kristina An Haack, Sanofi
Ruth Ladenstein, Head of the Clinical Trials Unit S2IRP-CCRI, Director OKIDS
15:45 Breakout Sessions
How to improve Patient & Parents involvement in Paediatric Research?
Moderator(s): Pamela Dicks, NHS & eYPAGnet Rhian Thomas-Turner, NHS
Chris Evans, Little Journey
Sheila Varadan, Leiden University
Paediatric Lay Summaries
Moderator(s): Begonya Nafria Escalera, SJD Children’s Hospital Barcelona & eYPAGnet Nicola Ruperto, PRINTO
Is it time to prioritise?
Moderator(s): Gilles Vassal, Gustave Roussy Institute Solange Corriol-Rohou, AstraZeneca
Dimitrios Athanasiou, EURORDIS
Karl-Heinz Huemer, EMA PDCO
17:15 Fireside Chat
Moderator(s): Claudio Fracasso, Pfizer
Angeliki Siapkara, MHRA
Koenraad Norga, former PDCO Chair
18:15 End of Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON
Wednesday 19 October 2022
11:45 Opening of Conference with a video with the young people from eYPAGnet (European Young Person’s Advisory Groups Network)
12:00 Welcome and Debrief from Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON
12:10 Panel Session Optimising Paediatric Drug Development - What progess has been made since the 2021 Paediatric Conference?
Moderator(s): Gilles Vassal, Gustave Roussy Institute Sabine Fuerst-Recktenwald, Roche
Innovative Techniques towards Drug Development
Hanna Bachelor , Institute of Pharmacy and Biomedical Sciences, University of Strathclyde
Use of RWD in paediatric drug development
Marie-Yvonne Douste Blazy, MYDB Paediatric Clinical Insights
Decentralised Clinical Trials
Isaac Rodriguez-Chavez, ICON
Adolescents in Adult Clinical Trials
Christina Bucci-Rechtweg, Novartis
13:30 Panel Session Neonatal Research - where are we and what could be next steps?
Moderator(s): Mark Turner, University of Liverpool Solange Corriol-Rohou, AstraZeneca
Jeff Barrett, C-Path Initiative
John van den Anker, Children’s National Medical Center Washington
Ralph Bax, EMA
Susan McCune, PPD
14:30 Panel Session What's new at ICH for paediatric drug development?
Moderator(s): Sabine Fuerst-Recktenwald, Roche Solange Corriol-Rohou, AstraZeneca
Pregnant & Breast feeding Individuals in CTs
Corinne de Vries, EMA
Lynne Yao, FDA
Kristin Karlsson, MPA
Nicolas Frey, Roche
15:30 Coffee Break
15:50 Panel Session Can drug repurposing benefit drug development in Children & in Paediatric Rare Diseases
Moderator(s): Alexandru Costescu, European Commission David Hayes, AstraZeneca
Billy Amzal, Quinten Health
Guillaume Canaud , Necker-Enfants malades – AP-HP Hospital
Gunter Egger, Enpr-EMA / EMA
Sam Blackman, DayOne
16:50 Feedback from the Breakout Sessions
How to improve Patient & Parents involvement in Paediatric Research?
Paediatric Lay Summaries
Is it time to prioritise?
17:50 End of Day 2 - Conclusion & Farewell
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON