EFGCP Better Medicines for Children Conference 2024
Common issues in clinical development amplified in paediatric drug development
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 2023, 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.
Last year, when we met in October 2023, gave us the opportunity to finally reconnect in person after 3 years of virtual conferences due to the pandemic. This year will also be an in-person conference to happen in Brussels, at the Crowne Plaza Airport Hotel.
The conference will focus on “Common issues in clinical development amplified in paediatric drug development”, its challenges and solutions, and in particular its value, with a focus on what can be done and has been achieved in Europe, and this year also in the US and globally. To this effect, the conference will bring together distinguished speakers from all around the world and relevant stakeholders and experts involved in paediatric medicines development.
On Day One, further to a Key Note focusing on Mental Health and Neurologic Disorders in Children, there will be the opportunity to discuss implications of the new Mechanism of Action Paediatric Investigation Plan, which while considered a child-centric option, is a new option considered by Policy makers who are still working on the revision of the Pharmaceutical Legislation. Examples of successful EU-funded paediatric projects. One session will focus on multi-stakeholders’ collaboration knowing a number of initiatives are already planned to start in 2024, which will benefit all involved in medicines development including children. The last session will provide the opportunity to discuss the Potential of Artificial Intelligence in Paediatric Drug Development
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 childrens’ access to new medicines, with a focus on the important topics:
1. Inclusion of Pregnant and Breastfeeding Individuals in Clinical Trials.
2. Use of Real-World Data/Registries in Paediatric Drug Development.
3. Paediatric-only medicines development / First-in-child companies.
Day two will provide the opportunity to share Paediatric Collaboration Activities at the Global Level with a panel of regulators from various regions, such as Africa & Middle East, Japan, Australia, Canada, Europe, and the USA. This session will be complemented by a session on Childrens’ Access to Medicines in Developing Countries. Prior to sharing the feedback from Day 1’s Breakout Sessions, a final session will explore the proposed changes to the EU Pharmaceutical Legislation and the implementation of the Health Technology Assessment (HTA) Regulation, to better understand what the major paediatric impacts for key stakeholders might be within early 2025.
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.
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
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10:15 |
KEYNOTE – Treatment of Psychiatric Disorders in children and adolescents: current situation and needs for research
Carmen Moreno, Hospital Gregorio Maranon
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10:45 |
Session 1: Neuro Health Moderator(s): Sabine Fuerst-Recktenwald, Roche Sara Carucci, Università degli studi di Cagliari |
Challenges and opportunities for paediatric drug development |
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Depression
Argyris Stingaris, University College of London
Louisa Braun Exner, PDCO representative Denmark
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Multiple Sclerosis
Ulrike Bonati, Roche
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Paediatric Irritability: From Definition and Assessment to Treatment
Lauren Wyatt, ADHD Foundation
Sara Carucci, Università degli studi di Cagliari
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11:45 |
Session 2: Mechanism of Action Paediatric Investigation Plan Moderator(s): Gesine Bejeuhr, Bayer Gilles Vassal, Gustave Roussy Institute
Angelika Joos, MSD
Chrissi Pallidis, EMA
Daniel Marin, Kids Barcelona
Sylvie Benchetrit, ANSM and PDCO EMA
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12:45 |
Lunch |
13:45 |
Session 3: Examples of successful EU-funded paediatric projects: Moderator(s): Christina Kyriakopoulou, European Commission Mark Turner, conect4children Stichting |
DEEP: DEferiprone Evaluation in Paediatrics
Mariagrazia Felisi, TEDDY Network & Consorzio per Valutazioni Biologiche et Farmacologiche
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DevelopAKUre: Clinical Development of Nitisinone for Alkaptonuria
Nick Sireau, CEO of the AKU Society
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Lena Project: Labeling of Enalapril from Neonates up to Adolescents
Stéphanie Läer, Institut für Klinische Pharmazie und Pharmakotherapie, Heinrich-Heine-Universität Düsseldorf
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14:45 |
Breakout session |
1. Inclusion of Pregnant and Breastfeeding Individuals in Clinical Trials: impact and benefits for both mother and child Moderator(s): Martine Dehlinger-Kremer, ICON & EUCROF Solange Corriol-Rohou, AstraZeneca
Corinne de Vries, European Medicines Agency
Lynne Yao, FDA
Marie Teil, UCB
Tyrza Boersma, Patient representative
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2. Use of RWD/Registries in Paediatric Drug Development Moderator(s): Angeliki Siapkara, AstraZeneca Jeffrey Barrett, Aridhia
Katherine Donegan, MHRA
Marina Kolochavina, Five Voices Consortium
Matthew Arnold , AstraZeneca
Roberto De Lisa, EMA
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3. Paediatric-only drug development / First-in-child companies Moderator(s): Gesine Bejeuhr, Bayer Rhian Thomas-Turner, Noah's Ark Children's Hospital for Wales
Cesare Saponi, Oncohereos
Marc Ramis, Venture capital
Mauricio Preciado Awad, Save the Children Fund
Sam Blackman, Day One Biopharmaceutical
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16:30 |
Coffee break |
17:00 |
Session 4: The Potential of Artificial Intelligence in Paediatric Drug Development Moderator(s): Claudio Fracasso, Hippocrates Research Srl Thomas Severin, Novartis
Danielle Belgrave, GSK
Karim Azer, Novartis
Roberto De Lisa, EMA
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18:00 |
Reflection from the PDCO Chair
Brian Aylward, Health Products Regulatory Authority Ireland and PDCO, EMA
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18:30 |
End of Day 1
Martine Dehlinger-Kremer, ICON & EUCROF
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19:00 |
Get-Together and Networking |
08:00 |
Welcome coffee |
08:30 |
Welcome and Debrief from Day 1
Begonya Nafria-Escalera, Barcelona Children’s Hospital & eYPAGnet
Martine Dehlinger-Kremer, ICON & EUCROF
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08:45 |
Session 5: Regulatory updates Moderator(s): Fahimeda Ali, MHRA United Kingdom Sabine Fuerst-Recktenwald, Roche
Fahimeda Ali, MHRA United Kingdom
Michiyo Sakiyama, Pharmaceuticals and Medical Devices Agency Japan
Natsuko Hayama, Pharmaceuticals and Medical Devices Agency Japan
Robyn Langham, Therapeutic Goods Administration Australia
Sabine Fuerst-Recktenwald, Roche
Yashwant Sinha, Therapeutic Goods Administration Australia
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09:45 |
Session 6: Pathways to Medicines in Developing Countries Moderator(s): Martina Penazzato, WHO Rhian Thomas-Turner, Noah's Ark Children's Hospital for Wales
Emmanuella Amoako, Yemaachi Biotech
Lenias Hwenda, Medicines for Africa
Moy Bracken, Access to Medicines Foundation
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10:30 |
Coffee break |
11:00 |
Session 7: Multi Stakeholder Collaboration in 2024 Moderator(s): Claudio Fracasso, Hippocrates Research Solange Corriol-Rohou, AstraZeneca
Christina Kyriakopoulou, European Commission
Flora Musuamba Tshinanu, FAMPH
Mark Turner, conect4children Stichting
Nathalie Seigneuret , IHI
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12:00 |
Lunch |
13:00 |
Session 8: European Legislations impact on paediatric medicines Moderator(s): Angeliki Siapkara, AstraZeneca Dimitrios Athanasiou, BoD Member World Duchenne Organization; PCWP member EMA
Anja Schiel, Norwegian Medicines Agency
Brian Aylward, Health Products Regulatory Authority Ireland
Gilles Vassal, Gustave Roussy Institute
Victoria Kitcatt, Pfizer
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14:00 |
Feedback from the Breakout Sessions: |
1. Inclusion of Pregnant and Breastfeeding Individuals in Clinical Trials: impact and benefits for both mother and child |
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2. Use of Registries in Paediatric Drug Development |
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3. Paediatric-only drug development / First-in-child companies |
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15:00 |
End of Day 2 – Conclusion & Farewell
Begonya Nafria-Escalera, Barcelona Children’s Hospital & eYPAGnet
Martine Dehlinger-Kremer, ICON & EUCROF
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15:15 |
End of Conference |