EFGCP Better Medicines for Children Conference 2023
Global Paediatric Drug Development: the value of multi-stakeholders’ collaboration
Description
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.
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.
Programme
9:00 |
Registration and Welcome Coffee |
10:30 |
Opening of the Conference and Introduction
Begonya Nafria Escalera, Barcelona Children's Hospital & eYPAGnet
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
|
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
Yann Le Cam, 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
Chrissi Pallidis, EMA
Gesine Bejeuhr, Bayer
|
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, Liverpool University
Nathalie Seigneuret, IHI
Ralph Bax, EMA
|
15:30 |
Breakout Sessions |
Inclusion of Adolescents in Adult Trials Moderator(s): Max Williamson, ACCELERATE FAIR Work Group Rhian Thomas Turner, Noah's Ark Children's Hospital for Wales Solange Corriol-Rohou, AstraZeneca |
|
Data Collection and Data Sharing Moderator(s): Angeliki Siapkara, AstraZeneca David Sibbald, Aridhia |
|
Paediatric Extrapolation - what progress has been made since 2022? Moderator(s): Richard Vesely, Allucent Sabine Fuerst-Recktenwald, Roche
Lynne Yao, FDA
|
|
17:00 |
Coffee Break |
17:30 |
Fireside Chat Moderator(s): Claudio Fracasso, Pfizer
Brian Aylward, PDCO Chair
Sylvie Benchetrit, PDCO Vice-Chair
|
18:30 |
End of Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
|
08:00 |
Welcome coffee |
08:30 |
Welcome and Debrief from Day 1
Begonya Nafria Escalera, Barcelona Children's Hospital & eYPAGnet
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
|
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
Mark Turner, Liverpool University
Pernille Skovby, National Coordinator DanPedMed
|
09:30 |
Panel Session: The Value of Digital Health Technlogies in Paediatric Drug Development Moderator(s): Angeliki Siapkara, AstraZeneca Laurent Servais, Oxford University
Francesca Ceretta, EMA
Kia Nazarpoor, Edinburgh University
Niina Kolehmainen, Newcastle University
Tim Buckinx, Epihunter
|
10:30 |
Coffee break |
11:00 |
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
James Eshelby, Pfizer
Virginie Hivert, Eurordis
Yanis Mimouni, EJP RD
|
11:30 |
Paediatric formulation development between patients’ needs, methodology and regulatory requirements Moderator(s): Claudio Fracasso, Pfizer Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
Sibylle Reidemeister, Novartis
Viviane Klingmann, Paediatric University Hospital Düsseldorf
|
12:30 |
Lunch |
14:00 |
Neonatal Research - What progress has been made since the 2022 Paediatric Conference? Moderator(s): Mark Turner, Liverpool University 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 |
16:15 |
End of Day 2 - Conclusion and Farewell
Begonya Nafria Escalera, Barcelona Children's Hospital & eYAPGnet
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON Plc.
|