EFGCP/DIA Better Medicines for Children Conference

Global Paediatric Drug Development Challenges & Solutions - The Path Forward


Event Conference
Type Virtual Event
Language English
Date(s) 18 & 19 Oct. 2021
Day(s) 3 Day(s)
Duration 7 Hours / Day
The Paediatric “Better Medicines for Children” Conference is an annual flagship event providing a unique opportunity to share best practice updates and a truly global outreach. Despite a challenging environment, and the need to meet virtually, we believe have been able to organise a dynamic, innovative, and highly engaging conference to inform our delegates of the latest scientific and regulatory developments.

This year the conference will focus on Global Paediatric Development, its challenges and solutions. To this effect, the conference brings together not only distinguished speakers from all around the world but also all the stakeholders involved, namely regulators, HTA body and payer representatives, industry, academia and patient representatives.

While focusing on paediatric research, i.e. what is happening, and how the future could look, Day One will bring regulators and patient representatives from around the world to discuss the impact of existing European and US paediatric regulations on paediatric R&D. The Programme Committee is proud to welcome Fabio d’Atri who will update us on the work done by the European Commission to revisit the Paediatric Regulation. He will be joined by regulators from the UK MHRA and the US FDA, while a panel moderated by a WHO representative will include regulators from Japanese PMDA, Chinese NMPA and Brazilian ANVISA, all involved in the review of paediatric dossiers and matters.

Active engagement of conference participants will be welcomed in Breakout Sessions to discuss challenges and identify solutions on what could be done to optimise children access to new medicines, with a focus on:
1. Public Private Partnerships
2. Paediatric Formulations
3. Remote Decentralised Clinical Trials.

At the end of Day 1, a fireside chat moderated by Elin Haf Davies (Aparito) will be the opportunity to hear from Dr Koen Norga (PDCO chair) and Dr Angeliki Siapkara (MHRA and former UK representative on PDCO) about their respective activities and priorities; and also to discuss the changing European landscape post-Brexit.

Day 2 will focus on hot topics in paediatric R&D showing the value of multi-stakeholders’ collaboration to address gaps in scientific knowledge, explore new innovative development avenues, and foster paediatric drug development.

The programme committee looks forward to welcoming you all to the conference for a global educational and networking event that will present a unique opportunity to learn from each other, to meet and interact with colleagues from all parts of the world.

Thank you for your attendance.


Monday 18 October 2021
11:45 Opening of Conference
12:00 Welcome and Introduction to the Conference
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON, Germany
12:10 Revision of the Paediatric Regulation
Fabio d’Atri, European Commission, Belgium
12:45 UK experience in paediatric development
Angeliki Siapkara, MHRA, United Kingdom
13:00 Impact of the RACE for Children Act on paediatric development
Gregory Reaman, FDA, USA
13:15 Panel session with regulators: Paediatrics in Japan, Chinese Taipei and Brazil
Moderator(s): Marie Valentin, WHO, Switzerland
Michiyo Sakiyama, PMDA, Japan
Priscila Lemos Costa, ANVISA, Brazil
Shinichi Kijima, PMDA, Japan
Ya-Lei Wang, TFDA, Chinese Taipei
14:15 Panel session: Impact of regulatory changes for the patients
Moderator(s): Begonya Nafria-Escalera, SJD Children’s Hospital Barcelona, Spain Dimitrios Athanasiou, PDCO Patient Representative & EURORDIS, Greece
Marieke van Meel, NephcEurope Foundation, The Netherlands
Nancy Goodman, CEO of Kids v Cancer, US
Xing Huanping, Meier SMA Care Center, China
15:00 Break
15:15 Breakout sessions
Public Private Partnerships
Moderator(s): Cecile Ollivier, Critical-Path Institute, the Netherlands Heidrun Hildebrand, Bayer, Germany Mark Turner, University of Liverpool, United Kingdom
Alexandru Costescu, European Commission, DG R&I, Health Innovations & Ecosystems, Belgium
Jeff Barrett, Critical Path Institute, USA
Magda Chlebus, EFPIA, Belgium
Nathalie Seigneuret, IMI, Belgium
Paediatric formulations
Moderator(s): Catherine Tuleu, European Paediatric Formulation Initiative, United Kingdom Simon Broady, AstraZeneca, United Kingdom
Siri Wang, Norwegian Medicines Agency, PDCO Member, Norway
Linda Lewis, Clinton Health Access Initiative, USA
Remote Decentralised Clinical Trials in Paediatrics
Moderator(s): Mira Zuidgeest, Utrecht University, The Netherlands Solange Corriol-Rohou, AstraZeneca, France
Isaac Rodriguez-Chavez, ICON, USA
Christa Van Kan, Healx, United Kingdom
Elin Haf Davies, Aparito, United Kingdom
Frederik Grell Nørgaard, DKMA, Denmark
Tony Hall, Healx, United Kingdom
17:00 Break
17:10 Fireside Chat with PDCO Chair & UK Paediatric Regulator
Moderator(s): Elin Haf Davies, Aparito, United Kingdom
Angeliki Siapkara, MHRA, United Kingdom
Koenraad Norga, Antwerp University Hospital, Belgium & Chair of EMA PDCO, The Netherlands
18:10 End of Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON, Germany
Tuesday 19 October 2021
11:45 Opening of Conference with a video with the young people from eYPAGnet European Young Person’s Advisory Groups Network) and ICAN (International Children Advisory Network)
12:00 Welcome and Debrief from Day 1
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON, Germany
12:10 Panel Session: Optimising Paediatric Drug Development
Moderator(s): Sabine Fuerst-Recktenwald, Roche, Switzerland Solange Corriol-Rohou, AstraZeneca, France
ICH E11A Paediatric Extrapolation Guideline
Lynne Yao, FDA, USA
Use of quantitative approaches
Amy Cheung, Certara, United Kingdom
Complex Innovative Trial Designs
Anja Schiel, SAWP Chair, NoMA, Norway
Use of RWE
Ralf Herold, EMA, The Netherlands
Adolescents in Adult Clinical Trials
Christina Bucci-Rechtweg, Novartis, USA
13:20 Panel Session: Vaccines (incl.COVID-19) in paediatrics
Moderator(s): Angelika Joos, MSD, Belgium Rhian Thomas-Turner, NHS, United Kingdom
Dina Apele-Freimane, PDCO Member & Pauls Stradins Clinical University Hospital, Latvia
Franck Devaux, HUDERF, Belgium
Freya Semple, YPAGs, United Kingdom
Katharine Wright, The Nuffield Council on Bioethics, United Kingdom
Sarah Cotte, [Recorded Video] Kids France
Sheila Varadan, Leiden University, Netherlands based in Thailand
Stephen P. Lockhart, Vaccine Clinical R&D Europe and Asia-Pacific Head at Pfizer, United Kindgom
14:20 Panel Session: Funding Paediatric Research in Europe
Moderator(s): Katie Rizvi, Youth Cancer Europe, Hungary Pirkko Lepola, FINPEDMED, Finland
Alexandru Costescu, European Commission, DG R&I, Health Innovations & Ecosystems, Belgium
Didier Caizergues, Genethon, France
Nathalie Seigneuret, IMI, Belgium
15:00 Panel Session: Pregnant women in clinical research (including in-utero treatment)
Moderator(s): Angeliki Siapkara, MHRA, United Kingdom Solange Corriol-Rohou, AstraZeneca, France
Harris Dalrymple, ICON, United Kingdom
Janet Nooney, MHRA, United Kindgom
John van den Anker, Children’s National Hospital, Washington (D.C.), USA
Lynne P. Yao, FDA, USA
15:40 Feedback from the Breakout Sessions:
16:00 Break
16:15 Panel Session: Paediatric Unmet Needs - Interaction with HTA bodies & payers
Moderator(s): Helen Shaw, Proveca, United Kingdom Fabio d’Atri, European Commission
Anja Schiel, SAWP Chair, NoMA, Norway
Claudia Scharl, MEDEV, Austria
Karl-Heinz Huemer, EMA PDCO, Austria
Michael Ermisch, GKV-Spitzenverband / National Association of Statutory Health Insurance Funds, Berlin, Germany
Niklas Hedberg, EunetHTA, TLV, Sweden
17:15 Key Note: Developing a vaccine for COVID-19 in the midst of the public health emergency
Stephen P. Lockhart,, Vaccine Clinical R&D Europe and Asia-Pacific Head at Pfizer, United Kindgom
17:45 Key Note: Paediatric Development for the Future; some personal reflections
Agnès Saint Raymond, Formerly EMA, France
18:15 End of Day 2 - Conclusion & Farewell
Martine Dehlinger-Kremer, EFGCP, EUCROF & ICON, Germany
Tuesday 19 October 2021