EFGCP Better Medicines for Children Conference 2023
Global Paediatric Drug Development: the value of multi-stakeholders’ collaboration
|Date(s)||17 & 18 Oct. 2023|
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.
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 Bryan Aylward (PDCO chair), Sylvie Benchetrit (PDCO vice-chair) and Angeliki Siapkara (Head of Paediatrics, Innovative Medicines at the UK MHRA and 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.
We do hope that the 2022 conference was the last one organised remotely, and 2023 will give us the opportunity to go back to face-to-face meetings.
For those interested in paediatric drug development, a pre-conference Workshop on "Dose-finding/Dose selection" has been set up on October 16, 2023. Determining the appropriate dose and regimen is one of the hardest and most important tasks during the development of new paediatric drugs to ensure an appropriate balance of risks and benefits for children. This workshop will be the opportunity to learn from the experts how to identify the right dose to be prescribed in children depending on their age/weigh, a topic detailed in the ICH E4 guideline which should be revisited in the future, according to the ICH Model Informed Drug Development (MIDD) Road Map released by the MIDD discussion group in March 2022.