Key Themes
1. Tackling Pediatric-Specific Needs in Drug Development
Traditional approaches often do not meet the unique needs of paediatric populations, leading to challenges with dosage and potential side effects when adult drugs are adapted for children. The conferences emphasise the importance of customising research methodologies, clinical trial designs, and formulations to improve safety and efficacy in pediatric treatments.
2. Regulatory Advancements and Challenges
Key discussions addressed regulatory frameworks, including the Paediatric Regulation in the EU and similar initiatives in the U.S. Although these regulations have facilitated paediatric drug development, challenges remain in aligning regulatory requirements with the realities of conducting pediatric trials. Streamlining these processes was a prominent theme, aiming for better regulatory support tailored to paediatric needs.
3. The Power of Collaboration
The conferences showcased how multi-stakeholder collaboration across sectors—industry, academia, government, and patient organisations—drives innovation in pediatric medicine. Success stories highlighted at the events reveal that shared knowledge and resources are instrumental in addressing the logistical and financial challenges that paediatric research often faces.
4. Innovative Clinical Trial Models
With children, clinical trials bring unique ethical and logistical hurdles. Adaptive trials, real-world data, and age-appropriate designs were presented as promising solutions. These approaches balance ethical concerns with scientific rigor, ensuring that paediatric trials yield meaningful data without compromising young patients’ safety.
5. Future Directions and Ongoing Challenges
Topics like adolescent inclusion in adult trials, the role of real-world evidence, and optimising the development process were central to discussions of what comes next. The EU’s ongoing revision of pharmaceutical regulations—including the Paediatric and Orphan Regulations—reflects an evolving commitment to improving access and innovation in paediatric care.
Abstract
For many years, the European Forum for Good Clinical Practice (EFGCP) Children Medicines Working Party has organised a Paediatric conference annually. In the past, this event was organised jointly with the European Medicines Agency who was used to host it, along with the Drug Information Association (DIA). This conference is the opportunity for all involved in paediatric drug development, i.e., regulators, HTA bodies, patients’ representatives, academia and industry, to share learnings and raise awareness about new regulatory requirements of interest to optimise paediatric drug development.
The theme of the 2021 conference was “Challenges and Solutions – the path forward” while in 2022 it focused on “Progress made and Continuing Challenges”. Because of the COVID-19 pandemic these two conferences were organised virtually. However, this has not impacted the attendance and value of the conference, since because of a broad and attractive agenda there was a wide stakeholder participation, which provided a compendious overview of the leading issues to improve children’s access to innovative medicines.