Background: Paediatric drug development faces recurrent barriers such as fragmentation of the stakeholders and geographical differences in research conduct.
Over the last years, important advances have been made through a pan-European network conect4children (c4c), funded by the Innovative Medicines Initiative 2 (IMI2). The Belgian Paediatric Clinical Research Network (BCPRN) within c4c, with its headquarters at Ghent University Hospital, plays a fundamental role in these developments. BPCRN was preceded by the SAFEPEDRUG project, which laid the ground for translational research for paediatric drug development in Belgium.
Method: This report describes the progress of c4c and BPCRN over the past three years, focused on the evolution from preclinical studies to pharmacological clinical trials, and its future growth and sustainable model.
Results: In the proof-of-viability (PoV) clinical studies, three academic studies have started including patients across Europe, in which Belgium is the first to receive national approval for the KD-CAAP study. Four industry PoV studies have also progressed with site development. BPCRN additionally aids in creating a sustainable model for after IMI2-funding as well as the development of data-standardization, with the support of the Health, innovation and research institute. Broadening the scope further, BPCRN is collaborating with the I-ACT for Children (US), conducting multiple clinical trials, and aiming towards global drug development partnerships.
Conclusion: The SAFEPEDRUG preclinical research has evolved into a clinical setting within the pan-European network of c4c and BPCRN. Platforms such as c4c and I-ACT for children are essential to achieve substantial improvement in medicines for children in the EU, the US and worldwide.