ORIGINAL RESEARCH article
Front. Pediatr.
Sec. Children and Health
Volume 13 - 2025 | doi: 10.3389/fped.2025.1523847
Mapping the Rare Disease Paediatric Clinical Trial Availabilities in Europe
Provisionally accepted
- 1Department of Internal Medicine and Pediatrics, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium
- 2University of Liverpool, Liverpool, North West England, United Kingdom
- 3conect4children Stichting, Utrecht, Netherlands, Netherlands
- 4Laboratory of Clinical Pharmacology and Therapeutics, Faculty of Medicine, University of Lisbon, Lisbon, Portugal
- 5Department of Pediatrics, Ghent University Hospital, Ghent, East Flanders, Belgium
- 6European Rare Kidney Disease Network (ERKNET), Ghent, East Flanders, Belgium
- 7European Rare Kidney Disease Network (ERKNet), Heidelberg, Belgium
- 8Heidelberg University, Heidelberg, Baden-Württemberg, Germany
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INTRODUCTION The prevalence and complexity of rare diseases (RDs) require concerted efforts inresearch and clinical trial capabilities. This paper aims to map the clinical trial sites within theCollaborative Network for European Clinical Trials for Children (conect4children, c4c) consortium andthe European Reference Networks for Rare Diseases (ERNs), assessing their potential overlap andopportunities for synergies to optimize the selection and preparedness of sites for paediatric RD clinicaltrials.METHOD A quantitative cross-mapping analysis was performed with publicly available data from ERNand c4c sites across 19 countries, complemented by information on paediatric site capabilities throughinterviews with network coordinators. Site analyses were done at country and setting levels. Heatmapsand an interactive matrix tool were developed using RStudio (v2023.12.0).RESULTS The highest overlap between ERN and c4c networks is found in the Netherlands, Belgium,Sweden, Denmark, and the Czech Republic, indicating strong integration in these regions, while Nordic(Sweden and Denmark), Eastern, and Southern European countries show varying levels of overlap. Themedian proportion of regional sites to University sites is 0.05 (IQR 0.12) across ERNs and 0.25 (IQR0.37) across c4c national networks. The matrix tool can identify overlap and its absence for bothuniversity and regional hospitals, enhancing the preparedness and reach of paediatric rare diseasetrials. ERN representatives confirm the heatmap and matrix tool's utility in improving site selection andfostering network cooperation.CONCLUSION Heatmap analyses reveal a significant but incomplete overlap of RD clinical trial sitesbetween ERNs and c4c in parts of Europe, suggesting strong
Keywords: Paediatric, Drug Development, networks, rare diseases c4c -ERN Mapping Clinical Trial Availabili5es, network engagement
Received: 06 Nov 2024; Accepted: 28 Apr 2025.
Copyright: © 2025 Degraeuwe, Mark, Fernandes, Raes, Vande Walle and Schaefer. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Eva Degraeuwe, Department of Internal Medicine and Pediatrics, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium
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