Degraeuwe Eva, Turner Mark A, Fernandes Ricardo M, Raes Ann, Vande Walle Johan, Schaefer Franz
Department of Internal Medicine and Paediatrics, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium.
Department of Paediatric Nephrology, Ghent University Hospital, Ghent, Belgium.
Front Pediatr. 2025 May 19;13:1523847. doi: 10.3389/fped.2025.1523847. eCollection 2025.
The prevalence and complexity of rare diseases (RDs) require concerted efforts in research and clinical trial capabilities. This paper aims to map the clinical trial sites within the Collaborative Network for European Clinical Trials for Children (conect4children, c4c) consortium and the European Reference Networks for Rare Diseases (ERNs), assessing their potential overlap and opportunities for synergies to optimize the selection and preparedness of sites for paediatric RD clinical trials.
A quantitative cross-mapping analysis was performed with publicly available data from ERN and c4c sites across 19 countries, complemented by information on paediatric site capabilities through interviews with network coordinators. Site analyses were done at country and setting levels. Heatmaps and an interactive matrix tool were developed using RStudio (v2023.12.0).
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. The median proportion of regional sites to University sites is 0.05 (IQR 0.12) across ERNs and 0.25 (IQR 0.37) across c4c national networks. The matrix tool can identify overlap and its absence for both university and regional hospitals, enhancing the preparedness and reach of paediatric rare disease trials. ERN representatives confirm the heatmap and matrix tool's utility in improving site selection and fostering network cooperation.
Heatmap analyses reveal a significant but incomplete overlap of RD clinical trial sites between ERNs and c4c in parts of Europe, suggesting strong potential for cross-network collaboration to enhance paediatric RD trial recruitment and outcomes.
罕见病（RDs）的患病率和复杂性需要在研究和临床试验能力方面做出协同努力。本文旨在绘制欧洲儿童临床试验协作网络（conect4children，c4c）联盟和欧洲罕见病参考网络（ERNs）内的临床试验地点，评估它们潜在的重叠情况以及协同增效的机会，以优化儿科罕见病临床试验地点的选择和准备工作。
利用来自19个国家的ERN和c4c地点的公开数据进行了定量交叉映射分析，并通过与网络协调员访谈获取有关儿科地点能力的信息进行补充。在国家和机构层面进行了地点分析。使用RStudio（v2023.12.0）开发了热图和交互式矩阵工具。
在荷兰、比利时、瑞典、丹麦和捷克共和国发现ERN和c4c网络之间的重叠度最高，表明这些地区有很强的整合度，而北欧（瑞典和丹麦）、东欧和南欧国家的重叠程度各不相同。ERNs中区域地点与大学地点的比例中位数为0.05（四分位距0.12），c4c国家网络中为0.25（四分位距0.37）。该矩阵工具可以识别大学医院和区域医院的重叠情况及其缺失情况，提高儿科罕见病试验的准备程度和覆盖范围。ERN代表确认热图和矩阵工具在改善地点选择和促进网络合作方面的效用。
热图分析显示，欧洲部分地区ERNs和c4c之间的罕见病临床试验地点存在显著但不完全的重叠，这表明跨网络合作在加强儿科罕见病试验招募和改善结果方面具有很大潜力。
