Evidence map›Paper›PMID 40458448›Full record

ArticleFrontiers in pediatrics2025

Mapping the rare disease paediatric clinical trial availabilities in Europe.

Eva Degraeuwe, Mark A Turner, Ricardo M Fernandes, Ann Raes, Johan Vande Walle, Franz Schaefer

Abstract read
In one paragraph

Article in Frontiers in pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Eva DegraeuweDepartment of Internal Medicine and Paediatrics, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium.
Mark A TurnerDepartment of Pediatrics, University of Liverpool (ULIV), Liverpool, United Kingdom.
Ricardo M FernandesLaboratory of Clinical Pharmacology and Therapeutics, Faculty of Medicine, University of Lisbon, Lisbon, Portugal.
Ann RaesDepartment of Internal Medicine and Paediatrics, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium.
Johan Vande WalleDepartment of Internal Medicine and Paediatrics, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium.
Franz SchaeferZentrum für Kinder- und Jugendmedizin, Universitätsklinikum Heidelberg, Heidelberg, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: 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. Method: 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). 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. 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. Conclusion: 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.

Indexed as

drug developmentnetwork engagementnetworkspaediatricrare diseases c4c -ERN mapping clinical trial availabilities

Identifiers

PMID40458448
PMCPMC12127375

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.