The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study
This study highlights gaps in the structured documentation of pediatric clinical data across European sites. While the routine collection of many variables is promising, the lack of structured and coded formats poses a barrier to reusing these data for observational studies or comparator arms. As a first step toward the broader integration of RWD into pediatric research, this study demonstrates the feasibility of assessing EHR data availability and sets the stage for future scaling across more diseases and sites.
Authors
- Rianne Oostenbrink (ORCID: https://orcid.org/0000-0001-7919-8934)
- Fenna Mahler (ORCID: https://orcid.org/0000-0001-6851-5455)
- Joanne Lee
- Volker Straub (ORCID: https://orcid.org/0000-0001-9046-3540)
- A. K. Sen (ORCID: https://orcid.org/0000-0001-6761-2632)
- Rebecca Leary (ORCID: https://orcid.org/0000-0002-8039-5334)
- Avril Palmeri (ORCID: https://orcid.org/0000-0002-6171-5793)
- Viviana Giannuzzi (ORCID: https://orcid.org/0000-0002-2534-6783)
- Simon Woodworth (ORCID: https://orcid.org/0000-0001-7406-8743)
- Beate Aurich (ORCID: https://orcid.org/0000-0002-8830-023X)
- Jens Declerck (ORCID: https://orcid.org/0000-0002-9743-9188)
- S. K. Malik (ORCID: https://orcid.org/0009-0000-1965-1266)
- Dipak Kalra (ORCID: https://orcid.org/0000-0002-2998-9882)
Institutions
- Radboud University Nijmegen (NL)
- University College Cork (IE)
- Erasmus MC (NL)
- Radboud University Medical Center (NL)
- Infant (IE)
- Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus (IT)
- European Clinical Research Infrastructure Network (FR)
- MNM Consulting (France) (FR)
- Newcastle University (GB)
Publication Details
- Journal
- Journal of Medical Internet Research
- Published
- 2025-05-30
- DOI
- https://doi.org/10.2196/72573
- Citations
- 3
- Primary Topic
- Genomics and Rare Diseases
- Type
- article
- Field-Weighted Citation Impact
- 5.45
Funders
- European Federation of Pharmaceutical Industries and Associations
- European Commission