Accepted for/Published in: Journal of Medical Internet Research
Date Submitted: Feb 12, 2025
Date Accepted: May 9, 2025
The potential to leverage Real-World Data for paediatric clinical trials: a proof-of-concept study
ABSTRACT
Background:
The reuse of real-world data has important applications in paediatrics, where patient populations are scarce. A fundamental prerequisite for the success of reusing real-world data for any type of clinical study, whether it be interventional or observations, is the routine collection of relevant data items by clinicians caring for paediatric patients who are not, at the time of routine data collection, enrolled in any form of clinical research. This boils down to a fundamental question of whether routinely collected clinical data is suitable as a background historic resource for the design and conduct of these types of study.
Objective:
This paper reports a proof-of-concept study to explore whether real-world data can be used for comparator arm and post-marketing surveillance studies in two disease areas, using a sample of hospitals that routinely care for paediatric patients.
Methods:
Neurofibromatosis (NF) and Atopic Dermatitis (AD) were selected as the two disease areas for consideration. Comparator arm data items relevant for disease were identified by an expert group from clinical trial eligibility criteria, the EU PEARL NF master protocol, and inputs from academic and industrial partners. For post-marketing surveillance, portfolio of recent non-confidential clinical safety study protocols across multiple therapeutic areas were analysed and data items that were common between these protocols were extracted. After review and de-duplication 113 data items made the final list. Hospital sites across Europe were contacted through the conect4children (c4c) National Hub network to complete a survey about the collection and storage of these data items.
Results:
Twenty-four responses from 11 countries were received. The major finding was that substantial data was still stored in paper or unstructured format, which is challenging for reuse.
Conclusions:
While the study used a small sample of hospitals, it could be scaled up to generate more robust evidence. This investigation could be considered an early call for greater documentation of structured and coded entries to scale up the value of the data for research. Clinical Trial: N/A
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