The article “The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study” has been recently published in the Journal of Medical Internet Research (JMIR). Viviana Giannuzzi from Fondazione Gianni Benzi is one of the authors. This publication is the result of collaborative work within the European conect4children (c4c) network, which brought together academic and industry partners to foster innovation in paediatric clinical research across Europe.
The study investigated the availability and structure of clinical data from routine paediatric care, collected from 24 hospitals across 11 European countries. The aim was to assess the potential of using real-world data to support paediatric clinical research. Researchers analysed more than one hundred key clinical data elements, including demographics, vital signs, and disease-specific information related to neurofibromatosis and atopic dermatitis.
The findings show that general clinical and drug safety data elements are often recorded in structured formats, while disease-specific medical information were often not documented in a standardized way. Most of them were captured in free text, making it more difficult to reuse them for secondary research purposes.
The study highlights the importance of leveraging real-world data by improving data capture, system design, and staff training in recording medical information in structured and computable formats, in order to facilitate data reuse in paediatric research. This would help tackling the challenge of the limited number of paediatric patients and the difficulty of finding them when developing clinical trials.
Data protection regulations and informed consent remain important challenges when reusing EHR data, particularly across international borders. Legal and ethical requirements can constrain data sharing, especially in retrospective research.
This publication represents a significant step toward the development of innovative, evidence-based approaches to paediatric clinical research, especially in rare diseases. The article is available in open access here.