The Potential to Leverage Real-World Data for Pediatric Clinical Trials: A Proof-of-Concept Study

Area of Science:

• Pediatric clinical research
• Real-world data (RWD) utilization
• Electronic Health Records (EHR) data standardization

Background:

• Pediatric clinical research, particularly for rare diseases, faces significant hurdles in patient identification, recruitment, and trial execution due to small, stratified populations and fragmented data.
• Real-world data (RWD) from electronic health records (EHRs) offers potential for innovative trial designs, including real-world comparator arms and postmarketing surveillance.
• The effective use of RWD hinges on the routine availability of structured and reusable clinical data.

Purpose of the Study:

• To evaluate the availability and structure of routine clinical data within European pediatric hospitals.
• To assess data elements relevant for constructing comparator arms and conducting postmarketing surveillance studies.
• To examine data collection practices for neurofibromatosis (NF) and atopic dermatitis (AD) as representative pediatric conditions.

Main Methods:

• Developed an inventory of 113 critical clinical data items based on expert analysis of NF, AD, and safety study protocols.
• Administered a structured web-based survey across European pediatric sites via the connect4children (c4c) National Hub network.
• Assessed data capture methods: structured/coded EHR fields, free text, external systems, or paper records.

Main Results:

• Survey responses from 24 hospitals in 11 European countries indicated substantial variability in data capture and storage.
• General clinical and drug safety data (e.g., demographics, vital signs, medications) were frequently structured.
• Disease-specific and contextual variables, lifestyle, and family history data were often recorded as free text or inconsistently, limiting EHR data reusability for research.

Conclusions:

• Significant gaps exist in the structured documentation of pediatric clinical data across European healthcare settings.
• The lack of standardized, coded data formats impedes the reuse of EHR data for observational studies and comparator arms.
• This proof-of-concept study confirms the feasibility of assessing EHR data availability and provides a foundation for expanding RWD integration in pediatric research.