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

ABSTRACT:

Background

Pediatric clinical research, especially in rare diseases, faces persistent challenges including the identification and recruitment of eligible patients, assessing protocol feasibility, and ensuring efficient trial execution. These issues are compounded by small, age-stratified populations and fragmented clinical data. Real-world data (RWD), especially when drawn from electronic health records (EHRs), present an opportunity to support innovative trial designs, such as real-world comparator arms and postmarketing surveillance. However, realizing this potential depends on the routine availability of structured, reusable clinical data.

Objective

This proof-of-concept study aimed to assess the availability and structure of routine clinical data in European pediatric ho

SUBMITTER: Declerck J 

PROVIDER: S-EPMC12166320 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature