Advancing evidence-based treatment of infectious diseases in children with real-world data: Opportunities and challenges

Abstract

There is an increased interest in utilizing real-world data (RWD) for pharmaceutical research and regulatory decision-making. The development and use of pediatric medicines could benefit greatly from real-world data studies given nearly half of drugs prescribed to children are “off-label”, meaning there is a lack of pediatric-specific evidence from controlled trials, while there is an abundance of data from routine clinical practice. Currently, the use of real-world data, such as data from electronic health records, is lacking in pediatric research, especially within infectious diseases. Here, we discuss opportunities and challenges for real-world data to generate evidence on the optimal treatment and management of infectious diseases in children.

Predictive Modeling of Drug‐Related Adverse Events with Real‐World Data: A Case Study of Linezolid Hematologic Outcomes

“…Electronic health records (EHRs) are a real-world data source that can be leveraged to address drug-and disease-related questions. 8,9 EHRs provide high-dimensional data from diverse populations as part of routine clinical care, filling information gaps not feasible or available from clinical trials. Because EHR data are collected for patient care purposes, considerations on data quality and integrity and potential biases need to be evaluated when used for research.…”

“…Electronic health records (EHRs) are a real‐world data source that can be leveraged to address drug‐ and disease‐related questions 8,9 . EHRs provide high‐dimensional data from diverse populations as part of routine clinical care, filling information gaps not feasible or available from clinical trials.…”