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Advancing evidence-based treatment of infectious diseases in children with real-world data: Opportunities and
Kendra K Radtke1, Atul J Butte1,2
1Bakar Computational Health Science Institute, University of California, San Francisco, San Francisco, CA, United States.
Insights
Real-world data (RWD) can improve pediatric medicine, especially for infectious diseases where off-label drug use is common. Utilizing RWD offers opportunities to generate crucial evidence for children's treatment and management.
Area of Science:
- Pediatric pharmacology
- Infectious disease research
- Real-world data (RWD) utilization
Background:
- Significant use of off-label medications in pediatric care due to limited pediatric-specific trial data.
- Abundance of real-world data from routine clinical practice in children.
- Underutilization of real-world data, including electronic health records, in pediatric infectious disease research.
Purpose of the Study:
- To explore the potential of real-world data (RWD) in advancing pediatric medicine.
- To identify opportunities and challenges for using RWD in pediatric infectious disease research.
- To generate evidence for optimal treatment and management strategies in pediatric infectious diseases.
Main Methods:
- Discussion of opportunities and challenges in applying real-world data (RWD) to pediatric infectious diseases.
- Analysis of existing clinical practice data for pediatric drug use.
- Review of literature on real-world data in pharmaceutical research.
Main Results:
- Real-world data (RWD) presents a significant opportunity to address the evidence gap in pediatric medicine.
- Challenges exist in applying RWD, particularly in specialized areas like pediatric infectious diseases.
- Potential for RWD to inform treatment guidelines and regulatory decisions for children's medications.
Conclusions:
- Real-world data (RWD) holds promise for improving the evidence base for pediatric medicines, especially infectious disease treatments.
- Overcoming challenges in RWD implementation is key to realizing its full potential in pediatric research.
- Further research and data infrastructure are needed to effectively leverage RWD for children's health outcomes.
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.
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