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.

Frontiers in Pharmacology
|January 30, 2023
PubMed

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.

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