Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review

Area of Science:

• Pediatric pharmacology
• Real-world evidence research
• Observational study methodology

Background:

• Real-world evidence (RWE) is crucial for pediatric medicine, as many child-specific drug uses lack randomized controlled trial data.
• The application of RWE in pediatrics remains limited, presenting a gap in evidence-based prescribing for children.

Purpose of the Study:

• To systematically review observational studies from 2016 that utilized RWE to evaluate medication safety or effectiveness in pediatric populations.
• To assess the current state and identify trends in pediatric RWE research.

Main Methods:

• Systematic literature search of PubMed and other sources for observational studies published in 2016.
• Inclusion criteria: pediatric subjects (<18 years), medication exposure, safety/effectiveness assessment, comparison group, English language.
• Data extraction and quality assessment using GRACE checklist; focus on study design, data sources, and statistical methods.

Main Results:

• 29 studies met eligibility criteria from 915 screened citations.
• Most studies used primary data or chart reviews from single institutions, underutilizing available administrative and electronic health record databases.
• A quarter of studies lacked robust statistical methods for confounder control; diverse age groups, conditions, and medications were studied.

Conclusions:

• The use of RWE in pediatrics is nascent, with limited studies in 2016 not fully aligning with RWE definitions.
• Significant opportunities exist to enhance pediatric RWE by leveraging electronic health records and administrative data for medication safety and effectiveness studies.
• The impact of the broader RWE movement on pediatric research is less pronounced, highlighting a need for increased development and adoption in this field.