Comparing drug effectiveness in children: A systematic review.

Area of Science:

• Pharmacology
• Pediatric Medicine
• Health Services Research

Background:

• Comparative effectiveness research (CER) is crucial for optimizing drug use in pediatric populations.
• Assessing the current landscape of pediatric CER identifies critical gaps and areas for enhancement.

Purpose of the Study:

• To evaluate the state-of-the-art in pediatric comparative effectiveness research.
• To identify potential gaps and opportunities for improvement in the field.

Main Methods:

• Systematic literature search of Embase and Medline databases (inception to February 2015).
• Screening of titles, abstracts, and full texts with independent validation.
• Data extraction on study characteristics, statistical analysis, and quality assessment using Newcastle-Ottawa Scale (NOS).

Main Results:

• 164 articles were included from 3,926 screened abstracts; most studies originated from North America.
• Cohort studies predominated (90.8%); neonates were the least studied age group.
• Systemic antibacterials, psycholeptics, and antiepileptics were most frequently studied; propensity score use for confounding was low (8.5%).

Conclusions:

• Significant opportunities exist to enhance pediatric comparative effectiveness research.
• Frequently prescribed medications, such as analgesics and drugs for obstructive airway diseases, were underrepresented in studies.
• Underutilization of modern confounding adjustment methods, like propensity scores, limits the robustness of findings.