Expert Opin Drug Saf. 2026 Mar 30. doi: 10.1080/14740338.2026.2652916. Online ahead of print.
ABSTRACT
INTRODUCTION: Drug utilization studies (DUS) provide important insights on how medications are used in routine clinical practice and can identify gaps between real-world use and existing evidence and guidance. While DUS are relevant across all therapeutic areas, they are particularly valuable in pediatrics as a substantial proportion of prescribing occurs off-label or with limited trial data.
AREAS COVERED: This paper describes five domains where pediatric DUS can contribute to improving safe and rational use of medications: (i) setting research priorities, (ii) supporting drug surveillance and stewardship, (iii) identifying and reducing unwarranted variation, (iv) assessing prescriber behavior, and (v) evaluating the impact of regulatory actions and clinical guidelines. The five domains are illustrated using examples of Danish register-based pediatric DUS.
EXPERT OPINION: Despite their value, pediatric DUS remain underused in clinical and regulatory decision-making. DUS should be systematically integrated into the development and revision of clinical guidelines on medication use and used routinely to evaluate the effects of major regulatory initiatives. Establishing near – real-time monitoring systems and extending DUS to inpatient settings would further strengthen their ability to impact clinical practice and support safer and more evidence-based prescribing for children and adolescents.
PMID:41910965 | DOI:10.1080/14740338.2026.2652916
