Off-label prescribing in Italian pediatric practice: insights from a nationwide survey

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Off-label prescribing in pediatric care is common due to the lack of age-specific drug formulations, posing risks to safety and efficacy. Understanding how Italian hospitals manage and regulate this practice is essential to improving oversight and promoting safer prescribing. A multicenter, cross-sectional survey was conducted from November 13, 2024, to January 31, 2025, under the Italian Network for Pediatric Clinical Trials (INCiPiT). Fifty facilities participated; 64% of those with < 5000 pediatric admissions had Neonatal Intensive Care Units (NICUs) and birth centers. Off-label prescribing was strongly associated with NICUs, increasing up to 50% following compassionate use refusal. Notably, 25% of centers were unaware of prescribing frequency. Facilities with digital tracking and dedicated research units had better regulatory compliance and higher compassionate use approval rates. CONCLUSION: The present analysis revealed that off-label prescribing is pervasive and predominantly driven by institutional specialization rather than hospital size. The implementation of structured oversight, digital infrastructure, and cost monitoring has been demonstrated to enhance regulatory compliance. The findings of this study support the need for standardized procedures and policy alignment to ensure safe pediatric pharmacotherapy. WHAT IS KNOWN: • Off-label drug prescribing in pediatric care is a prevalent practice, primarily attributable to the paucity of age-appropriate formulations and clinical trial data for children.This practice entails potential risks associated with drug safety, efficacy, and dosing accuracy, particularly in vulnerable populations such as neonates and infants. • Regulatory oversight and institutional awareness of off-label use vary significantly across healthcare systems, with limited data available on how hospitals manage these prescriptions at the organizational level. WHAT IS NEW: • This nationwide Italian survey reveals that off-label prescribing in Italian pediatric hospitals is primarily driven by institutional specialization-especially NICU presence-rather than hospital size, with digital tracking and research units linked to better regulatory compliance. • A significant portion of facilities lack awareness of their off-label prescribing rates, highlighting the need for standardized oversight and national policy alignment to ensure safer pediatric drug use.

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