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Off-label prescribing in Italian pediatric practice: insights from a nationwide survey
Anna Arbo1, Anna Flamigni2, Giulia Zamagni2
1Institute for Maternal and Child Health, IRCCS Burlo Garofolo, 34137, Trieste, Italy. anna.arbo@burlo.trieste.it.
Insights
Off-label prescribing in pediatric hospitals is common, driven by specialized units like Neonatal Intensive Care Units (NICUs), not hospital size. Improved digital tracking and oversight enhance regulatory compliance for safer pediatric pharmacotherapy.
Area of Science:
- Pediatric Pharmacology
- Healthcare Management
- Regulatory Science
Background:
- Off-label drug prescribing in pediatric care is prevalent due to a lack of age-specific formulations and data.
- This practice poses risks to drug safety, efficacy, and dosing accuracy, especially in neonates and infants.
- Existing regulatory oversight and institutional awareness of off-label use vary significantly, with limited data on hospital management practices.
Purpose of the Study:
- To understand how Italian hospitals manage and regulate off-label prescribing in pediatric care.
- To identify factors associated with regulatory compliance and safer prescribing practices.
- To inform improvements in oversight and promote safer pharmacotherapy for children.
Main Methods:
- A multicenter, cross-sectional survey was conducted across 50 Italian facilities from November 13, 2024, to January 31, 2025.
- Data were collected under the Italian Network for Pediatric Clinical Trials (INCiPiT).
- Analysis focused on the association between institutional specialization (e.g., NICUs), hospital size, digital tracking, research units, and regulatory compliance.
Main Results:
- Off-label prescribing was strongly associated with Neonatal Intensive Care Units (NICUs), increasing up to 50% after compassionate use refusal.
- 64% of facilities with <5000 pediatric admissions had NICUs and birth centers.
- 25% of participating centers were unaware of their off-label prescribing frequency.
- Facilities with digital tracking and dedicated research units demonstrated better regulatory compliance and higher compassionate use approval rates.
Conclusions:
- Off-label prescribing in pediatric hospitals is pervasive and driven by institutional specialization, particularly NICU presence, rather than hospital size.
- Structured oversight, digital infrastructure, and cost monitoring enhance regulatory compliance.
- Standardized procedures and policy alignment are crucial for ensuring safe pediatric pharmacotherapy.
Abstract:
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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