Use of unlicensed and off-label drugs in neonates in a Brazilian university hospital

Adriana Cristina De Souza Gonçalves,Maria Cândida Ferrarez Bouzada,Adriano Max Moreira Reis,Ana Carolina Gusmão Marçal

doi:10.1590/s2175-97902017000300252

Adriana Cristina De Souza Gonçalves, Maria Cândida Ferrarez Bouzada + Show 2 more

Open Access

https://doi.org/10.1590/s2175-97902017000300252

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Abstract

ABSTRACT This study was designed to investigate the use of off-label and unlicensed drugs in a Neonatal Care Unit (NCU) and to compare the frequency of use of off-label drugs according to the drug regulatory agencies in Brazil (Agencia Nacional de Vigilância Sanitaria-ANVISA) and the United States Food and Drug Administration (FDA). A prospective observational study was carried out in the NCU. Prescriptions were classified as off-label and unlicensed using both ANVISA and FDA criteria. A total of 157 newborns and 1187 prescriptions were analyzed. The most prescribed drug was fentanyl (9.3%), followed by multivitamin (8.4%) and gentamicin (7.9%). According to ANVISA criteria, there were 665 (56.0%) off-label prescriptions and 86 (7.2%) unlicensed prescriptions and 95.5% of newborns received at least one drug off-label. By contrast, according to FDA criteria, there were 592 (49.9%) off-label prescriptions and 84 (7.1%) unlicensed prescriptions, and 72.0% of newborns received at least one drug off-label. The off-label use of drugs registered by ANVISA differed significantly from that of drugs registered by the FDA. There was a high frequency of off-label and unlicensed drug use in the investigated NCU, and there was an inverse relationship between off-label and unlicensed usage and the gestational age of the newborns.

Highlights

IntroductionPediatric newborns are excluded from clinical trials because of ethical and methodological factors, which include difficulties in conducting clinical trials because of ethical issues, the small number of specialists in pediatric pharmacology, difficulties in the development of pediatric formulations, and low financial returns for the pharmaceutical industry (Dell’Aera et al, 2007; Cuzzolin, Atzei, Fanos, 2006)
Pediatric newborns are excluded from clinical trials because of ethical and methodological factors, which include difficulties in conducting clinical trials because of ethical issues, the small number of specialists in pediatric pharmacology, difficulties in the development of pediatric formulations, and low financial returns for the pharmaceutical industry (Dell’Aera et al, 2007; Cuzzolin, Atzei, Fanos, 2006).The lack of participation of children in clinical research studies is most evident during analyses of the use of drugs in neonates
We investigated the use of offlabel and unlicensed drugs in a neonatal intensive care unit and compared the frequency of off-label and unlicensed drug use according to the drug regulatory agency in Brazil (National Health Surveillance Agency-ANVISA) and Food and Drug Administration (FDA) in the United States

Summary

Introduction

Pediatric newborns are excluded from clinical trials because of ethical and methodological factors, which include difficulties in conducting clinical trials because of ethical issues, the small number of specialists in pediatric pharmacology, difficulties in the development of pediatric formulations, and low financial returns for the pharmaceutical industry (Dell’Aera et al, 2007; Cuzzolin, Atzei, Fanos, 2006). The lack of participation of children in clinical research studies is most evident during analyses of the use of drugs in neonates. The scarce number of clinical trials that involve neonates is a consequence of scientific and regulatory challenges, but investigating drugs in neonates is critical (Davis, Connor, Wood, 2012; Dell’Aera et al., 2007; Cuzzolin, Atzei, Fanos, 2006). Appropriate formulations for use in neonates may not exist (Davis, Connor, Wood, 2012)

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