Background and aim Data regarding the association between the off-label drug use and adverse drug reactions (ADRs) in neonates is scarce. The main aims of this study are to evaluate prospectively the relationship between adverse drug reactions and off-label or unlicensed (OLUL) drugs in 2 neonatal centres in Lyon (France), and to provide more information on prescribing practice, the amplitude, nature and consequences of OLUL drug use.
Methods The French summaries of product characteristics in Theriaque 2012 (a prescription products guide) are being used as a primary reference source for determining drug labelling. Detection of ADRs is carried out by health care professionals and research groups using a trigger tool and patients’ electronic health records. The causality between suspected ADRs and drug is evaluated using the WHO and the French methods of imputability.
Preliminary results for a 12 month period 910 neonates were included. 671 (73,7%) were preterm. 94,8% (CI 95: 93,3–96,3) of children received at least one OLUL drug, 66% of 8891 prescriptions were used in OLUL manner. 80% of the 96 validated ADRs were classified as severe. 84% of drugs related to ADRs were used in OLUL manner.
Conclusion Our study confirmed that OLUL drug utilisation is common in neonates. This study will bring more evidence on the correlation between OLUL drug use and ADRs. Institutions and pharmaceutical industry should develop clinical trials for neonates, and ensure they do not remain a “therapeutic orphan”. A specific reference source for drug used in neonates would be helpful.