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Article

The Use of Off-Label Medications in Newborn Infants Despite an Approved Alternative Being Available—Results of a National Survey

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Department of Pediatrics, Helios HSK, 65199 Wiesbaden, Germany
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Department of Pediatrics, Justus-Liebig University, 35385 Giessen, Germany
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The Ritchie Centre, Hudson Institute of Medical Research, Monash University, Melbourne 3168, Australia
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Pharmacy, St. Vincenz Hospital, 65549 Limburg, Germany
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Department of Pediatrics, J.W. Goethe University Hospital, 60590 Frankfurt, Germany
*
Author to whom correspondence should be addressed.
Academic Editor: Sandra Benavides
Pharmacy 2022, 10(1), 19; https://doi.org/10.3390/pharmacy10010019
Received: 14 December 2021 / Revised: 17 January 2022 / Accepted: 22 January 2022 / Published: 25 January 2022
Neonates continue to be treated with off-label or unlicensed drugs while in hospital. However, some medications that have previously been used in adults underwent clinical testing and licensure for use with a different indication in the neonatal and pediatric population. Almost always, the marketing of these newly approved substances in a niche indication is accompanied by a steep increase in the price of the compound. We investigated the use of the approved formulation or the cheaper off-label alternative of Ibuprofen (Pedea®), Propanolol (Hemangiol®) and Caffeine Citrate (Peyona®) in neonatal clinical practice by conducting a National Survey of 214 Perinatal Centers in Germany. We also assessed price differences between on- and off-label alternatives and the extend of the clinical development program of the on-label medication in the neonatal population. On-label medication was more frequently used than the off-label alternative in all indications (PDA: on-label to off-label ratio 1:0.26, Apnea: 1:0.56, Hemangioma 1:0.76). All sponsors did conduct placebo-controlled Phase III trials with efficacy and safety endpoints in the target population and the number of participants in the target population varied between 82 and 497. Costs for the three drugs in their approved and marketed formulations increased in median 405-fold compared with the corresponding off-label alternative. Overall, about one out of three neonatologists prescribed an off-label or non-approved drug to patients despite an alternative medication that is approved for the indication in the target population being available. View Full-Text
Keywords: off-label drug use; neonate; NICU; European pediatric regulation; prescribing off-label drug use; neonate; NICU; European pediatric regulation; prescribing
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MDPI and ACS Style

Veldman, A.; Richter, E.; Hacker, C.; Fischer, D. The Use of Off-Label Medications in Newborn Infants Despite an Approved Alternative Being Available—Results of a National Survey. Pharmacy 2022, 10, 19. https://doi.org/10.3390/pharmacy10010019

AMA Style

Veldman A, Richter E, Hacker C, Fischer D. The Use of Off-Label Medications in Newborn Infants Despite an Approved Alternative Being Available—Results of a National Survey. Pharmacy. 2022; 10(1):19. https://doi.org/10.3390/pharmacy10010019

Chicago/Turabian Style

Veldman, Alex, Eva Richter, Christian Hacker, and Doris Fischer. 2022. "The Use of Off-Label Medications in Newborn Infants Despite an Approved Alternative Being Available—Results of a National Survey" Pharmacy 10, no. 1: 19. https://doi.org/10.3390/pharmacy10010019

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