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Safety and Efficacy of Off-label and Unlicensed Medicines in Children

BACKGROUND: The aim of this study was to explore the use of off-label/unlicensed drugs to confirm the safety and efficacy of their prescription in children in Korea. METHODS: In this retrospective study, we analyzed data of patients who received any of the 32 drugs between January–December 2014 in t...

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Autores principales: Lee, Ji-Hyun, Byon, Hyo-Jin, Choi, Seungeun, Jang, Young-Eun, Kim, Eun-Hee, Kim, Jin-Tae, Kim, Hee-Soo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Korean Academy of Medical Sciences 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6125315/
https://www.ncbi.nlm.nih.gov/pubmed/30190655
http://dx.doi.org/10.3346/jkms.2018.33.e227
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author Lee, Ji-Hyun
Byon, Hyo-Jin
Choi, Seungeun
Jang, Young-Eun
Kim, Eun-Hee
Kim, Jin-Tae
Kim, Hee-Soo
author_facet Lee, Ji-Hyun
Byon, Hyo-Jin
Choi, Seungeun
Jang, Young-Eun
Kim, Eun-Hee
Kim, Jin-Tae
Kim, Hee-Soo
author_sort Lee, Ji-Hyun
collection PubMed
description BACKGROUND: The aim of this study was to explore the use of off-label/unlicensed drugs to confirm the safety and efficacy of their prescription in children in Korea. METHODS: In this retrospective study, we analyzed data of patients who received any of the 32 drugs between January–December 2014 in tertiary hospitals in Korea, including demographics, diagnoses, reasons for the medication, administration route, and details of adverse drug reactions. Additionally, the mortality in the cohort was assessed. The primary outcomes were efficacy and safety, including mortality, of these drugs in pediatric patients. The secondary outcomes were the current statuses of the use of off-label/unlicensed drugs in two centers. RESULTS: Totally, 5,130 prescriptions were found in 2,779 patients. Age (73.5%) and indication (11.7%) were the most frequent reasons for prescriptions being off-labeled/unlicensed. Approximately 88% of the prescriptions were effective, and 19% of the patients developed adverse drug reactions. The number of prescriptions was significantly higher in children with adverse drug reactions than it was in those without (2.8 vs. 1.5; P < 0.001). The number of prescribed off-label/unlicensed medicines and age at prescription were independently associated with adverse drug events (odds ratio, 1.55 and 1.1; P < 0.001 and 0.034, respectively). CONCLUSION: Children are still prescribed medicines that are not authorized in terms of age, weight, indications, or routes of administration. Therefore, many old products require re-assessment of authorization. More prospective clinical studies should be performed to confirm the efficacy and safety of drugs in the pediatric population.
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spelling pubmed-61253152018-09-10 Safety and Efficacy of Off-label and Unlicensed Medicines in Children Lee, Ji-Hyun Byon, Hyo-Jin Choi, Seungeun Jang, Young-Eun Kim, Eun-Hee Kim, Jin-Tae Kim, Hee-Soo J Korean Med Sci Original Article BACKGROUND: The aim of this study was to explore the use of off-label/unlicensed drugs to confirm the safety and efficacy of their prescription in children in Korea. METHODS: In this retrospective study, we analyzed data of patients who received any of the 32 drugs between January–December 2014 in tertiary hospitals in Korea, including demographics, diagnoses, reasons for the medication, administration route, and details of adverse drug reactions. Additionally, the mortality in the cohort was assessed. The primary outcomes were efficacy and safety, including mortality, of these drugs in pediatric patients. The secondary outcomes were the current statuses of the use of off-label/unlicensed drugs in two centers. RESULTS: Totally, 5,130 prescriptions were found in 2,779 patients. Age (73.5%) and indication (11.7%) were the most frequent reasons for prescriptions being off-labeled/unlicensed. Approximately 88% of the prescriptions were effective, and 19% of the patients developed adverse drug reactions. The number of prescriptions was significantly higher in children with adverse drug reactions than it was in those without (2.8 vs. 1.5; P < 0.001). The number of prescribed off-label/unlicensed medicines and age at prescription were independently associated with adverse drug events (odds ratio, 1.55 and 1.1; P < 0.001 and 0.034, respectively). CONCLUSION: Children are still prescribed medicines that are not authorized in terms of age, weight, indications, or routes of administration. Therefore, many old products require re-assessment of authorization. More prospective clinical studies should be performed to confirm the efficacy and safety of drugs in the pediatric population. The Korean Academy of Medical Sciences 2018-07-19 /pmc/articles/PMC6125315/ /pubmed/30190655 http://dx.doi.org/10.3346/jkms.2018.33.e227 Text en © 2018 The Korean Academy of Medical Sciences. https://creativecommons.org/licenses/by-nc/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (https://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Original Article
Lee, Ji-Hyun
Byon, Hyo-Jin
Choi, Seungeun
Jang, Young-Eun
Kim, Eun-Hee
Kim, Jin-Tae
Kim, Hee-Soo
Safety and Efficacy of Off-label and Unlicensed Medicines in Children
title Safety and Efficacy of Off-label and Unlicensed Medicines in Children
title_full Safety and Efficacy of Off-label and Unlicensed Medicines in Children
title_fullStr Safety and Efficacy of Off-label and Unlicensed Medicines in Children
title_full_unstemmed Safety and Efficacy of Off-label and Unlicensed Medicines in Children
title_short Safety and Efficacy of Off-label and Unlicensed Medicines in Children
title_sort safety and efficacy of off-label and unlicensed medicines in children
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6125315/
https://www.ncbi.nlm.nih.gov/pubmed/30190655
http://dx.doi.org/10.3346/jkms.2018.33.e227
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