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Off-label use and pharmacovigilance among infants: analysis of the Thai Health Product Vigilance National Database

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Abstract

Background

A lack of drug safety and efficacy studies in pediatric patients is a major problem contributing to inappropriate drug use among this population. The prevalence of ‘off-label’ drug use is high in pediatric patients, leading to an increased risk of adverse events (AEs). A pharmacovigilance database is useful for healthcare providers in monitoring AEs associated with off-label drug use.

Objectives

The aim of this study was to explore the characteristics of AEs reported among pediatric patients aged 1 month to 2 years who received off-label drugs, using information derived from the Thai pharmacovigilance database.

Methods

Spontaneous reports of AEs in pediatric patients aged 1 month to 2 years were derived from the Thai Health Product Vigilance Center (HPVC) database for the period of January 1984 to November 2013. Off-label drug use was defined as drug use outside the age range indicated by US FDA and European Medicines Agency (EMA) drug labeling. Descriptive statistics were used to describe demographics and Anatomical Therapeutic Chemical (ATC) drug classes. Reporting odds ratios were used to evaluate associations between off-label drug use and AE seriousness and the occurrence of critical AEs.

Results

A total of 22,294 AE reports were collected, with 21,390 AE reports meeting the inclusion criteria. These reports were further classified as those involving off-age drug use (5.2 %), age-label drug use (65.2 %), and unclear/unfound age labeling (29.6 %). Of those containing off-age drug use, most were in the ATC anatomical main group of nervous system agents (24.1 %), followed by systemic anti-infective agents (20.5 %). There was a significant association between off-label drug use and the occurrence of critical AEs.

Conclusions

Off-label drug use is associated with unwanted AEs. Therefore, assessing the risk versus benefit of each drug should be taken into consideration before prescribing off-label drugs for pediatric patients.

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Acknowledgments

The authors would especially like to acknowledge and thank the Health Product Vigilance Center (HPVC), Food and Drug Administration, Ministry of Public Health, Thailand, for data permission. We would also like to thank Assoc. Prof. Dr. Michael Oszko, Lawrence Memorial Hospital, Kansas, USA, for his support in manuscript writing.

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Authors and Affiliations

  1. College of Pharmacotherapy of Thailand, The Pharmacy Council of Thailand, Nonthaburi, Thailand

    Pawalee Niamtaworn & Suphat Subongkot

  2. Clinical Pharmacy Division, Faculty of Pharmaceutical Sciences, Khon Kaen University, 123 Mittraphap Road, Naimuang, Muang District, Khon Kaen, 40002, Thailand

    Pawalee Niamtaworn & Suphat Subongkot

  3. Health Product Vigilance Center (HPVC), Food and Drug Administration, Ministry of Public Health, Nonthaburi, Thailand

    Wimon Suwankesawong

  4. Center of Pharmaceutical Outcomes Research, Department of Pharmacy Practice, Faculty of Pharmaceutical Sciences, Naresuan University, Muang, Phitsanulok, Thailand

    Piyameth Dilokthornsakul & Nathorn Chaiyakunapruk

  5. Center for Pharmaceutical Outcomes Research, University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Aurora, CO, USA

    Piyameth Dilokthornsakul

  6. School of Pharmacy, Monash University Malaysia, Bandar Sunway, Selangor Darul Ehsan, Malaysia

    Nathorn Chaiyakunapruk

  7. School of Population Health, University of Queensland, Brisbane, QLD, Australia

    Nathorn Chaiyakunapruk

  8. School of Pharmacy, University of Wisconsin-Madison, Madison, WI, USA

    Nathorn Chaiyakunapruk

Authors
  1. Pawalee Niamtaworn
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  2. Wimon Suwankesawong
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  3. Suphat Subongkot
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  4. Piyameth Dilokthornsakul
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Correspondence to Suphat Subongkot.

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The authors declare no conflicts of interest.

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Niamtaworn, P., Suwankesawong, W., Subongkot, S. et al. Off-label use and pharmacovigilance among infants: analysis of the Thai Health Product Vigilance National Database. Drugs Ther Perspect 32, 304–310 (2016). https://doi.org/10.1007/s40267-016-0300-y

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  • DOI: https://doi.org/10.1007/s40267-016-0300-y

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