Yackey Katelyn, Stukus Kristin, Cohen Daniel, Kline David, Zhao Sonia, Stanley Rachel
Department of Emergency Medicine, University of Kentucky, Lexington, Kentucky;
Nationwide Children's Hospital, Columbus, Ohio; and.
Hosp Pediatr. 2019 Mar;9(3):186-193. doi: 10.1542/hpeds.2018-0168. Epub 2019 Feb 11.
To describe the frequency of off-label drug use in 2014 as defined by the Food and Drug Administration (FDA)-approved age ranges in patients ≤18 years of age, to determine the rate of off-label drug use in 2014 by drug classification, and to compare current off-label medication usage rates with historical rates.
This is a retrospective cohort study of an administrative database containing inpatient resource use data from January 1, 2014, to December 31, 2014. Patients ≤18 years of age receiving 1 of 76 selected commonly prescribed medications are included. Off-label drug use is defined as use in a patient younger than the lower limit of the FDA-approved age range for any indication or dosage form of that drug.
At least 1 drug was prescribed off label in 779 270 of 2 773 770 (28.1%) patient visits during the study period. Younger age, longer hospital stays, and mortality were associated with higher rates of off-label medication prescription. Off-label usage of certain medications differed between care settings. Rates of off-label medication use were higher in observational (45.5%), inpatient (53.9%), and ambulatory (54.2%) settings.
Although off-label drug use at major US pediatric hospitals is declining, 1 out of every 4 medications is not in accordance with FDA label indications for patient age. There exists substantial variation in off-label drug use among drug categories and encounter types. Although many commonly prescribed medications are FDA-approved for use in subpopulations of pediatric patients, studies of their safety, efficacy, pharmacokinetics, and optimal dosing are ongoing.
描述2014年食品药品监督管理局（FDA）批准的年龄范围所定义的18岁及以下患者中标签外用药的使用频率，按药物分类确定2014年标签外用药的使用率，并将当前标签外用药使用率与历史使用率进行比较。
这是一项对行政数据库的回顾性队列研究，该数据库包含2014年1月1日至2014年12月31日的住院资源使用数据。纳入18岁及以下接受76种选定常用处方药中一种的患者。标签外用药定义为在任何适应症或剂型中，用于年龄低于FDA批准年龄范围下限的患者。
在研究期间，2773770次患者就诊中有779270次（28.1%）至少开具了一种标签外用药。年龄较小、住院时间较长和死亡率与标签外用药处方率较高相关。某些药物的标签外使用在不同护理环境中有所不同。观察性（45.5%）、住院（53.9%）和门诊（54.2%）环境中的标签外用药使用率较高。
尽管美国主要儿科医院的标签外用药使用正在下降，但每四种药物中就有一种不符合FDA对患者年龄的标签适应症。标签外用药在药物类别和就诊类型之间存在很大差异。尽管许多常用处方药已获FDA批准用于儿科患者亚群，但对其安全性、有效性、药代动力学和最佳剂量的研究仍在进行中。
