Pharmacoepidemiological safety studies in children: a systematic review

Abstract

PurposeIn order to identify challenges in pediatric pharmacoepidemiological safety studies, we assessed the characteristics of such (published) studies.MethodsRelevant articles from inception to 2013 were retrieved from Embase and Medline. We sequentially screened titles, abstracts and full texts with independent validation. We systematically collected data regarding general information, study methods and results.ResultsOut of 4825 unique articles, 268 full texts (5.6%) were retained; 147 (54.9%) pertained to drugs rather than vaccines. Considering the 268 studies, 202 (75.4%) concerned children and adolescents (2 to 11 years) and 14 (5.3%) included preterm newborns. Most studies originated from North America (154 [57.5%]) or Europe (92 [34.3%]). Only 47 studies (17.5%) were privately funded. The majority (174 [64.9%]) were cohort studies. Out of 268 studies, 196 (73.1%) collected data retrospectively; paper medical charts were the most common data source for the exposures (85 [31.7%]) and outcomes (122 [45.5%]). Only 3 (2.0%) drug‐only studies investigated rarely used drugs. Considering all 268 studies, only 27 (10.1%) reported sample size or power calculation. Most (75 [51.0%]) drug‐only studies corrected confounding by multivariate modeling unlike stratification in 66 (55.9%) vaccine‐only studies. Considering 75 child‐only studies without any statistically significant result, 41 (54.7%) did not discuss lack of power.ConclusionsAlthough the field of pediatric pharmacoepidemiology is steadily developing evaluation seldom includes neonates, is mainly focused on few drug classes and safety outcomes and concerns mainly drug use in developed countries. Small study size is a specific challenge in pediatrics. Reporting should be improved. © 2016 The Authors. Pharmacoepidemiology and Drug Safety Published by John Wiley & Sons Ltd.

Electronic health record (EHR) based postmarketing surveillance of adverse events associated with pediatric off‐label medication use: A case study of short‐acting beta‐2 agonists and arrhythmias

“…Pediatric pharmacoepidemiology and pharmacovigilance are of particular importance, given the lack of pediatric data for many medications and the high rate of off‐label use in the pediatric population . Yet, the total number of safety studies in pediatrics remains relatively low despite the increase in number of studies over the years …”

“…27 Yet, the total number of safety studies in pediatrics remains relatively low despite the increase in number of studies over the years. 28 These important pediatric safety studies require large reliable sources of data with necessary information. For many years, the pri- Our case study illustrates both the power of such a system for studying rare outcomes associated with commonly used medications in pediatrics, as well as the ongoing challenges.…”

Identifying the “Blip on the Radar Screen”: Leveraging Big Data in Defining Drug Safety and Efficacy in Pediatric Practice

“…EHRs and claims databases offer a rich source of data to perform safety studies. Osokogu et al . reported their systematic review of pharmacoepidemiologic safety studies in children.…”

“…[33][34][35] EHRs and claims databases offer a rich source of data to perform safety studies. Osokogu et al 36 reported their systematic review of pharmacoepidemiologic safety studies in children. They found the number of pharmacoepidemiologic safety studies to be low, with most being done in developed countries.…”

Off-label prescribing for children with chronic diseases in Nigeria; findings and implications

“…However, there are concerns about the safety, economic costs, and potentially ethical issues involved with such prescribing [2]. The off-label use of medicines is common in children because only a few clinical trials have been conducted to evaluate new medicines in paediatric population due to their vulnerability as well as ethical challenges [13]. Furthermore, major clinically significant medicines, as well as essential medicines, frequently used for adults are neither tested nor officially approved for use in children.…”