Pediatric post-marketing safety systems in North America: assessment of the current status

McMahon, Ann; Wharton, Gerold T.; Bonnel, Renan A.; DeCelle, Mary; Swank, Kimberley; Testoni, Daniela; Cope, Judith U.; Smith, P. Brian; Wu, Eileen; Murphy, M. Dianne

doi:10.1002/pds.3813

Cited by 11 publications

(10 citation statements)

References 24 publications

(40 reference statements)

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“…While most concede the higher accuracy of data collected in a research setting, many accept that databases collected for non-research purposes may offer larger sample sizes and a more generalizable study population. Others have also noted limitations of the databases that have pediatric populations, citing a lack of clinical detail (and basic information such as birth weight and gestational age) and validation of pediatric outcomes [57][58][59][60][61][62]. Some scientists have noted that RWE studies can have sample sizes orders of magnitude larger than those in RCTs [63].…”

Section: Discussionmentioning

confidence: 99%

Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review

Lasky

Carleton

Horton

et al. 2020

Drugs - Real World Outcomes

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Background The promise of real-world evidence (RWE) is especially relevant to pediatrics, where medicines prescribed for children are often used without evidence derived from randomized clinical trials. Objectives The aim of this systematic review was to describe the state of RWE in pediatrics by identifying observational studies published during 2016 that used RWE to assess medication safety or effectiveness in children. Methods An electronic search of PubMed was combined with an extended search of references within systematic reviews and expert suggestions. Studies were included if they reported on an infant or child under 18 years with exposure to medications; assessed safety or effectiveness; specified a comparison or control group, and were published in English in 2016. Data extraction was conducted by one team member using a standardized form and reviewed by a second team member. Study quality was assessed using the GRACE checklist for rating the quality of observational studies. Results After removing duplicates, 915 citations were screened and 29 studies met the eligibility criteria. Most of the eligible studies relied on primary data collection or chart review at a single institution and did not use the growing number of administrative or electronic health record databases available. One-quarter of the studies did not use well-established statistical methods to control for confounders. No single disease group or medication predominated, and age groups ranged from infants to adolescents. Conclusions A small body of observational studies published in 2016 were categorized by the study team as using real-world data to assess medication safety or effectiveness in children. Studies varied in age groups, diseases or conditions, and methods, and may not have fully met the FDA definition of RWE. Our review indicates that the use of RWE is not fully developed in pediatrics, and suggests an opportunity to further develop capabilities and more fully leverage administrative and electronic health record databases to study medication safety and effectiveness in children. Our systematic review appears generalizable to pediatrics broadly, and documents that the high level of activity in RWE in general has had less of an impact on pediatrics.

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Section: Discussionmentioning

confidence: 99%

Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review

Lasky

Carleton

Horton

et al. 2020

Drugs - Real World Outcomes

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“…Given these limitations, there has been growing interest in developing systems for active surveillance and study of medications in pediatric patients. Research infrastructures such as CER 2 , which are based on EHR systems, are especially attractive for pharmacovigilance because of the combination of medication and clinical data with longitudinal follow‐up . The need for clinical data and longitudinal follow‐up is particularly important for pediatric studies, as the response to medications may vary based on the age of the child and the long‐term effects may differ by timing and length of exposure over the various developmental stages…”

Section: Discussionmentioning

confidence: 99%

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

Czaja

Ross

Liu

et al. 2018

Pharmacoepidemiology and Drug

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“…2 million). 6,7 Because these networks are often practice based, they can also be used to obtain patient-reported data on effectiveness, adverse events, and preferences through innovative approaches such as the use of mobile health tools, including smartphone apps. These data, in combination, can then inform a more comprehensive risk-benefit assessment of medications by using 1 of numerous proposed methods.…”

Section: Effectiveness Research Throughmentioning

confidence: 99%

Beyond the Label: Steering the Focus Toward Safe and Effective Prescribing

et al. 2017

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Each year, hundreds of millions of prescription medications are dispensed to pediatric patients. 1 A significant proportion of prescriptions are used in an off-label manner, outside the specifications approved by the US Food and Drug Administration (FDA), rendering off-label prescribing a "public health issue for infants, children and adolescents, " as described by the Committee on Drugs for the American Academy of Pediatrics. 2 The committee also explicitly states that off-label use "does not imply an improper, illegal, contraindicated or investigational use. " Yet, when used in research, clinical practice, or even the lay media, the term off-label commonly carries a negative connotation. This interpretation probably reflects the sense of uncertainty in understanding the risk-benefit balance of a medication without FDA review and approval. However, prescribing according to the package insert does not necessarily translate to the safe and effective use of a medication. 3 The clinical trial data required for FDA approval often represent highly select populations in controlled settings with limited follow-up. These data may not translate well to real-world use, hence the need for postmarketing surveillance and research. Conversely, lack of pediatric labeling for a medication does not necessarily indicate a lack of evidence. It may simply mean the pharmaceutical company has not submitted an application for FDA approval to add a new indication or population.This apparent paradox gives us the opportunity to consider how we, the pediatric community of clinicians, researchers, and policymakers, think about the issue of off-label prescribing and how best to direct future efforts in medication research and policy. The Best Pharmaceuticals for Children Act prioritization process focuses on gathering expert opinion to inform research priorities related to therapeutic needs, with a primary goal of identifying drugs for pediatric clinical trials. 4 We argue that such approaches should be complemented by a structured consideration of risks and benefits informed by increasingly available data from practice. Pediatric research on off-label prescribing often focuses on the dichotomy of on-label versus off-label prescribing rates, defined mainly by age limits and occasionally indication. Understanding the extent of off-label practice is important, but it represents only a single dimension of a complex issue. This information does not necessarily determine whether the way a

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Pediatric post-marketing safety systems in North America: assessment of the current status

Cited by 11 publications

References 24 publications

Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review

Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review

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

Beyond the Label: Steering the Focus Toward Safe and Effective Prescribing

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