Assessing Drug Safety in Children — The Role of Real-World Data

McMahon, Ann; Pan, Gerald J. Dal

doi:10.1056/nejmp1802197

Cited by 41 publications

(32 citation statements)

References 5 publications

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“…[70]. Studies using RWD offer the opportunity to assess long-term effects and the safety of medications used to treat chronic conditions (especially rare events) as well as the chance to study rare conditions [76]. Given the ethical and logistic constraints on the conduct of traditional pediatric clinical trials, it is incumbent upon us to foster the optimal use of RWD/RWE for the benefit of children.…”

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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“…[19][20][21] Finally, real-world data may be more suitable than RCTs for answering drug safety questions regarding rare adverse effects or adverse effects that present themselves years after the initial drug exposure. 18,22 To deliver the best medical practice tomorrow, it is important that we harness the full potential of the data collected today. At the moment, data in EHRs are still primarily collected for medical practice and may sometimes be ill suited for secondary use as research data.…”

Section: • Limited Control • Complex Data Analysis • No Expert Supervmentioning

confidence: 99%

“…In addition to data from site-centric RCTs, in which most data is collected in one or more physical study sites, 22 the collection of patient-centric data has the potential to increase the capacity for data collection (Figure 1). 22,23 Patient-centric data refers to data collected from the patient at home or during other parts of their daily routine. Depending on the context, data could be collected using mobile applications, wearables, and social media.…”

Section: Patient-centric Data Collectionmentioning

confidence: 99%

Beyond the Randomized Clinical Trial: Innovative Data Science to Close the Pediatric Evidence Gap

Goulooze

Zwep

Vogt

et al. 2020

Clin Pharma and Therapeutics

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Despite the application of advanced statistical and pharmacometric approaches to pediatric trial data, a large pediatric evidence gap still remains. Here, we discuss how to collect more data from children by using real‐world data from electronic health records, mobile applications, wearables, and social media. The large datasets collected with these approaches enable and may demand the use of artificial intelligence and machine learning to allow the data to be analyzed for decision making. Applications of this approach are presented, which include the prediction of future clinical complications, medical image analysis, identification of new pediatric end points and biomarkers, the prediction of treatment nonresponders, and the prediction of placebo‐responders for trial enrichment. Finally, we discuss how to bring machine learning from science to pediatric clinical practice. We conclude that advantage should be taken of the current opportunities offered by innovations in data science and machine learning to close the pediatric evidence gap.

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“…In light of these changes, exploring these questions in new settings and using new approaches may contribute to shed more light on the problem. Use of data from nationwide health registries is a new approach that makes it possible to address several of the limitations from earlier studies . Linking data from multiple registries allows for large, unselected study populations and identification of relevant comparison groups.…”

Section: Introductionmentioning

confidence: 99%

Prenatal exposure to opioid maintenance treatment and neonatal outcomes: Nationwide registry studies from the Czech Republic and Norway

Handal

Nechanská

Lund

et al. 2019

Pharmacology Res & Perspec

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There is lack of knowledge about the safety of treatment with methadone and buprenorphine as part of opioid maintenance treatment (OMT) during pregnancy. The purpose of this study was to examine neonatal outcomes concerning the use of OMT during pregnancy. We used nationwide registry linkages from the Czech Republic (2000‐2014) and Norway (2004‐2013). We compared prenatally OMT–exposed newborns with (a) newborns of women hospitalized with opioid use disorder during pregnancy in the Czech sample and (b) newborns with neonatal abstinence syndrome (NAS) in Norway. We performed multivariate linear and binary logistic regression exploring the associations between OMT and neonatal outcomes (growth parameters, gestational age, fetal death, small for gestational age, Apgar score, and NAS). Regression coefficients ( b ) and odds ratios (ORs) were estimated. The cohorts consisted of 333 Czech, and 235 Norwegian OMT–exposed newborns, and 106 and 294 newborns in the comparison groups, respectively. In both countries, the neonatal growth parameters were similar in the OMT and the comparison groups. In Norway, OMT exposure prolonged gestational age (adjusted b = 0.96 weeks, 95% confidence interval [CI] =0.39‐1.53) while the odds of preterm birth and Apgar score at 5 minutes were lower than in the comparison group (adjusted OR = 0.35, 0.16‐0.75 and aOR = 0.21, 0.06‐0.78, respectively). Newborns of women in OMT had similar growth parameters as newborns of women with opioid use disorders who were not in OMT during pregnancy. Overall, our findings do not suggest that OMT results in worse neonatal outcomes.

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Assessing Drug Safety in Children — The Role of Real-World Data

Cited by 41 publications

References 5 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

Beyond the Randomized Clinical Trial: Innovative Data Science to Close the Pediatric Evidence Gap

Prenatal exposure to opioid maintenance treatment and neonatal outcomes: Nationwide registry studies from the Czech Republic and Norway

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