Comparing drug effectiveness in children: A systematic review

Dukanovic, Julijana; Osokogu, Osemeke U.; Patel, Krupa; Ferrajolo, Carmen; Sturkenboom, Miriam

doi:10.1002/pds.4676

Cited by 2 publications

(1 citation statement)

References 17 publications

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“…Despite the limitations of our search strategy, we note that our yield (29 eligible studies out of 915 unique citations) was 3.2%, comparable to that obtained in a recent systematic review on a similar topic. Dukanovic et al, in a review of comparative effectiveness studies in children, found that 4.2% of the studies were eligible (164/3926; the large denominator here reflects their inclusion of studies from the inception of Embase and Medline) [69]. While comparative effectiveness is not the same topic as RWE, it may be similar enough to provide an indication of a reasonable yield for most search strategies.…”

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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Drug Safety in Translational Paediatric Research: Practical Points to Consider for Paediatric Safety Profiling and Protocol Development: A Scoping Review

Aurich

Jacqz-Aigrain

2021

Pharmaceutics

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Translational paediatric drug development includes the exchange between basic, clinical and population-based research to improve the health of children. This includes the assessment of treatment related risks and their management. The objectives of this scoping review were to search and summarise the literature for practical guidance on how to establish a paediatric safety specification and its integration into a paediatric protocol. PubMed, Embase, Web of Science, and websites of regulatory authorities and learned societies were searched (up to 31 December 2020). Retrieved citations were screened and full texts reviewed where applicable. A total of 3480 publications were retrieved. No article was identified providing practical guidance. An introduction to the practical aspects of paediatric safety profiling and protocol development is provided by combining health authority and learned society guidelines with the specifics of paediatric research. The paediatric safety specification informs paediatric protocol development by, for example, highlighting the need for a pharmacokinetic study prior to a paediatric trial. It also informs safety related protocol sections such as exclusion criteria, safety monitoring and risk management. In conclusion, safety related protocol sections require an understanding of the paediatric safety specification. Safety data from carefully planned paediatric research provide valuable information for children, parents and healthcare providers.

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Comparing drug effectiveness in children: A systematic review

Cited by 2 publications

References 17 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

Drug Safety in Translational Paediatric Research: Practical Points to Consider for Paediatric Safety Profiling and Protocol Development: A Scoping Review

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