Evidence-based guidelines for pediatric clinical trials: focus on StaR Child Health

Abstract

Clinical trials in children are challenging and filled with important ethical considerations that differ from adults. Given difficulties associated with pediatric clinical trials, off-label prescribing is a common practice in pediatrics, which can lead to adverse safety events and efficacy failures. To overcome these consequences, in the past 15 years, legislation in the USA and Europe has provided incentives to industry and increased government funding to conduct pediatric trials. Pediatric trial networks have also been formed to decrease the knowledge gap. However, challenges to performing pediatric trials and lack of standardization and guidelines regarding studies in children still exist. Standards for Research (StaR) in Child Health, begun in 2009, aims to improve the design, conduct and reporting of pediatric trials. This organization uses a consensus guideline approach involving academic, government and industry stakeholders to identify and disseminate best practices for pediatric trials. Six out of 11 planned standards are currently published.

Keywords::

• clinical trials
• guidelines
• pediatrics

Financial & competing interests disclosure

MR Sampson is supported by a training grant (1 T32 GM 86330-1 A1) from the US NIH, National Institute of General Medical Sciences. M Cohen-Wolkowiez received support from NICHD 1K23HD064814-01 for his work in pediatric clinical pharmacology and from industry for neonatal and pediatric drug development (www.dcri.duke.edu/research/coi.jsp). DK Benjamin received support from the US Government for his work in pediatric and neonatal clinical pharmacology (1R01HD057956-02, 1R01FD003519-01, 1U10-HD45962-06, 1K24HD058735-01 and Government Contract HHSN267200700051C), the nonprofit organization Thrasher Research Foundation for his work in neonatal candidiasis (www.thrasherresearch.org), and from industry for neonatal and pediatric drug development (www.dcri.duke.edu/research/coi.jsp). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Notes

DMC: Data-monitoring committee.

Data taken from [102].