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Review
. 2015 Mar;79(3):357-69.
doi: 10.1111/bcp.12305.

Clinical trials in children

Pathma D Joseph  1 , Jonathan C Craig, Patrina H Y Caldwell
Affiliations
Review

Clinical trials in children

Pathma D Joseph et al. Br J Clin Pharmacol. 2015 Mar.

Abstract

Safety and efficacy data on many medicines used in children are surprisingly scarce. As a result children are sometimes given ineffective medicines or medicines with unknown harmful side effects. Better and more relevant clinical trials in children are needed to increase our knowledge of the effects of medicines and to prevent the delayed or non-use of beneficial therapies. Clinical trials provide reliable evidence of treatment effects by rigorous controlled testing of interventions on human subjects. Paediatric trials are more challenging to conduct than trials in adults because of the paucity of funding, uniqueness of children and particular ethical concerns. Although current regulations and initiatives are improving the scope, quantity and quality of trials in children, there are still deficiencies that need to be addressed to accelerate radically equitable access to evidence-based therapies in children.

Keywords: clinical trials; ethics; medicines in children; paediatric drug therapy.

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Figures

Figure 1
Figure 1
Timeline of paediatric regulations in the US and EU
See this image and copyright information in PMC

References

    1. Wilson JT. An update on the therapeutic orphan. Pediatrics. 1999;104(Suppl. 3):585–590. - PubMed
    1. Shirkey H. Therapeutic orphans. J Pediatr. 1968;72:119–120. - PubMed
    1. Shirkey H. Editorial comment: therapeutic orphans. J Pediatr. 1999;104:583–584. - PubMed
    1. Sammons H, Gray C, Hudson H, Cherrill J, Choonara I. Safety in paediatric clinical trials – a 7-year review. Acta Paediatr. 2008;97:474–477. - PubMed
    1. Klassen TP, Hartling L, Hamm M, van der Lee JH, Ursum J, Offringa M. StaR Child Health: an initiative for RCTs in children. Lancet. 2009;374:1310–1312. - PubMed

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