Drug toxicity in children: paediatric randomised controlled drug trials and global child health

Nor Aripin, Khairun Nain Bin (2010) Drug toxicity in children: paediatric randomised controlled drug trials and global child health. PhD thesis, University of Nottingham.

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Concern with potential toxicity due to the widespread use of unlicensed and off label drugs in children has led to regulatory changes aimed to strengthen the evidence base for paediatric drugs. This thesis examines paediatric randomised controlled trials (RCTs), the highest level of evidence, and assesses them in relation to global child health.

A systematic review was performed using validated methods to search three major databases for paediatric RCTs published in 2007. More than 600 RCTs were identified involving more than 100,000 children. The RCTs appear to study the appropriate clinical areas however few studies involved neonates. The RCTs also seem to be of good methodological quality with a mean Jadad score of 3.22.

The reporting of RCTs that involve both adults and children needs to be improved to add to the evidence base of paediatric medicines. More attention is also needed on the reporting of safety information from the RCTs to provide useful toxicity data. Although severe and moderate ADRs were seen in 25% of the RCTs, few RCTs (12%) established safety monitoring committees (SMCs). SMCs are vital to ensure patients in paediatric RCTs are protected from toxicity.

The burden of childhood disease is heaviest in low and middle income countries (LMIC). A minority of the RCTs were performed in LMIC, although they are increasingly globalised. RCTs conducted in LMIC appear to have lower methodological quality, and reported less well on ethical approval and adverse events.

In conclusion high quality, ethical paediatric RCTs should add to the evidence base for paediatric medicines. However they should correspond with the health needs of children on a global basis.

Item Type: Thesis (University of Nottingham only) (PhD)
Supervisors: Choonara, I.A.
Sammons, H.
Subjects: QS-QZ Preclinical sciences (NLM Classification) > QV Pharmacology
Faculties/Schools: UK Campuses > Faculty of Medicine and Health Sciences > School of Graduate Entry Medicine and Health
Item ID: 11506
Depositing User: EP, Services
Date Deposited: 08 Apr 2011 10:25
Last Modified: 15 Oct 2017 09:35
URI: https://eprints.nottingham.ac.uk/id/eprint/11506

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