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Clinical trials in children: Equity, quality and relevance

Abstract

This thesis investigates the equity, quality and relevance of clinical trials in children to inform better evidence-based child healthcare and outcomes worldwide. A comprehensive review of the literature revealed that despite current initiatives to encourage more trials in children, there is still a paucity of safety and efficacy data of many medicines prescribed in this population. An analysis of trials registered in children showed that disease burden was moderately correlated to trials and this scarcity was particularly prevalent in low-and middle-income countries. We explored the contributory factors to this inequity by conducting a systematic review of stakeholders’ views of trials in children in low-and middle-income countries. In the study evaluating the completeness of protocols of trials in children submitted to ethics committees, we found that protocols are generally comprehensive, but many key domains in trial design and conduct are not reported. Key-informant trial stakeholders who were interviewed proposed strategies to improve trials such as addressing the unique needs of children, embedding trials as part of routine clinical care and streamlining regulatory approvals. Increasing international collaboration, establishing sustainable centralised trials infrastructure, and aligning research to child health priorities were proposed to encourage more high-quality trials that address global child healthcare needs

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