Posted on 23 June 2023
There are challenges associated with recruiting children to take part in research and as a result, compared to adults, for many diseases, we are less certain about which treatments are most safe and effective. This can lead to weaker recommendations about which treatments to prescribe to patients. However, it may be possible to include adult evidence to improve our understanding of which treatments work best in children, and many different statistical methods are available to conduct these analyses.
In this paper, Ruth and colleagues discuss these different statistical methods and explore which might lead to the most reliable results in children. They demonstrate that methods that can account for scenarios where children’s responses to medical intervention differ from adults may be the 'safer' option and produce more reliable estimates of treatment effects in children.
Ruth Walker says "These methods are applicable in many areas within paediatric research where evidence is sparse. Evidence from adult trials may help to improve our understanding of how well medicines work in children and help to reach conclusions about new medicines for children and young people sooner, without needing to do so many trials in children and young people. Instead, smaller clinical trials could be used to confirm findings."
This work has informed the statistical methods currently being used in an NIHR Doctoral Fellowship award held by Ruth Walker that aims to improve our understanding of which anti-sickness medicines work best for preventing chemotherapy-induced nausea and vomiting in children and young people undergoing treatment for cancer.
This research was conducted by Ruth Walker, Sofia Dias and Bob Philipps and was funded through an NIHR Pre-Doctoral Fellowship award held by Ruth Walker.
Walker R, Phillips B, Dias S. Comparison of Bayesian methods for incorporating adult clinical trial data to improve certainty of treatment effect estimates in children. PLoS ONE 2023;18:e0281791