Article Text

Download PDFPDF
Making a difference: the clinical research programme for children
  1. Rosalind L Smyth
  1. Rosalind L Smyth, School of Reproductive and Developmental Medicine, University of Liverpool, Alder Hey Children’s Hospital, Liverpool L12 2AP, UK; r.l.smyth{at}

Statistics from

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.

High quality paediatric clinical research will ensure that tomorrow’s children receive new and better treatments

All of us involved in the clinical care of children have a duty to improve that care and one way of achieving this is through research. Attention has rightly been drawn to the lack of clinical trials which have addressed issues of relevance to children’s health.1 There are some demoralising statistics to support these arguments. For example, a review of clinical trials published in this journal over 15 years found that a high proportion had important methodological flaws and in around half the sample size was less than 40.2 There are similar findings in paediatric specialities,3 and in community paediatrics only 40% of decisions were supported by research evidence.4 Yet we are all aware of the dramatic impact which the results of clinical trials have had on the care and survival of children with malignant disease and those born preterm. To illustrate the impact of high quality research on children’s health worldwide, I have tried to identify some of the most important clinical trials which have benefited children.


It is tempting, when addressing such a topic, to consider the most important trials within one’s own practice, but I tried to provide a more objective assessment, of broad relevance to paediatricians, by using the number of citations of the study report as an approximate measure of the impact of the trial. All measures of “impact” are subject to limitations, and papers describing clinical trials may be widely cited for reasons other than their importance to clinical care, for example if their findings are controversial, they address a topical disease area, or they were published a long time ago. So, aware of these limitations, but in the absence of a better measure of …

View Full Text


  • Competing interests: The author is Director of the UK Medicines for Children Research Network.

Linked Articles

  • Précis
    BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health
  • Atoms
    Howard Bauchner