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UK Medicines for Children Research Network
The randomised controlled trial is the primary research tool for assessing and developing medical therapies, and undertaking high quality trials must be a central activity for any modern health system. Richard Peto has presented compelling arguments for large simple randomised controlled trials (RCTs) as the main way to control bias and random error, and points out that carefully combining large size and simplicity of design is a highly successful strategy for detecting moderate benefits from treatments.1
Within paediatrics there are challenges to this simple and appealing approach.2,3 For example, in chronic illnesses, the burden of disease in children is usually disproportionately small compared with adults; diagnostic criteria may be less precise and more difficult to apply; and clinical outcomes, used routinely in clinical trials in adults, may be either impossible for children to perform or lack an age specific measure (such as quality of life). At a local level, there are many barriers to recruitment to multicentre clinical trials. A recent study evaluated the attitudes and beliefs held by paediatricians about clinical trials and made a number of interesting observations.4 Firstly, the perception of many paediatricians was that, in many instances, the risks to children of participating in a clinical trial outweighed the gains. Paediatricians’ personal treatment preferences were seen as a hindrance to support for a clinical trial and clearly many paediatricians are often not truly in equipoise around the research question being addressed by the clinical trial. In general, paediatricians with previous research experience were most knowledgeable about RCTs and perceived greatest gains from the participation of their patients in clinical trials. However, even clinicians wishing to be involved in trials will admit that there has been, until recently, a lack of infrastructure to support clinical trials.