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It is 100 years since Robert Grieve Hutchison first described the entity that we now recognise as neuroblastoma with bony metastases. Since then, we have moved from a situation where virtually all children with malignancy died to one where more than three-quarters of patients will be long-term survivors. Nevertheless, of the 1400 children with cancer seen in the UK each year, on average around 350 will die, each death untimely and tragic. Much of the early improvement in outcome was due to developments in anaesthesia and surgery, the advent of effective supportive care, and the discovery of effective drugs, such as the folic acid antagonists and adrenocorticoids for the treatment of acute lymphoblastic leukaemia (ALL).1 The development of multiagent regimens led to further improvements and saw the advent of large multicentre cooperative treatment groups such as the UK Children’s Cancer Study Group (UKCCSG), the US Children’s Cancer Group (CCG) and the International Society of Paediatric Oncology (SIOP), which made use of formally structured clinical trials. The randomised clinical trial has thus been the mainstay of paediatric oncological practice for decades.
Classically, a trial is run to evaluate the ability of new drugs or combinations of drugs to increase the proportion of children who are cured of their malignancy. Over the past few decades, increasing numbers of trials have been conducted with the aim of reducing therapy so that patients are cured with fewer treatment-related sequelae. During the 1980s and 1990s, the great majority of patients attending most paediatric oncology centres in the UK were entered into clinical trials. However, that period of endeavour and achievement seems now to be drawing to a close. Recently, not only in the UK but also across Europe as a result of the EU Clinical Trials Directive2 and its associated national legislation, there …