Therapeutic trials in childhood ALL: what's their future?

Evidence based medicine

Since the first randomised controlled medical research trial in the United Kingdom on the use of streptomycin in tuberculosis (1947–1948),1 such trials have progressively become the gold standard by which the choice of treatment for patients with any particular disease, and the evidence to support such choice, can be judged.2 In the 1940s there were worries that doctors would be “unwilling to relinquish the doctrine of anecdotal experience” and accept the idea of any individual patient being randomised between a new treatment and a placebo, nothing at all, or what was perceived as standard treatment up until that time.3 Ultimately the physician always must decide for his or her patient what should be recommended, based on an assessment of the patient's overall health status, and of course the doctor's own experience and awareness, modified by external evidence. There is no doubt that the physician either hearing a presentation of a similar case or cases or reading of other peoples' experiences will be influenced by such evidence. It is therefore essential that such evidence must be as objective, balanced, and unbiased as possible. The perfectly conducted randomised controlled trial should be the optimal way to produce such evidence. The impetus to collate all information for either the treatment or the prevention of disease has been increased by greater public awareness and education on health matters, coupled with easier access to information, which regrettably is not always reliable in the media or over the internet. In the Western world certainly, the public increasingly see through dogma and the “trust me I'm a doctor” approach. Development of the methodology to combine results from randomised controlled trials into systematic overviews is enhancing the ability of researchers to reach conclusions, or confirm evidence from individual trials. Such overview analysis has been particularly …