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Unformatted text preview: Review The importance of clinical trials in children Clinical trials in children have resulted in significant improvements in their health care. A well-known example is childhood acute lymphoblastic leukaemia, in which the 5-year survival improved from 25% to more than 70% as a result of multicentre trials. 1 Unfortunately, since there are few paediatric trials, 2 the list of improvements in child health resulting from clinical trials is not long and is restricted to some childhood diseases, heavily clustering around cancer. Consequently, many ineffective and even harmful interventions are used in children before they have been appropriately assessed in randomised trials, 3,4 and other useful interventions have had a delayed introduction into practice. In the absence of specific trial-based data in children, clinicians, families and policy-makers are forced to extrapolate from results of studies in adults. This extrapolation is often inappropriate because children have a different range of diseases, and metabolise medications differently, resulting in responses to treatment that are unpredictably different to adults. 2,5,6 For example, the adverse effects to medications such as thalidomide (phocomelia in the unborn child), (staining of the teeth), (the grey baby syndrome), and aspirin (Reyes syndrome in children with viral infections) are specific to children. Benefits of trial participation Participants in randomised controlled trials (RCTs) derive many benefits, including access to new treatments that might not be routinely available. The Declaration of Helsinki requires that treatment offered to the control group should be the current best standard treatment, and that those allocated to the experimental group receive a treatment proposed to be as good as or better than standard treatments. 7 Hence, a well-designed RCT could arguably offer a patient the optimum treatment approach. 8,9 However, studies that breach this provision are still sometimes done to gain regulatory approval. 10 There might be additional benefits for patients who receive treatment at a hospital or institution involved in RCTs. In studies in adults, doctors who participate in clinical trials are more likely to incorporate trial findings and published data into clinical practice. 11 Many reports show inclusion benefits for all trial participants, including children (the Hawthorn effect). 5,12,13 Participants of RCTs, including those assigned to placebo, have outcomes similar to or better than those of eligible non-participants. Participants have lower mortality, fewer clinical events, and lower complication rates than similar patients treated outside RCTs. This survival advantage is not explained by differences in pre-treatment disease status or factors of known prognostic importance....
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This note was uploaded on 01/01/2012 for the course PHRM 7230 taught by Professor Tackett during the Spring '11 term at University of Georgia Athens.
- Spring '11
- The Land