People making choices in health care should use the findings of clinical trials (ideally, incorporated in systematic reviews) to inform their decisions. If these findings are to be useful and reliable, researchers must select appropriate outcomes, measure them in a scientifically robust manner, and report results thoroughly. There are difficulties, however, relating to the selection and measurement of outcomes in clinical trials, and special considerations are needed when these studies are conducted in children.

This article provides guidance for researchers working on clinical trials in children. Although this article is focused on trials of effectiveness, which are similar to therapeutic confirmatory trials, certain sections may also relate to efficacy trials, pharmacokinetic trials, therapeutic exploratory trials, and trials conducted earlier in drug development.

To be useful, clinical trials that evaluate potential benefits and harms of health care interventions must measure outcomes of relevance to practitioners and patients who make shared decisions...

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