Standard 5: Selection, Measurement, and Reporting of Outcomes in Clinical Trials in Children

Abstract

* Abbreviations: CONSORT — : Consolidated Standards of Reporting Trials OMERACT — : Outcome Measures in Rheumatology PROM — : patient-reported outcome measure 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 about treatment options; regulatory authorities who consider applications for marketing authorizations for medicines; organizations who decide whether to provide funding for an intervention (eg, health care commissioners, insurance companies); and policy makers interested in the impact of an intervention. It can be difficult, however, to decide which outcomes to measure in clinical trials. One reason is that the impact of illnesses is very variable, and some, but not all, may be improved by an intervention. Various frameworks for considering the effects of illnesses have been suggested,1–4 and others are in development. It may not be immediately obvious which of these effects are of particular importance, and the key aspects of the effects of health care interventions might vary between trials. For example, domains such as short-term measures of disease activity, prevention of symptoms, functional status, longer-term consequences of the illness, overall well-being, and utilization of health care … Address correspondence to Rosalind L. Smyth, Department of Women’s and Children’s Health, Institute of Translational Medicine, University of Liverpool, Liverpool, United Kingdom. E-mail: r.l.smyth{at}liv.ac.uk