Statistical issues in the assessment of health outcomes in children: a methodological review

The lack of outcome measures that are validated for use on children limits the effectiveness and generalizability of paediatric health care interventions. Statistical epidemiology is a broad concept encompassing a wide range of useful techniques for use in child health outcome assessment and development. However, the range of techniques that are available is often confusing and prohibits their adoption. In the paper an overview of methodology is provided within the paediatric context. It is demonstrated that in many cases assessment can be performed relatively straightforwardly by using standard statistical techniques, although sometimes more sophisticated techniques are required. Examples of both physiological and questionnaire-based outcomes are given. The usefulness of these techniques is highlighted for achieving specific objectives and ultimately for achieving methodological rigour in clinical outcome studies that are performed in the paediatric population.