Purpose: Children with chronic conditions experience medical issues over long-term periods of time which can have lasting emotional and social consequences impacting daily life and functioning. Activities and participation outcomes are needed in order to comprehensively assess child-important health in clinical trials. Our objective was to review the extent to which activity and participation outcomes are included in clinical trials of childhood chronic disease and to determine what trial characteristics are associated with their use.
Methods: A review of a large clinical trial registration database (clinicaltrials.gov) was conducted over the 2010 calendar year. The measures used to assess primary and secondary endpoints were coded according to the ICF classification system. Trial characteristics that might be associated with activity and participation outcome use such as sponsorship type, intervention type, health condition, whether the trial was focused on pediatric patients, phase of trial and sample size were also extracted and explored with univariable and multivariable regressions.
Results: Four hundred and ninety-nine trials met inclusion criteria, 495 of which had complete information about hypothesized predictors. Only 36 out of 495 trials included an activity and participation outcome as part of the trial evaluation process. Both univariable and multivariable regression models showed that non-drug trials and late phase of trial (phase IV) showed the strongest likelihood with whether a trial would include an activity and participation outcome.
Discussion: Most registered clinical trials for children with chronic or ongoing medical conditions do not include a comprehensive approach to health outcomes assessment, especially drug trials and early phase trials. Outcome measures in pediatric clinical trials are lagging relative to World Health Organization standards for comprehensive health evaluation.