Statistical analysis of Goal Attainment Scaling endpoints in randomised trials.

ABSTRACT: Goal Attainment Scaling is an assessment instrument to evaluate interventions on the basis of individual, patient-specific goals. The attainment of these goals is mapped in a pre-specified way to attainment levels on an ordinal scale, which is common to all goals. This approach is patient-centred and allows one to integrate the outcomes of patients with very heterogeneous symptoms. The latter is of particular importance in clinical trials in rare diseases because it enables larger sample sizes by including a broader patient population. In this paper, we focus on the statistical analysis of Goal Attainment Scaling outcomes for the comparison of two treatments in randomised clinical trials. Building on a general statistical model, we investigate the properties of different hypothesis testing

SUBMITTER: Urach S 

PROVIDER: S-EPMC6566461 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature