Figure 2
From: A methodological framework for drug development in rare diseases
Modeling of Dornase effect on mucociliary clearance in cystic fibrosis patients: simulation of 7 RCTs experimental designs and comparative assessment of design performances. 50 patients were included per trial (except N-of-1). (a) Power expressed in %, (b) Variability (coefficient of variation of results) expressed in %, (c) No. of patients under active treatment, (d) Trial duration expressed in arbitrary units.