Preference-based utility weights for the Individualized Neuromuscular Quality of Life Questionnaire (INQoL), with a focus on non-dystrophic myotonia (NDM).

The hybrid modelling approach allows us to combine data from the two methodologies and maximize the information from each to inform the utility weights for the INQoL. The TTO is the more conventional valuation method, but combined with the larger DCE study produced better descriptive coverage. This is a relatively novel method for estimating weights which we think is particularly well suited to economic evaluations of orphan drugs.