OBJECTIVES: Many health technology assessments require generic measures like the EQ-5D or HUI to estimate HRQoL impact. The EQ-5D and HUI descriptive systems cover similar HRQoL dimensions; however, they differ in how impact on these dimensions is assessed. The objective was to compare the content validity of the EQ-5D and HUI for assessing HRQoL in DMD.

METHODS: Individuals with DMD <40 years or caregivers reporting on their behalf completed the EQ-5D and HUI. Cognitive debriefing interviews were conducted to understand the relevance of 1) dimensions included within both descriptive systems, and 2) response options on survey questions related to those dimensions. Selected EQ5D dimensions included mobility, anxiety/depression, pain/discomfort and self-care; vs ambulation, emotion, pain, and ability to perform basic activities on the HUI. Data were analyzed through content analysis.

RESULTS: Sixteen assessments (7 caregiver-reports and 9 patient-reports) were included in this analysis. Mean patient age was 19 years and 12 were non-ambulatory. All participants regarded the dimensions covered within both the EQ-5D and HUI as relevant to living with DMD in general, even if that dimension was no longer relevant to their current DMD disease state. Most participants found it easier to classify health status on the HUI because response options were more granular; participants also reported some HUI response options were relevant for a longer timeframe in their DMD course. Participants had difficulty with response options that assumed an impact occurred at a constant level; or when the question referenced multiple activities grouped together but an individual’s ability to perform those different activities varied.

CONCLUSIONS: While both instruments assess DMD-relevant dimensions, HUI response options allowed an easier classification of current health status. With DMD symptom progression, the HUI permitted more nuanced categorization for individuals with more varied health statuses. These patient-derived data can help inform instrument selection in HRQoL studies using generic instruments.