OBJECTIVES: The progression of DMD is characterized by loss of ambulation (LOA) and upper limb function, cardiorespiratory impairment, and early mortality. Published utility values are limited, and generally restricted to health states defined solely by ambulatory status. The objective of this study was to estimate societal utility values for health states describing the progression of DMD.

METHODS: Health states characterizing DMD progression were developed based on literature review, clinical expert consultation, and qualitative interviews with 8 patients with DMD and 26 caregivers. Members of the US general public were recruited from a market research panel, online advertising, and social media. Time trade-off utility values were elicited by a trained interviewer over Zoom, using health state descriptions and visual aids. Demographics were summarized and mean (standard deviation [SD]) utility values summarized for a set of core health states. Trends were explored according to participant sex and age (categorized as 18-24, 25-44, 45-64, and >65 years).

RESULTS: Of 190 participants, 98 (51.6%) were female and the mean (SD) age was 47 (15.3) years. Mean (SD) utility values ranged from 0.74 (0.29) for preserved/mildly impaired ambulatory function; 0.65 (0.33) for moderately/severely impaired ambulatory function; 0.48 (0.34) for non-ambulatory with preserved/mildly impaired upper limb function and non-invasive ventilation; and 0.27 (0.31) for non-ambulatory with severe loss of upper limb function and non-invasive ventilation. Utility values were relatively consistent when stratified according to participant sex; but tended to be lower with higher participant age until the oldest age category.

CONCLUSIONS: In DMD, utility values decrease in health states describing progressively more severe DMD symptoms. While utility values for the preserved ambulatory function health state are consistent with other published estimates, utility values for the range of non-ambulatory health states are higher than previous estimates that did not explicitly account for levels of upper limb function.