Patients with late-onset Pompe disease (PD) seem to rate and describe health state utility values differently than the general population, according to a study recently published in the Journal of Health Economics and Outcomes Research.
“The health state valuation results capture the progressive nature of the disease and highlighted the decline in [health-related quality of life] with increased dependence on assistive technology, which was consistently observed in the [visual analog scale], [time trade-off], and EQ-5D-5L utilities,” the authors explained.
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This observational study included 7 health state vignettes pertinent to the adult population regarding the level of mobility and ventilatory support. These included: no support, intermittent mobility support, intermittent ventilatory support, intermittent ventilatory and mobility support, mobility support-dependent, mobility support-dependent and intermittent ventilatory support, and mobility support-dependent and invasive ventilatory support-dependent. Later, 4 other statements inquired about dependence on others, impaired bladder control, balance problems and/or fear of falling, and frustration.
The researchers conducted 100 interviews within the United Kingdom, observing mean time trade-off utilities that ranged from 0.123, corresponding to mobility support- and invasive ventilatory support-dependent, to 0.754, which related to no support.
Moreover, the EuroQol (EQ-5D-5L) scoring system showcased scores between -0.078 and 0.608. These values correlate with those previously reported in the literature.
The results of this study emphasize the great burden patients with late-onset PD carry and its negative effects on their quality of life. Importantly, the general population consistently rated health states lower with disease progression. In addition, the data display an overall uncertainty regarding utility estimates for the severe states. Hence, many participants had a harder time rating these states.
Although utility values for the early stages of PD have been described, these findings greatly contribute to understanding utilities in advanced PD states.
“Our findings highlight the high disease burden of [late-onset PD] and reinforce the societal value of slowing disease progression,” the authors concluded.
Hubig L, Sussex AK, MacCulloch A, et al. Quality of life with late-onset Pompe disease: qualitative interviews and general public utility estimation in the United Kingdom. J Health Econ Outcomes Res. Published online March 3, 2023. doi:10.36469/001c.68157