Health utilities and costs for neuromyelitis optica spectrum disorder

Background Neuromyelitis optica spectrum disorder (NMOSD) is a rare, neurological disease that places a significant burden on patients, their carers, and healthcare systems. Objectives To estimate patient and carer health utilities and costs of NMOSD within the UK setting. Methods Patients with NMOSD and their carers, recruited via a regional specialist treatment centre, completed a postal questionnaire that included a resource use measure, the EuroQoL (EQ)-5D-5L, EQ-5D-VAS, Vision and Quality of Life Index (VisQoL), Carer Experience Survey (CES) and the Expanded Disability Status Scale (EDSS). The questionnaire asked about respondents' use of health and community care services, non-medical costs, informal care and work capacity. Data were analysed descriptively. Uncertainties in costs and utilities were assessed using bootstrap analysis. Results 117 patients and 74 informal carers responded to the survey. Patients' mean EQ-5D-5L and VisQoL health utilities (95% central range) were 0.54 (- 0.29, 1.00) and 0.79 (0.11, 0.99), respectively. EQ-5D-5L utility decreased with increasing EDSS score bandings, from 0.80 (0.75, 0.85) for EDSS <= 4.0, to 0.20 (- 0.29, 0.56) for EDSS 8.0 to 9.5. Mean, 3-month total costs were 5623 pound (2096 pound, 12,156) pound, but ranged from 562 pound (381 pound, 812) pound to 32,717 pound (2888 pound, 98,568) pound for these EDSS bandings. Carer-reported EQ-5D-5L utility and CES index scores were 0.85 (0.82, 0.89) and 57.67 (52.69, 62.66). Mean, 3-month costs of informal care were 13,150 pound to 24,560 pound. Conclusions NMOSD has significant impacts on health utilities and NHS and carer costs. These data can be used as inputs to cost-effectiveness analyses of new medicines for NMOSD.