Patient and Caregiver Health State Utilities in Lennox-Gastaut Syndrome and Dravet Syndrome

PurposeLennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are rare, treatment-resistant forms of epilepsy characterized by childhood onset of seizures. Cost-effectiveness analysis for new antiseizure medications typically requires health state utilities (HSUs) that reflect the patient and caregiver burden of the relevant conditions. This study aimed to estimate HSUs for LGS and DS. Focus was placed on valuing the impact of seizure frequency and seizure-free days on health-related quality of life (HRQL) for patients and caregivers.MethodsHealth state vignettes that described the experience of living with and caring for a child with LGS or DS were developed based on a targeted literature review and feedback from interviews with LGS and DS clinical experts and DS caregivers. Vignettes varied by the number of seizures and seizure-free days per month. Twenty-four vignettes were developed that described patient HRQL (6 per condition) and caregiver HRQL (6 per condition) for LGS or DS. Vignettes were evaluated via interviews from the general population in the United Kingdom and Sweden using a visual analog scale and time trade-off (TTO) method. Participants were recruited by interviewers based in different regions of the United Kingdom and Sweden using convenience sampling.FindingsTwo hundred interviews were conducted by video call from March to April 2020. One hundred participants evaluated each of the 6 patient and 6 caregiver vignettes for LGS; a different 100 participants evaluated each of the DS vignettes. The estimated utility values vary consistently according to seizure frequency and number of seizure-free days. Patient TTO utility values range from −0.186 (highest seizure frequency and fewest seizure-free days) to 0.754 (seizure-free state) for LGS and from 0.171 to 0.778 for DS. The caregiver TTO utility values range from 0.032 to 0.810 and 0.510 to 0.881 for LGS and DS, respectively. Fewer seizures and additional seizure-free days are associated with better patient and caregiver HRQL.ImplicationsThis study estimated utility values for patients with LGS or DS and their caregivers using visual analog scale and TTO valuation among a general public sample. These utility values can be used in cost-effectiveness assessments of new antiseizure medications. The findings indicate the importance of seizure-free days as well as seizure frequency in patient and caregiver HRQL, which may inform the design of future clinical trials.