Estimating health-state utility values for family-caregivers of patients with Duchenne muscular dystrophy using time trade-off valuation

Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disease. Long-term care is primarily provided by unpaid family-caregivers. This time trade-off (TTO) study aimed to generate family-caregiver utility values associated with different stages of DMD. Eight vignettes were developed to reflect the experience of caring for patients with DMD across health states (HS) defined in the Project HERCULES model. Vignettes were informed by literature and input from family-caregivers and healthcare professionals. TTO interviews were conducted online with members of the UK general public. Participants reviewed all vignettes and completed HS ranking, visual analog scale (VAS) ratings, and TTO valuation tasks. 200 participants (mean age 44.0 years, 51% female) completed interviews. Mean utility scores were highest for HS1 (early ambulatory: 0.717) and lowest for HS8 (no hand-to-mouth function, full-time ventilation: 0.477). VAS scores showed a similar pattern, with HS1 scoring 65.49 and HS8 scoring 31.665. This study highlights the increasing burden on family-caregivers as DMD progresses, with declining health-related quality of life across HS. The resulting utility values support inclusion of family-caregiver outcomes in future DMD cost-effectiveness models.