Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method

Preferences between treatment characteristics can be understood and quantified by combining discrete choice experiment (DCE) and time trade-off (TTO) (DCETTO) methods. The goal is to elicit preferences for people with hemophilia A (PWHA) and quantify impacts of treatment attribute changes on patient health utility with DCETTO. A total of 119 PWHA from a hemophilia treatment center and the National Bleeding Disorders Foundation Community Voices in Research were recruited. Their sociodemographics, clinical characteristics, and EQ-5D-5L scores were gathered. Treatment attributes for the DCE survey were based on the core outcome set for hemophilia gene therapy (coreHEM). For DCETTO, 10-, 15-, and 20-year life durations were used. In total 115 PWHA completed 12 DCE and DCETTO tasks, which were analyzed using conditional logistic models. Moderately burdensome treatment (24% treat > once/week) was reported by 67%. Mean EQ-5D-5L visual analogue score was 75; mean utility score was 0.684. All DCE attributes were significant; treatment administration was most important (2–3 intravenous [IV] infusions/week vs. a single IV infusion with 10-year durability, regression coefficient [RC], − 1.99), followed by mental health (always vs. no concern, RC, − 1.37), annual bleeding frequency (≥ 5 vs. none, RC, − 0.73), and chronic pain (yes vs. no, RC, − 0.36). For DCETTO, multiple IV infusions weekly and multiple monthly subcutaneous (SQ) injections were associated with annualized utility decrements (0.046 vs. 10-year durability, 0.044 vs. 5-year; 0.037 vs. 10-year, 0.030 vs. 5-year, respectively). All coreHEM outcomes are important for treatment choices of PWHA. One-time IV infusion with 5- to 10-year durability can provide important patient utility gains over lifelong repeated SQ injections or IV infusions.