| Summary: | Eliglustat (Cerdelga) is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are poor metabolizers, intermediate metabolizers, or extensive metabolizers of the enzyme cytochrome P450 2D6 (CYP2D6). The dosage is 84 mg once daily in CYP2D6 poor metabolizers, and 84 mg twice daily in CYP2D6 intermediate and extensive metabolizers. It is administered orally. The submitted price of eliglustat is $695 per capsule ($253,675 or $507,350 annually). The manufacturer submitted a cost-utility analysis comparing eliglustat with two enzyme replacement therapies (ERTs), imiglucerase and velaglucerase, in treatment-naive and treatment-stable adult patients with GD1 over a lifetime time horizon (up to 100 years). The cost-utility analysis is from the perspective of the Canadian public health care payer. The manufacturer assumed equivalence between eliglustat and the ERTs based on a review of available clinical trial data. The same transition probabilities for eliglustat and the ERTs for health states were adopted from the ENGAGE and ENCORE studies (short-term) and a retrospective cohort study (long-term). Data from manufacturer-commissioned reports and the published studies of eliglustat, imiglucerase, and velaglucerase were used to determine the discontinuation and adverse event rates applied in the model. Other inputs such as costs and utility values were obtained from published literature. In its base case, the manufacturer reported that eliglustat dominated (was more effective and less costly than) imiglucerase or velaglucerase. In a probabilistic sensitivity analysis of 50 simulations, at a willingness-to-pay threshold of $50,000 per quality adjusted life-year (QALY), imiglucerase or velaglucerase were preferred treatment strategies in approximately 40% of simulations
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