Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt

Objectives: Ataluren and eteplirsen are orphan drugs that delay progression of Duchenne muscular dystrophy in mutationspecific subgroups. They have yet to be approved in Egypt but are expected to reach the market soon. This study describes 2 cost-utility models comparing the drugs with the standard of care.Methods: We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years. Baseline curves were obtained from a published model; then the ambulation loss curve was updated using the Kaplan-Meier curve of the standard of care from a study by McDonald et al. Other curves were updated by calibration to this curve. Costs and utilities were from a local study. Deterministic and probabilistic sensitivity analyses were conducted. Prices were estimated based on other orphan drugs' prices.Results: In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial. The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs. At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial.Conclusions: Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs.