The Cost-Effectiveness of Eteplirsen vs. Standard of Care in Delaying the Progression of Duchenne Muscular Dystrophy
Author(s)
Kejsi Begaj, MS, PharmD1, Leo Yi-Ru Chen, MS, PharmD1, Kamil Branicki, PharmD2, Kosha Ravani, PharmD2, Monya Elrais, PharmD2, Olamide C. Olujohungbe, MS, PharmD1, Laura A. Clark, BS, MS, PhD1;
1Rutgers University, Center of Health Outcomes, Policy and Economics, New Brunswick, NJ, USA, 2Rutgers University, Ernest Mario School of Pharmacy, New Brunswick, NJ, USA
1Rutgers University, Center of Health Outcomes, Policy and Economics, New Brunswick, NJ, USA, 2Rutgers University, Ernest Mario School of Pharmacy, New Brunswick, NJ, USA
Presentation Documents
OBJECTIVES: Duchenne Muscular Dystrophy (DMD) is a progressive genetic disorder that affects1 in 3,500 males worldwide. Corticosteroids (i.e., standard of care [SoC]) delays progression to non-ambulatory states, but with limited long-term benefits. Eteplirsen has shown potential improvement in health-related quality of life (HRQoL) and delayed progression. The aim of this study was to analyze the cost-effectiveness of eteplirsen vs. SoC.
METHODS: A predictive Markov state-transition model was developed to simulate the progression of two DMD cohorts in the US. Costs and quality-adjusted life years (QALYs) were derived from published literature for male DMD patients diagnosed at 5 years old utilizing a societal perspective, including direct and indirect costs (adjusted for inflation in 2023). A time horizon of 25 years with a 1-year cycle shift and transition probabilities were also derived from published literature. A cost-effectiveness analysis was conducted to estimate the incremental cost-effectiveness ratios (ICERs) of eteplirsen vs. SoC per one additional year of prevented progression and/or death against the willingness-to-pay (WTP) threshold of $500,000 per QALY. A one-way probabilistic sensitivity analysis was conducted with a +/- 10% variation in all model inputs.
RESULTS: Eteplirsen demonstrated enhanced effectiveness across all stages of the disease when compared to SoC, resulting in a greater cumulative number of QALYs. However, the ICER for eteplirsen was $1,476,578 per QALY, which is significantly higher than the WTP threshold. The sensitivity analysis determined QALYs in the early ambulatory phase of eteplirsen treatment to be the most influential variable.
CONCLUSIONS: Eteplirsen demonstrated clinical advantages in delaying the progression of DMD. However, its high cost restricts its cost-effectiveness relative to SoC. Further research is needed to generate current real-world utilities and costs associated with the use of eteplirsen among patients with DMD in the US, which can be leveraged to update this model.
METHODS: A predictive Markov state-transition model was developed to simulate the progression of two DMD cohorts in the US. Costs and quality-adjusted life years (QALYs) were derived from published literature for male DMD patients diagnosed at 5 years old utilizing a societal perspective, including direct and indirect costs (adjusted for inflation in 2023). A time horizon of 25 years with a 1-year cycle shift and transition probabilities were also derived from published literature. A cost-effectiveness analysis was conducted to estimate the incremental cost-effectiveness ratios (ICERs) of eteplirsen vs. SoC per one additional year of prevented progression and/or death against the willingness-to-pay (WTP) threshold of $500,000 per QALY. A one-way probabilistic sensitivity analysis was conducted with a +/- 10% variation in all model inputs.
RESULTS: Eteplirsen demonstrated enhanced effectiveness across all stages of the disease when compared to SoC, resulting in a greater cumulative number of QALYs. However, the ICER for eteplirsen was $1,476,578 per QALY, which is significantly higher than the WTP threshold. The sensitivity analysis determined QALYs in the early ambulatory phase of eteplirsen treatment to be the most influential variable.
CONCLUSIONS: Eteplirsen demonstrated clinical advantages in delaying the progression of DMD. However, its high cost restricts its cost-effectiveness relative to SoC. Further research is needed to generate current real-world utilities and costs associated with the use of eteplirsen among patients with DMD in the US, which can be leveraged to update this model.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
SA57
Topic
Study Approaches
Topic Subcategory
Decision Modeling & Simulation
Disease
SDC: Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), SDC: Neurological Disorders, SDC: Rare & Orphan Diseases