Utilization, Expenditure and Price of Muscular Dystrophy-Duchenne Medications in the US Medicaid Programs: Trends From 2017 to 2022

OBJECTIVES: Duchenne Muscular Dystrophy (DMD) is a rare neuromuscular disorder which occurs due to the nonsense mutation in the dystrophin gene. With the increase in the treatment costs for DMD, the importance of evaluating the therapeutic expenditure and utilization in real-world data is obvious. The study aim was to highlight the trends of drug expenditure, utilization, and the cost of DMD medications for the US Medicaid program.

METHODS: A descriptive retrospective study design was conducted by using the Medicaid State Drug Utilization pharmacy summary data collected by the Center for Medicare and Medicaid Services. The study period was from 2017 to 2022. Five study medications were Exondys, Emflaza, Vyondys, Viltepso, and Amondys. All study drug prescription data were extracted using NDC codes, then were calculated for their quarterly trends of reimbursement and utilization (prescription counts). The proxy for quarterly drug prices was also calculated by the average of reimbursement per prescription count.

RESULTS: The utilization, expenditure, and price of DMD medications have rapidly increased over the last five years. From 2017 to 2022, expenditure rose from less than $10 million to nearly $35 million. Among five drugs, the overall Medicaid utilization increased from 12 prescriptions in the first quarter of 2017 to 4685 prescriptions in the third quarter of 2022. Around $882,945,422 as the total reimbursement for Exondys, $451,841,711 for Emflaza, $132,842,688 for Vyondys, $60,906,776 for Viltepso, and $138,864,158 for Amondys. The price ranges from $4,270 of (Emflaza) to $89,476 of (Exondys).

CONCLUSIONS: As DMD medications increasingly manage this severe disease, the spending and utilization have also increased greatly. The study findings help in providing insightful details about the cost drivers of DMD medications to be explored in health policies.