Study Highlights Significant Increases in Utilization, Spending on DMD Drugs in Medicaid


The findings add to recent research on the growing utilization, expenditure, and prices of Duchenne muscular dystrophy (DMD) therapies in the current landscape, an area health care policy could potentially address.

A poster presented at the ISPOR—The Professional Society for Health Economics and Outcomes Research meeting held in Atlanta, Georgia, delved into trends in the utilization, expenditure, and price of medications for Duchenne muscular dystrophy (DMD) in the US Medicaid program. The findings highlight increased spending and utilization of DMD medications in recent years, providing useful information for policy makers to consider, according to the authors.1

“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 authors wrote. “The study aim was to highlight the trends of drug expenditure, utilization, and the cost of DMD medications for the US Medicaid program.”

The investigators utilized data from the Medicaid State Drug Utilization pharmacy summary, which is collected by CMS, to conduct the descriptive retrospective study. Drug prescription data were extracted by National Drug Code identifiers, then the researchers assessed quarterly reimbursement and utilization trends based on prescription counts.

Research highlights rising costs and utilization of medications for DMD | Image credit: janews094 -

Research highlights rising costs and utilization of medications for DMD | Image credit: janews094 -

Data from 2017 to 2022 were included in the analysis, which assessed spending and utilization of 5 medications: eteplirsen (Exondys), deflazacort (Emflaza), golodirsen (Vyondys), viltolarsen (Viltepso), and casimersen (Amondys). Eteplirsen, golodirsen, vitolarsen, and casimersen are antisense oligonucleotides; deflazacort is a glucocorticoid, a derivative of prednisone.2

The analysis showed rapidly increasing utilization, expenditure, and prices of DMD treatments in the 5-year study period, with spending jumping from less than $10 million to nearly $35 million from 2017 to 2022.1 Medicaid utilization of the 5 drugs in the study increased from 12 prescriptions in the first quarter of 2017 to 4685 in quarter 3 of 2022.

Overall reimbursements for each drug were substantial. Total reimbursement was about $882,945,422 for eteplirsen, $451,841,711 for deflazacort, $132,842,688 for golodirsen, $60,906,776 for viltolarsen, and $138,864,158 for casimersen. The authors also noted the wide range of pricing, with deflazacort on the low end at $4270 and eteplirsen on the high end at $89,476.

Drug pricing and expenditure have been much-discussed topics across the health care space overall in recent years, and experts have noted DMD specifically as an area where there is a need for reform.3 Specifically, genetically targeted treatments for DMD have been a driver of increased public and private spending despite a lack of completed confirmatory trials and little evidence of efficacy in several cases.

In a recently published analysis, researchers estimated that Medicaid and Medicare spending on eteplirsen, golodirsen, and casimersen alone increased from $25 million in 2017 to $327 million in 2022, with spending totaling $1.2 billion.4 In the study, Medicaid spending dwarfed Medicare spending on these drugs ($1.1 billion vs $104 million).

The findings presented at ISPOR reiterate the growing utilization, expenditure, and prices of DMD therapies in the current landscape, further highlighting an area health care policy makers should consider.1

“As DMD medications increasingly manage this severe disease, the spending and utilization have also increased greatly,” the authors wrote.1 “The study findings help in providing insightful details about the cost drivers of DMD medications to be explored in health policies.”


1. Ghawaa Y, Winkle JL, Ferguson A, et al. Utilization, expenditure and price of muscular dystrophy-Duchenne medications in the US Medicaid programs: trends from 2017 to 2022. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 2046.

2. What are the new drugs for Duchenne muscular dystrophy? Updated April 1, 2024. Accessed May 16, 2024.

3. Shaw M. Experts issue call for reform to DMD drug pricing, trial approval process. AJMC. March 14, 2024. Accessed May 16, 2024.

4. Bendicksen L, Kesselheim AS, Rome BN. Spending on targeted therapies for Duchenne muscular dystrophy. JAMA. 2024;331(13):1151-1153. doi:10.1001/jama.2024.2776

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