The Utilization, Reimbursement, and Cost of Targeted Therapies for Duchenne Muscular Dystrophy (DMD) in US Medicaid Programs: A Descriptive Trend Analysis from 2017 to 2022.

IF 4.5 Q2 PHARMACOLOGY & PHARMACY

Pharmaceutical Medicine Pub Date : 2025-09-20 DOI:10.1007/s40290-025-00587-6

Yazeed Ghawaa, Alex C Lin, Jeff Jianfei Guo

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引用次数: 0

Abstract

Background: Duchenne muscular dystrophy (DMD) is a rare hereditary neuromuscular disorder caused by mutations in the dystrophin gene, leading to progressive muscle deterioration, loss of ambulation, and reduced life expectancy. The US Food and Drug Administration (FDA) has approved several novel drugs for DMD; however, their utilization, reimbursement, and cost patterns within US Medicaid programs remain unexamined.

Objectives: The purpose of this study was to evaluate the utilization, reimbursement, and cost trends of targeted therapies for DMD in the US Medicaid population from 2017 to 2022.

Methods: A retrospective descriptive drug utilization study was conducted using the national Medicaid drug utilization files that were extracted from the Centers for Medicare and Medicaid Services (CMS) from 2017 to 2022. The study drugs comprised the following four FDA-approved drugs: eteplirsen (Exondys 51^®), golodirsen (Vyondys 53^®), viltolarsen (Viltepso^®), and casimersen (Amondys 45^®). The annual number of prescriptions represented drug utilization and the annual amount of reimbursement represented the total spending, which were calculated for time-series secular trend analyses. The average cost per prescription was also computed as an estimate of drug cost trends.

Results: Over the 6-year period, the total count of DMD prescriptions increased significantly by 2988%, from 643 prescriptions in 2017, when only eteplirsen (Exondys 51^®) was available, to a peak of 19,855 prescriptions in 2022, including all four novel DMD drugs. Additionally, the overall reimbursement grew by nearly 2809%, increasing from US$22,027,999 in 2017 to US$640,890,515 in 2022. However, the average cost per prescription decreased by about 6%, from US$34,258 in 2017 to US$32,279 in 2022.

Conclusion: The considerable rise in the utilization and spending of novel DMD drugs has imposed a significant burden on the Medicaid budget, underlining the need for policy measures to manage rising costs and maintain equal access to treatment.

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美国医疗补助计划中杜氏肌营养不良症（DMD）靶向治疗的使用、报销和成本：2017年至2022年的描述性趋势分析

背景：杜氏肌营养不良症（DMD）是一种罕见的遗传性神经肌肉疾病，由肌营养不良蛋白基因突变引起，可导致进行性肌肉退化、行动能力丧失和预期寿命缩短。美国食品和药物管理局（FDA）已经批准了几种治疗DMD的新药；然而，它们在美国医疗补助计划中的使用、报销和成本模式仍未得到检验。目的：本研究的目的是评估2017年至2022年美国医疗补助人群中DMD靶向治疗的使用、报销和成本趋势。方法：采用从美国联邦医疗保险和医疗补助服务中心（CMS）提取的2017 - 2022年国家医疗补助药物使用档案进行回顾性描述性药物使用研究。研究药物包括以下四种fda批准的药物：eteplirsen （Exondys 51®），golodirsen （Vyondys 53®），viltolarsen （Viltepso®）和casimersen （Amondys 45®）。年度处方数量代表药物利用，年度报销金额代表总支出，计算后进行时间序列长期趋势分析。每个处方的平均成本也被计算为药物成本趋势的估计。结果：在6年的时间里，DMD处方总数从2017年的643张（当时只有eteplirsen （Exondys 51®））显著增加2988%，到2022年达到19,855张的峰值，包括所有四种新型DMD药物。此外，总体报销额增长了近2809%，从2017年的22,027,999美元增加到2022年的640,890,515美元。然而，每张处方的平均成本下降了约6%，从2017年的34258美元降至2022年的32279美元。结论：新型DMD药物的使用和支出的大幅增加对医疗补助预算造成了重大负担，强调需要采取政策措施来管理不断上升的成本并保持平等的治疗机会。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pharmaceutical Medicine PHARMACOLOGY & PHARMACY-

CiteScore

5.10

自引率

4.00%

发文量

期刊介绍： Pharmaceutical Medicine is a specialist discipline concerned with medical aspects of the discovery, development, evaluation, registration, regulation, monitoring, marketing, distribution and pricing of medicines, drug-device and drug-diagnostic combinations. The Journal disseminates information to support the community of professionals working in these highly inter-related functions. Key areas include translational medicine, clinical trial design, pharmacovigilance, clinical toxicology, drug regulation, clinical pharmacology, biostatistics and pharmacoeconomics. The Journal includes:Overviews of contentious or emerging issues.Comprehensive narrative reviews that provide an authoritative source of information on topical issues.Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by PRISMA statement.Original research articles reporting the results of well-designed studies with a strong link to wider areas of clinical research.Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pharmaceutical Medicine may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.