Medicaid spending and utilization of gene and RNA therapies for rare inherited conditions.

Health affairs scholar Pub Date : 2024-04-26 eCollection Date: 2024-05-01 DOI:10.1093/haschl/qxae051
Ilina C Odouard, Jeromie Ballreich, Mariana P Socal
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Abstract

Gene and RNA therapies are promising treatments for many rare diseases. Pediatric populations that could benefit from these drugs are overrepresented among state Medicaid programs. Using Medicaid State Drug Utilization Data, we examined Medicaid spending and utilization of rare disease gene and RNA therapies. Between 2017 and 2022, the number of available gene and RNA therapies increased from 3 to 13, yearly Medicaid spending increased from $148.3 million to $879.7 million, and the number of yearly treatments (a proxy for number of patients) increased from 327 to 1638. Nearly all spending was attributed to spinal muscular atrophy (SMA) and Duchenne muscular dystrophy drugs. States participating in Medicaid pooled purchasing initiatives had 39% higher treatments per 100 000 enrollees with no differences in spending. Compared to states without a carve-out, states that carved SMA drugs out of managed Medicaid contracts had higher utilization (54%). Spending among carve-out states varied according to managed care enrollment, being higher for those with <80% of enrollees in managed care as compared with those with ≥80% of enrollees in managed care. This suggests that multi-state purchasing initiatives and managed care carve-outs can help increase access to gene and RNA therapies among Medicaid beneficiaries, but it is unclear if these strategies are effective at managing spending.

医疗补助支出以及罕见遗传性疾病基因和 RNA 疗法的使用情况。
基因和 RNA 疗法是许多罕见病的有望治疗方法。可以从这些药物中获益的儿科人群在各州医疗补助计划中的比例过高。我们利用《医疗补助州药物使用数据》(Medicaid State Drug Utilization Data)研究了罕见病基因和 RNA 疗法的医疗补助支出和使用情况。从 2017 年到 2022 年,可用的基因和 RNA 疗法从 3 种增加到 13 种,医疗补助年度支出从 1.483 亿美元增加到 8.797 亿美元,年度治疗次数(代表患者人数)从 327 次增加到 1638 次。几乎所有支出都用于脊髓性肌萎缩症(SMA)和杜氏肌营养不良症药物。参与医疗补助联合采购计划的州,每 10 万名参保者的治疗次数增加了 39%,但支出没有差异。与没有划出计划的州相比,将 SMA 药物划出医疗补助管理合同的州的使用率更高(54%)。各州的支出因管理性医疗保险参保人数而异,参保人数越多,支出越高。
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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