Center for Pharmacoepidemiology and Treatment Science, Institute for Health, Health Care Policy and Aging Research, Rutgers University, New Brunswick, New Jersey.
Department of Pediatrics, Rutgers Robert Wood Johnson Medical School, Rutgers University, New Brunswick, New Jersey.
JAMA Neurol. 2023 Aug 1;80(8):860-867. doi: 10.1001/jamaneurol.2023.2125.
Many disease-modifying therapies (DMTs) have been approved for multiple sclerosis (MS) in the past 2 decades. Research evaluating how these approvals have changed real-world prescribing patterns is scarce.
To evaluate patterns in DMT initiations between 2001 and 2020 among commercially insured US adults and children with MS.
DESIGN, SETTING, AND PARTICIPANTS: This serial cross-sectional study was conducted from 2001 through 2020 (mean patient enrollment duration, 4.8 years) and used US commercial claims data (MarketScan). Analysis took place between January 2022 and March 2023. Of 287 084 patients with MS identified, 113 583 patients (113 095 adults and 488 children) with MS newly initiated at least 1 DMT.
New initiation episode of a DMT, defined as no claim for the same DMT in the previous year.
The proportion of total DMT initiations per year attributable to each DMT. Trends in initiations were evaluated annually.
The study team identified 153 846 DMT initiation episodes among adults (median age, 46 [IQR, 38-53) years]; 86 133 female [76.2%]) and 583 among children (median age, 16 (IQR, 14-17) years; 346 female [70.9%]). Among adults, use of platform injectables showed an absolute decline of 73.8% over the study period, driven by a 61.2% reduction in interferon β initiations (P < .001 for trend). In contrast, the 2010 introduction of oral DMTs led to a rise in their use from 1.1% (2010) to 62.3% (2020) of all DMT initiations (P = .002 for trend). Infusion therapy initiations remained relatively low, accounting for 3.2% of all initiations since their introduction in 2004 but increased modestly annually after ocrelizumab was introduced (2017), reaching 8.2% of all initiations in 2020 (P < .001 for trend). Children showed similar initiation patterns, except for preferred oral therapy. Between 2019 and 2020, dimethyl fumarate was the most commonly initiated DMT in adults (23.3% to 27.2% of all initiations), while in children fingolimod was the most commonly initiated (34.8% to 68.8%).
Current MS treatment guidelines emphasize shared decision-making between patients and clinicians to balance treatment efficacy, safety, cost, and convenience. This study found that oral DMTs were the predominant DMT type initiated by 2020. The cause of this shift cannot be determined from this study, but may reflect several factors, including convenience of administration, direct-to-consumer advertising, or insurance restrictions.
在过去的 20 年里，许多疾病修正疗法 (DMT) 已被批准用于多发性硬化症 (MS)。评估这些批准如何改变现实世界的处方模式的研究很少。
评估 2001 年至 2020 年间，美国商业保险覆盖的成年和儿童 MS 患者中 DMT 的起始模式。
设计、设置和参与者：这项连续的横断面研究于 2001 年至 2020 年进行（平均患者入组时间为 4.8 年），使用了美国商业索赔数据（MarketScan）。分析于 2022 年 1 月至 2023 年 3 月之间进行。在确定的 287084 名 MS 患者中，有 113583 名 MS 患者（113095 名成年人和 488 名儿童）首次新启用了至少 1 种 DMT。
新开始使用 DMT，定义为在前一年没有同一种 DMT 的索赔。
每年归因于每种 DMT 的总 DMT 起始比例。每年评估起始趋势。
研究小组在成年患者中发现了 153846 次 DMT 起始事件（中位数年龄，46[IQR，38-53]岁）；86133 名女性[76.2%]）和 583 名儿童（中位数年龄，16[IQR，14-17]岁；346 名女性[70.9%]）。在成年患者中，平台注射剂的使用绝对减少了 73.8%，这主要是由于干扰素 β 起始治疗减少了 61.2%（趋势 P<0.001）。相比之下，2010 年口服 DMT 的推出导致其使用增加，从所有 DMT 起始治疗的 1.1%（2010 年）增加到 62.3%（2020 年）（趋势 P=0.002）。输注疗法的起始治疗仍然相对较低，自 2004 年推出以来占所有起始治疗的 3.2%，但在奥瑞珠单抗推出后每年略有增加（2017 年），在 2020 年达到所有起始治疗的 8.2%（趋势 P<0.001）。儿童的起始模式相似，除了首选口服治疗。在 2019 年至 2020 年期间，富马酸二甲酯是成人中最常用的 DMT（所有起始治疗的 23.3%至 27.2%），而在儿童中，芬戈莫德是最常用的起始治疗药物（34.8%至 68.8%）。
当前的 MS 治疗指南强调患者和临床医生之间的共同决策，以平衡治疗效果、安全性、成本和便利性。本研究发现，口服 DMT 是 2020 年最主要的 DMT 类型。这种转变的原因无法从本研究中确定，但可能反映出多种因素，包括给药便利性、直接面向消费者的广告或保险限制。
