a IBM Watson Health , Cambridge , MA , USA.
b Novartis Pharmaceutical Corporation , East Hanover , NJ , USA.
Curr Med Res Opin. 2019 Jun;35(6):1103-1110. doi: 10.1080/03007995.2018.1558883. Epub 2019 Jan 14.
Everolimus is the only FDA approved drug to treat renal angiomyolipoma or subependymal giant-cell astrocytoma (SEGA) in tuberous sclerosis complex (TSC). Potential differences exist between patients with commercial and Medicaid insurance on everolimus use; however, there is limited information from the real world. This study compared compliance and persistence of everolimus between commercial and Medicaid patients using US claims data.
Patients with ≥1 claim of TSC with renal angiomyolipoma or SEGA were selected from the MarketScan commercial (1 January 2009-31 August 2016) and Medicaid (1 January 2009-30 June 2015) databases. Patients were followed from index date (the earliest date of TSC, renal angiomyolipoma or SEGA diagnosis) to death or end of data. Non-persistence, defined as ≥60 day gap without everolimus, and medication possession ratio (MPR) were assessed among the subset of patients with ≥1 year of follow-up from the first everolimus claim.
A total of 1497 TSC patients met the study criteria (896 renal angiomyolipoma only, 411 SEGA only and 190 both). Compared to Medicaid patients (N = 513), commercial patients (N = 984) had the same ages (22 years) but a shorter length of follow-up (38 vs. 48 months, p < .001). Medicaid and commercial patients had similar rates of being treated with everolimus (14.4% vs. 13.6%, p = .668), but it took Medicaid patients a longer time to start everolimus (871 vs. 704 days, p < .001). Although the non-persistence rate was not significantly different between commercial and Medicaid patients (42.5% vs. 35.1%, p = .561), the number of days from everolimus initiation to non-persistence was significantly lower for commercial patients (945 vs. 1132, p < .001). During the 1 year post everolimus initiation, commercial patients had a significantly higher MPR (0.81 vs. 0.74, p < .001) and higher percentage of patients with MPR ≥0.80 (67.8% vs. 58.1%, p < .001).
Among TSC patients with renal angiomyolipoma or SEGA and treated with everolimus, everolimus MPR was between 0.74 and 0.81. Medicaid patients had lower MPR than commercial patients but better persistence.
依维莫司是唯一获美国食品药品监督管理局批准用于治疗结节性硬化症相关肾血管平滑肌脂肪瘤或室管膜下巨细胞星形细胞瘤的药物。商业保险和医疗补助保险在依维莫司的使用上可能存在差异；然而，来自真实世界的数据有限。本研究使用美国索赔数据比较了商业保险和医疗补助保险患者使用依维莫司的依从性和持久性。
从 MarketScan 商业保险（2009 年 1 月 1 日-2016 年 8 月 31 日）和医疗补助保险（2009 年 1 月 1 日-2015 年 6 月 30 日）数据库中选择至少有 1 次结节性硬化症伴肾血管平滑肌脂肪瘤或室管膜下巨细胞星形细胞瘤诊断的患者。从索引日期（最早的结节性硬化症、肾血管平滑肌脂肪瘤或室管膜下巨细胞星形细胞瘤诊断日期）开始随访患者，直至死亡或数据结束。在至少有 1 年依维莫司使用随访记录的患者亚组中，评估非持续性（无依维莫司使用的≥60 天间隔）和药物利用率（MPR）。
共纳入 1497 例符合研究标准的 TSC 患者（肾血管平滑肌脂肪瘤患者 896 例，室管膜下巨细胞星形细胞瘤患者 411 例，两者均有的患者 190 例）。与医疗补助保险患者（n=513）相比，商业保险患者（n=984）的年龄相同（22 岁），但随访时间较短（38 个月 vs. 48 个月，p<0.001）。医疗补助保险和商业保险患者接受依维莫司治疗的比例相似（14.4% vs. 13.6%，p=0.668），但医疗补助保险患者开始接受依维莫司治疗的时间较长（871 天 vs. 704 天，p<0.001）。尽管商业保险和医疗补助保险患者的非持续性率无显著差异（42.5% vs. 35.1%，p=0.561），但商业保险患者从开始依维莫司治疗到非持续性的天数明显较少（945 天 vs. 1132 天，p<0.001）。在依维莫司起始后 1 年内，商业保险患者的 MPR 显著更高（0.81 与 0.74，p<0.001），MPR≥0.80 的患者比例更高（67.8% vs. 58.1%，p<0.001）。
在接受依维莫司治疗的结节性硬化症相关肾血管平滑肌脂肪瘤或室管膜下巨细胞星形细胞瘤患者中，依维莫司 MPR 在 0.74 到 0.81 之间。与商业保险患者相比，医疗补助保险患者的 MPR 较低，但持久性较好。
