Hope MS Center, Knoxville, TN, USA.
J Med Econ. 2013;16(4):547-51. doi: 10.3111/13696998.2013.774281. Epub 2013 Feb 21.
Adherence to medication is essential for optimal outcomes, especially for chronic diseases such as multiple sclerosis (MS). Studies in MS indicate that lower adherence is associated with an increased risk of relapse, hospitalization or emergency room (ER) visits, and higher medical costs. A previous investigation assessed the cost per relapse avoided for patients with MS receiving first-line disease modifying therapies (DMTs); however, the model assumed 100% adherence.
Because real-world utilization patterns influence the actual effectiveness of medications, this analysis assessed the impact of real-world adherence from a US commercial payer perspective, using updated costs.
As was seen in the original study, in this revised model, fingolimod was associated with the lowest cost per relapse avoided ($90,566), followed by SC IFN β-1b (Extavia: $127,024), SC IFN β-1b (Betaseron: $137,492), SC IFN β-1a ($144,016), glatiramer acetate ($160,314), and IM IFN β-1a ($312,629). The model inputs that had the greatest impact on the results were adherence-adjusted relative relapse rate reduction (RRR) of fingolimod, the wholesale acquisition costs of fingolimod, and the average number of relapses in untreated patients with MS.
The estimates of DMT adherence are from a single claims database study of a large national pharmacy benefit manager that only measured adherence, not actual relapses, and the model does not incorporate manufacturer discounts and rebates, which are not publicly available.
These results suggest that economic analyses of MS therapies should incorporate real-world adherence rates where available, rather than relying exclusively on trial-based efficacy estimates when considering the economic value of treatment alternatives, and that highly efficacious therapies with low adherence may yield real-world efficacy that is substantially lower than that observed in closely monitored clinical trials.
药物依从性对于获得最佳疗效至关重要，尤其是对于多发性硬化症（MS）等慢性病。MS 相关研究表明，药物依从性较低与疾病复发、住院或急诊就诊风险增加以及医疗费用增加相关。既往研究评估了接受一线疾病修正治疗（DMT）的 MS 患者避免每次复发的成本效益；然而，该模型假设了 100%的依从性。
由于实际用药情况会影响药物的实际疗效，因此本分析从美国商业支付方的角度，使用更新后的成本，评估了实际用药依从性的影响。
与原始研究一致，在该修订模型中，芬戈莫德（fingolimod）避免每次复发的成本最低（90566 美元），其次是皮下注射 IFNβ-1b（特立氟胺：Extavia）（127024 美元）、皮下注射 IFNβ-1b（干扰素β-1b：Betaseron）（137492 美元）、皮下注射 IFNβ-1a（144016 美元）、那他珠单抗（glatiramer acetate）（160314 美元）和肌肉注射 IFNβ-1a（312629 美元）。对结果影响最大的模型输入包括：芬戈莫德的调整后相对复发率降低（RRR）、芬戈莫德的批发采购成本，以及未经治疗的 MS 患者的平均复发次数。
DMT 依从性的估计值来自于大型全国药房福利管理机构的一项单一索赔数据库研究，该研究仅测量了依从性，而未测量实际复发情况，并且该模型未纳入制造商折扣和回扣，这些信息无法公开获得。
这些结果表明，在考虑治疗替代方案的经济价值时，MS 治疗的经济分析应纳入实际的依从性数据，而不是仅仅依赖基于试验的疗效估计。对于依从性低但疗效较高的治疗方法，其实际疗效可能远低于密切监测临床试验中的疗效。
