Research Centre on Public Health (CESP), University of Milano Bicocca, Villa Serena, Via Pergolesi 33, 20900, Monza, Italy.
Fondazione Charta, Milan, Italy.
Clin Drug Investig. 2020 Apr;40(4):319-326. doi: 10.1007/s40261-020-00895-6.
Multiple sclerosis (MS) is a highly symptomatic disease, with a wide range of disabilities affecting many bodily functions, even in younger persons with a short disease history. The availability of a cannabinoid oromucosal spray (Sativex) for the management of treatment-resistant MS spasticity has provided a new opportunity for many patients.
Our study aimed to assess the cost effectiveness of Sativex in Italian patients with treatment-resistant MS spasticity. The analysis was based on the real-world data of a large registry of Italian patients.
A cost-utility analysis was conducted using data collected prospectively from an electronic registry of all patients who began to use Sativex for MS-resistant spasticity between January 2014 and February 2015 in 30 specialized MS units across Italy and were followed up for ≤ 6 months. Data on drug consumption and spasticity/utility were used to estimate the incremental cost-effectiveness ratio (ICER) of Sativex, as compared with no intervention. No costs or spasticity/utility changes were assumed for no treatment intervention. The ICER was expressed as quality-adjusted life-years (QALYs) gained, using the Italian NHS perspective and a 6-month time horizon.
Sativex effectiveness and consumption was estimated analyzing data of 1350 patients from the registry. These patients reported a mean (SD) utility increment of 0.087 (0.069) after 1 month of treatment, 0.118 (0.073) after 3 months' treatment and 0.127 (0.080) after 6 months' treatment. The 6-month cost of treating the entire population with Sativex was €1,361,266, with a €1008 cost and 0.0284 QALYs gained per patient. The estimated ICER was €35,516 per QALY gained, with little variability around the central estimate of cost-effectiveness, as shown by the cost-effectiveness acceptability curve.
The use of Sativex could improve the quality of life of patients with a reasonable incremental cost resulting as a cost-effective option for patients with MS-resistant spasticity. These results could help clinicians and decision makers to develop improved management strategies for spasticity in patients with MS, optimizing the use of available resources.
多发性硬化症（MS）是一种高度症状性疾病，其残疾范围广泛，影响许多身体功能，即使是病史较短的年轻患者也是如此。一种大麻素口腔喷雾剂（Sativex）的出现为许多治疗抵抗性多发性硬化症痉挛患者提供了新的机会。
我们的研究旨在评估 Sativex 在意大利治疗抵抗性多发性硬化症痉挛患者中的成本效益。该分析基于意大利大型患者登记处的真实数据。
使用从 2014 年 1 月至 2015 年 2 月期间在意大利 30 个专门的多发性硬化症单位开始使用 Sativex 治疗多发性硬化症抵抗性痉挛的所有患者的电子登记处前瞻性收集的数据进行成本效用分析，并对患者进行了随访 ≤ 6 个月。使用药物消耗和痉挛/效用数据来估计 Sativex 的增量成本效益比（ICER），与无干预措施相比。对于无治疗干预，假设没有成本或痉挛/效用变化。ICER 表示为使用意大利 NHS 观点和 6 个月时间范围获得的质量调整生命年（QALY）。
从登记处分析了 1350 名患者的数据，以评估 Sativex 的有效性和消耗。这些患者报告在治疗 1 个月后平均（SD）效用增加了 0.087（0.069），在治疗 3 个月后增加了 0.118（0.073），在治疗 6 个月后增加了 0.127（0.080）。整个 Sativex 治疗人群的 6 个月成本为 1361266 欧元，每位患者的成本为 1008 欧元，QALY 增加 0.0284。估计的 ICER 为每获得 1 QALY 的 35516 欧元，成本效益的有效性接受曲线表明，成本效益的中心估计值周围存在较小的变异性。
使用 Sativex 可以提高患者的生活质量，而增量成本合理，因此是治疗抵抗性多发性硬化症痉挛患者的一种具有成本效益的选择。这些结果可以帮助临床医生和决策者制定更好的多发性硬化症痉挛患者管理策略，优化可用资源的使用。
