预算影响分析的Eliglustat戈谢病的治疗1型在美国。

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Nalysnyk L,苏格曼R,肿物C, Uyei J,病房

预算影响分析的Eliglustat戈谢病的治疗1型在美国。

J等护理规范制药。2018年10月,24 (10):1002 - 1008。doi: 10.18553 / jmcp.2018.24.10.1002。

PubMed ID

30247105 (在PubMed

]

文摘

背景:戈谢病1型(GD1)是一种罕见的遗传,溶酶体储存疾病没有治愈。目前的治疗方法包括静脉注射(IV)酶替代疗法((ERT);imiglucerase、velaglucerase阿尔法或taliglucerase阿尔法)或口服底物还原疗法((SRT);eliglustat或miglustat)。美国纳税人的成本IV-administered药物可以取决于医疗网站(例如、家庭、门诊或医院设置)。治疗口腔eliglustat可能存在一个节省成本的机会。目的:评估相关的预算从美国人的角度影响过渡病人接受ERTs口服SRT eliglustat GD1治疗的成年人。方法:预算影响模型估计制药和管理成本的变化导致增加eliglustat的市场份额从12%(当前)到44%(新)。eliglustat的市场份额是同样来自现有的股票imiglucerase(40%)和velaglucerase阿尔法(40%)和被认为是静态的分析。ERT成本调整占网站care-based标记和接受输液的病人的比例在每个站点的护理(家里、输液中心或医院门诊)。 Annual ERT costs were calculated assuming a biweekly dose of 47.4 U per kg, a 72-kg patient weight, and 24 infusions per year. The effect of key variables was tested in the sensitivity analyses. All costs are expressed in 2017 U.S. dollars. RESULTS: In a new plan with 5 million members and 25 GD1 treated patients, increased use of eliglustat resulted in an annual savings of $1,526,710 and a total savings of $4,580,130 (13.6%) over 3 years. The corresponding annual per member per month savings was $0.025. This is further illustrated in the sensitivity and scenario analyses where the use of eliglustat was cost saving in all cases. Shifting more patients receiving ERT in the hospital outpatient setting to eliglustat resulted in increased savings. CONCLUSIONS: Based on these analyses, increased use of eliglustat resulted in meaningful cost savings to a payer's overall budget. Cost savings are highest among patients switching from ERT administered in a hospital outpatient setting. The results suggest that cost savings are also likely achievable from initiating patients on oral eliglustat instead of infusion-based therapy from the outset of treatment. DISCLOSURES: This study was sponsored by Sanofi Genzyme. Evidera received funding from Sanofi Genzyme to conduct this study and prepare the manuscript. The sponsor collaborated on the study design, analysis, interpretation of results, and writing of the manuscript. Nalysnyk is an employee of and shareholder in Sanofi Genzyme. Ward, Cele, and Uyei are employees of Evidera, which provides consulting and other research services to biopharmaceutical companies. Sugarman was also an Evidera employee when the study was being conducted and the manuscript written. This study was presented as a poster at the Academy of Managed Care Pharmacy Nexus 2016, October 3-6, 2016; National City, MD, and at the International Society for Pharmacoeconomics and Outcomes Research, 22nd Annual International Meeting; May 20-24, 2017; Boston, MA.

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药物

Eliglustat