eligustat在美国治疗戈谢病1型的预算影响分析
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Nalysnyk L, Sugarman R, Cele C, Uyei J, Ward A
eligustat在美国治疗戈谢病1型的预算影响分析
J Manag Care Spec Pharm. 2018年10月;24(10):1002-1008。doi: 10.18553 / jmcp.2018.24.10.1002。
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30247105 (PubMed视图］
- 摘要
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背景:戈谢病1型(GD1)是一种罕见的遗传性溶酶体储存疾病，目前尚无治愈方法。目前的治疗方案包括静脉注射(IV)酶替代疗法([ERT];或口服底物减少疗法([SRT];Eliglustat或miglustat)。美国支付静脉注射药物的费用可能因护理地点(即家庭、门诊或医院环境)而异。口服eligustat治疗可能提供节省成本的机会。目的:从美国付款人的角度评估将接受ert的成人患者过渡到口服SRT eligustat治疗GD1的预算影响。方法:预算影响模型估计了将eligustat的市场份额从12%(当前)增加到44%(新)导致的药物和管理成本的变化。eliglustat的市场份额平等地来自于imiglucerase(40%)和velaglucerase alfa(40%)的现有份额，并假设在分析期间保持不变。对ERT成本进行了调整，以考虑基于护理地点的加价和每个护理地点(家庭、输液中心或医院门诊)接受输液的患者比例。 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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