Stollenwerk Björn, Iannazzo Sergio, Cooper Kerry, Belozeroff Vasily
a Amgen Europe (GmbH) , Zug , Switzerland.
b SIHS Health Economics Consulting , Torino , Italy.
J Med Econ. 2017 Oct;20(10):1110-1115. doi: 10.1080/13696998.2017.1360309. Epub 2017 Aug 14.
This study explored the use of a value-based pricing approach for the new calcimimetic etelcalcetide indicated for the treatment of secondary hyperparathyroidism (SHPT) in patients receiving hemodialysis. It used the US payer perspective and applied the cost-effectiveness framework. Because etelcalcetide is an intravenous therapy that can be titrated for individual patients, and because its utilization is yet to be assessed in real world settings, a range of plausible doses were estimated for etelcalcetide to define a range of prices. These were either in relation to the existing oral calcimimetic cinacalcet or compared to no calcimimetic treatment.
The value-based price of etelcalcetide was determined via a Markov model. This model combined data from the etelcalcetide trials and previously published cost-effectiveness models in SHPT, and allowed extrapolation of treatment effects on mortality, cardiovascular events, fracture, and parathyroidectomy. Several dosing scenarios were explored covering the dose ranges of 30.0-64.18 mg per day for cinacalcet and 1.07-3.11 mg per day for etelcalcetide. These included the mean dose from the etelcalcetide trials, the preliminary defined daily dose, and the expected most common dose in real world. An acceptable price range for etelcalcetide was assessed by comparing the incremental cost-effectiveness ratios obtained with the willingness-to-pay threshold range of $100,000-$300,000/quality-adjusted life-years.
Cost-effectiveness analysis supported value-based prices for etelcalcetide ranging from $21.15-$49.97/mg vs cinacalcet, and $13.79-$119.45/mg vs no calcimimetics.
There is uncertainty around what the real-world dosing will be for etelcalcetide. Another important nuance is that no studies have examined etelcalcetide effects on hard outcomes and, therefore, this modeling exercise relied on an extrapolation approach.
This cost-effectiveness analysis, including scenarios to address uncertainties, allowed estimation of a value-based price range to aid reimbursement decisions in the US.
本研究探讨了基于价值定价方法在用于治疗接受血液透析患者继发性甲状旁腺功能亢进（SHPT）的新型拟钙剂依特卡肽上的应用。研究采用美国医保支付方视角并应用成本效益框架。由于依特卡肽是一种可针对个体患者进行滴定的静脉治疗药物，且其在现实环境中的使用情况尚未得到评估，因此估计了依特卡肽一系列合理的剂量以确定一系列价格。这些价格要么与现有的口服拟钙剂西那卡塞相关，要么与不使用拟钙剂治疗的情况进行比较。
依特卡肽基于价值的价格通过马尔可夫模型确定。该模型结合了依特卡肽试验数据以及先前发表的SHPT成本效益模型数据，并允许推断治疗对死亡率、心血管事件、骨折和甲状旁腺切除术的影响。探索了几种给药方案，涵盖西那卡塞每天30.0 - 64.18毫克以及依特卡肽每天1.07 - 3.11毫克的剂量范围。这些方案包括依特卡肽试验中的平均剂量、初步确定的每日剂量以及现实环境中预期的最常用剂量。通过将获得的增量成本效益比与100,000 - 300,000美元/质量调整生命年的支付意愿阈值范围进行比较，评估依特卡肽的可接受价格范围。
成本效益分析支持依特卡肽基于价值的价格范围，与西那卡塞相比为每毫克21.15 - 49.97美元，与不使用拟钙剂相比为每毫克13.79 - 119.45美元。
依特卡肽在现实中的给药剂量存在不确定性。另一个重要细微差别是，尚无研究考察依特卡肽对硬性结局的影响，因此，本建模工作依赖于外推法。
这项成本效益分析，包括解决不确定性的方案，使得能够估计基于价值的价格范围，以协助美国的报销决策。
