Department of Pharmacy Systems, Outcomes and Policy, College of Pharmacy, University of Illinois at Chicago, Chicago, Illinois.
Institute for Clinical and Economic Review, Boston, Massachusetts.
Pharmacotherapy. 2018 May;38(5):520-530. doi: 10.1002/phar.2108. Epub 2018 Apr 25.
Sacubitril/valsartan (SAC/VAL) has been shown to reduce mortality and hospitalization in patients with heart failure with reduced ejection fraction (HFrEF) compared with enalapril but at a substantially higher cost. This study evaluates the cost-effectiveness of SAC/VAL versus enalapril in patients with HFrEF over a 5-year time horizon from the U.S. payer perspective.
A cohort-based Markov model was developed to compare costs and quality-adjusted life years (QALYs) between SAC/VAL and enalapril in patients with HFrEF over a 5-year time horizon. Markov states included New York Heart Association (NYHA) class (II-IV) and death. Treatment discontinuation, HF-related hospitalizations, and NYHA class progression were modeled as transition states based on data from the PARADIGM trial. Other probabilities, costs, and utilities were obtained from published literature and public databases.
In the base case analysis, SAC/VAL cost more than enalapril ($81,943 vs $67,287) and was more effective (2.647 QALYs vs 2.546 QALYs), resulting in an incremental cost-effectiveness ratio of $143,891/QALY gained. At a willingness to pay (WTP) of $100,000/QALY, SAC/VAL was cost-effective up to a cost of $298/month. Results were most sensitive to SAC/VAL cost, SAC/VAL mortality benefit, and NYHA progression probability. SAC/VAL had a 10% and 52% probability of being cost-effective at WTP thresholds of $100,000/QALY and $150,000/QALY, respectively.
SAC/VAL is associated with clinical benefit and may be cost-effective compared with the current standard of care over realistic treatment durations from the payer perspective. Results of this analysis can inform discussions on the value and position of SAC/VAL in the current market.
与依那普利相比，沙库巴曲缬沙坦（SAC/VAL）已被证明可降低射血分数降低的心力衰竭（HFrEF）患者的死亡率和住院率，但成本要高得多。本研究从美国支付者的角度评估了 SAC/VAL 与依那普利相比在 HFrEF 患者中的成本效益，时间范围为 5 年。
采用基于队列的马尔可夫模型，比较了 SAC/VAL 和依那普利在 HFrEF 患者 5 年时间范围内的成本和质量调整生命年（QALYs）。马尔可夫状态包括纽约心脏协会（NYHA）心功能分级（II-IV）和死亡。根据 PARADIGM 试验的数据，治疗停药、HF 相关住院和 NYHA 心功能分级进展被建模为转移状态。其他概率、成本和效用来自已发表的文献和公共数据库。
在基础案例分析中，SAC/VAL 的成本高于依那普利（81943 美元 vs. 67287 美元），且更有效（2.647 QALYs vs. 2.546 QALYs），增量成本效益比为 143891 美元/QALY。在支付意愿（WTP）为 10 万美元/QALY 的情况下，SAC/VAL 的成本效益最高可达每月 298 美元。结果对 SAC/VAL 成本、SAC/VAL 死亡率获益和 NYHA 进展概率最为敏感。SAC/VAL 在支付意愿阈值分别为 10 万美元/QALY 和 15 万美元/QALY 时，有 10%和 52%的概率具有成本效益。
从支付者的角度来看，SAC/VAL 与当前标准治疗相比，具有临床获益，并且在现实治疗持续时间内可能具有成本效益。本分析结果可为 SAC/VAL 在当前市场中的价值和定位提供信息。
