1 Division of Pharmaceutical, Administrative and Social Sciences, Duquesne University School of Pharmacy, Pittsburgh, Pennsylvania.
2 Pharmacy Practice, Philadelphia College of Osteopathic Medicine, Suwanee, Georgia.
J Manag Care Spec Pharm. 2018 Oct;24(10):987-997. doi: 10.18553/jmcp.2018.24.10.987.
Cystic fibrosis (CF) is a chronic, progressive, genetic disease affecting more than 30,000 people in the United States and 70,000 people globally. The goals of treatment are to slow disease progression, reduce pulmonary exacerbations, relieve chronic symptoms, and improve the patient's quality of life. Lumacaftor/ivacaftor is a new therapy for CF that has demonstrated good clinical outcomes, including improved absolute percentage predicted forced expiratory volume in 1 second (FEV%). However, given the high cost of therapy, there is a need to evaluate the overall value of lumacaftor/ivacaftor in CF management.
To (a) conduct a cost-effectiveness analysis (CEA) of lumacaftor/ivacaftor to understand the overall effectiveness of the drug compared with its costs and (b) conduct a budget impact analysis (BIA) to understand the potential financial effect of introducing a new drug in a health plan.
Two static decision models were developed using Microsoft Excel to evaluate the cost-effectiveness and budget impact of lumacaftor/ivacaftor over a 1-year time frame from a payer perspective. Model inputs included drug costs (wholesale acquisition costs), drug monitoring schedules (package inserts), drug monitoring costs (Centers for Medicare & Medicaid physician fee schedule and published literature), FEV% predicted and pulmonary exacerbation values (clinical trials), and cost to treat pulmonary exacerbations (published literature). The outcomes in the CEA included total cost of therapy; average cost-effectiveness ratio (ACER), defined as cost per FEV% predicted; and incremental cost-effectiveness ratio (ICER), defined as the difference in the ratio of cost per FEV% predicted of lumacaftor/ivacaftor and placebo. Outcomes in the BIA included total budget impact; cost per member per month (PMPM), defined as total budget impact per hypothetical plan population; and cost per treated member per month (PTMPM), defined as total budget impact per target CF population. All costs were adjusted to 2016 dollars, and one-way sensitivity analyses were conducted to test the model robustness given uncertainty in model inputs and study assumptions.
The annual cost of therapy per patient for lumacaftor/ivacaftor was $379,780. The ACER for lumacaftor/ivacaftor was $151,912, while the ICER for lumacaftor/ivacaftor compared with placebo was $95,016 per FEV% predicted. The annual total budget impact due to the inclusion of lumacaftor/ivacaftor on the health plan formulary was $266,046. The PMPM cost was $0.02 and the PTMPM cost was $6.21.
In patients with CF, lumacaftor/ivacaftor has demonstrated better clinical effectiveness compared with placebo alongside an increased drug acquisition cost. However, the therapy may be a viable alternative to existing standard therapy over a short time horizon. Health care payers, both private and public, need to evaluate the cost-effectiveness and the financial effect when considering expansion of new drug coverage in CF management.
No outside funding supported this study. Covvey and Kamal have received research funding from Novartis Pharmaceuticals. Covvey, Giannetti, and Kamal have received research funding from the College of Psychiatric and Neurologic Pharmacists. Kamal serves as a consultant to the Lynx Group (Cranbury, NJ) and Manticore Consulting Group (Scottsdale, AZ). Mukherjee has nothing to disclose. A related poster abstract was presented at the AMCP Managed Care & Specialty Pharmacy Annual Meeting; March 27-30, 2017; Denver, CO.
囊性纤维化（CF）是一种影响美国超过 30000 人和全球 70000 人的慢性、进行性、遗传性疾病。治疗的目标是减缓疾病进展、减少肺部恶化、缓解慢性症状和提高患者的生活质量。Lumacaftor/ivacaftor 是一种新的 CF 治疗方法，已证明具有良好的临床疗效，包括改善绝对预测 1 秒用力呼气量（FEV%）。然而，鉴于治疗费用高昂，需要评估 Lumacaftor/ivacaftor 在 CF 管理中的总体价值。
（a）进行 Lumacaftor/ivacaftor 的成本效益分析（CEA），了解药物的总体疗效与其成本的关系；（b）进行预算影响分析（BIA），了解新药物在医疗保险计划中引入的潜在财务影响。
使用 Microsoft Excel 开发了两个静态决策模型，从支付者的角度评估 Lumacaftor/ivacaftor 在 1 年时间内的成本效益和预算影响。模型输入包括药物成本（批发收购成本）、药物监测计划（包装说明书）、药物监测成本（医疗保险和医疗补助服务中心医生费用表和已发表文献）、FEV%预测值和肺部恶化值（临床试验）以及治疗肺部恶化的成本（已发表文献）。CEA 的结果包括治疗总费用；平均成本效益比（ACER），定义为每 FEV%预测值的成本；增量成本效益比（ICER），定义为 Lumacaftor/ivacaftor 和安慰剂的每 FEV%预测值成本比的差异。BIA 的结果包括总预算影响；每成员每月成本（PMPM），定义为每个假设计划人群的总预算影响；每治疗成员每月成本（PTMPM），定义为每个目标 CF 人群的总预算影响。所有成本均按 2016 年美元进行调整，并进行了单因素敏感性分析，以测试模型在模型输入和研究假设不确定性下的稳健性。
Lumacaftor/ivacaftor 每位患者的年治疗费用为 379780 美元。Lumacaftor/ivacaftor 的 ACER 为 151912 美元，而 Lumacaftor/ivacaftor 与安慰剂相比的 ICER 为每 FEV%预测值 95016 美元。由于 Lumacaftor/ivacaftor 被纳入医疗保险计划的 formulary，健康计划的总预算影响为 266046 美元。PMPM 成本为 0.02，PTMPM 成本为 6.21。
在 CF 患者中，Lumacaftor/ivacaftor 与安慰剂相比具有更好的临床疗效，同时增加了药物采购成本。然而，在短期内，这种治疗方法可能是现有标准治疗的可行替代方案。私人和公共医疗保健支付者都需要在考虑扩大 CF 管理中新药物覆盖范围时，评估其成本效益和财务影响。
本研究没有外部资金支持。Covvey 和 Kamal 从诺华制药公司获得了研究经费。Covvey、Giannetti 和 Kamal 从精神病学和神经病学药剂师学院获得了研究经费。Kamal 是 Lynx Group（Cranbury，NJ）和 Manticore Consulting Group（Scottsdale，AZ）的顾问。Mukherjee 没有什么可披露的。一篇相关的海报摘要在 AMCP 管理式医疗和特种药房年会；2017 年 3 月 27 日至 30 日；丹佛，CO。
