从新加坡私人保险支付方角度评估 tisagenlecleucel 治疗成人复发/难治弥漫性大 B 细胞淋巴瘤的成本效果和预算影响分析。
Cost-effectiveness and budget impact analyses of tisagenlecleucel in adult patients with relapsed or refractory diffuse large B-cell lymphoma from Singapore's private insurance payer's perspective.
机构信息
Novartis Singapore Pte Ltd., Singapore, Singapore.
Novartis Pharmaceuticals Asia-Pacific Pte Ltd., Singapore, Singapore.
出版信息
J Med Econ. 2021 Jan-Dec;24(1):637-653. doi: 10.1080/13696998.2021.1922066.
BACKGROUND
Patients experiencing relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) have limited treatment options and poor prognosis. Tisagenlecleucel (TIS) has shown improved clinical outcomes, but at a high upfront cost. Singapore has a multi-payer healthcare system where private insurance is one of the major payers. This study evaluated the cost-effectiveness and budget impact of TIS against salvage chemotherapy regimen (SCR) for treating r/r DLBCL patients who have failed ≥2 lines of systemic therapy from Singapore's private insurance payer's perspective.
METHODS
Over a life-time horizon, a partitioned survival model with three health-states was developed to evaluate the cost-effectiveness of TIS vs. SCR with or without hematopoietic stem cell transplantation (HSCT). Efficacy inputs for TIS and SCR were based on 43 months of observation data from pooled JULIET and UPenn trials, and CORAL extension studies respectively. Direct costs for pre-treatment, treatment, adverse events, follow-up, subsequent-HSCT, relapse, and terminal care were included. Incremental cost-effectiveness ratios (ICERs) were calculated as the total incremental costs per quality-adjusted life-year (QALY) gained. Additionally, the financial implication of introducing TIS in Singapore from a private payer's perspective was analyzed, comparing the current treatment pathway (without TIS) with a future scenario (with TIS) over 5 years.
RESULTS
Compared with SCR, TIS was the dominant option, with cost savings of S$8,477 alongside an additional gain of 2.78 QALYs in privately insured patients who shifted from private to public hospitals for TIS treatment. Scenario analyses for patients starting in public hospitals show ICERs of S$99,623 (no subsidy) and S$133,261 (50% subsidy for SCR treatment, no subsidy for TIS), supporting the base case. The projected annual budget impact ranges from S$850,000 to S$3.4 million during the first 5 years.
CONCLUSIONS
TIS for treating r/r DLBCL patients who have failed ≥2 lines of systemic therapies, is likely to be cost effective with limited budget impact.
背景
复发或难治性弥漫性大 B 细胞淋巴瘤（r/r DLBCL）患者的治疗选择有限，预后较差。Tisagenlecleucel（TIS）已显示出改善的临床结果，但成本较高。新加坡拥有多付款人医疗保健系统，私人保险是主要付款人之一。本研究从新加坡私人保险付款人的角度评估了 TIS 与挽救化疗方案（SCR）治疗已接受≥2 线全身治疗后复发或难治性 DLBCL 患者的成本效益和预算影响。
方法
在终生范围内，开发了一个具有三个健康状态的分割生存模型，以评估 TIS 与 SCR 联合或不联合造血干细胞移植（HSCT）的成本效益。TIS 和 SCR 的疗效输入基于 JULIET 和 UPenn 试验的汇总数据以及 CORAL 扩展研究分别观察了 43 个月的数据。包括治疗前、治疗、不良事件、随访、后续-HSCT、复发和终末护理的直接成本。增量成本效益比（ICER）计算为每获得一个质量调整生命年（QALY）的增量总成本。此外，从私人付款人的角度分析了新加坡引入 TIS 的财务影响，比较了当前的治疗途径（不包括 TIS）与未来方案（包括 TIS）在 5 年内的情况。
结果
与 SCR 相比，TIS 是一种更具成本效益的选择，在从私人医院转至公立医院接受 TIS 治疗的私人保险患者中，节省了 8477 新元的费用，并额外获得了 2.78 个 QALYs。对于从公立医院开始的患者的情景分析显示，ICER 分别为 99623 新元（无补贴）和 133261 新元（SCR 治疗补贴 50%，TIS 无补贴），支持基础案例。预计在最初的 5 年内，年度预算影响范围为 850,000 新元至 3400 万新元。
结论
对于已接受≥2 线全身治疗后复发或难治性 DLBCL 患者，TIS 治疗可能具有成本效益，且预算影响有限。
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