Dai Wei Fang, Beca Jaclyn M, Nagamuthu Chenthila, Liu Ning, de Oliveira Claire, Earle Craig C, Trudeau Maureen, Chan Kelvin K W
Temerty Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.
Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada.
JAMA Oncol. 2022 Apr 1;8(4):597-606. doi: 10.1001/jamaoncol.2021.8049.
The initial assessment of pertuzumab use for treatment of metastatic breast cancer by health technology assessment agencies suggested that pertuzumab was not cost-effective. In Ontario, Canada, pertuzumab became funded in November 2013 based on the substantial clinical benefit. To date, there is a paucity of analysis of pertuzumab using real-world data for cost-effectiveness.
To assess the cost-effectiveness of pertuzumab, trastuzumab, and chemotherapy vs trastuzumab and chemotherapy for patients with metastatic breast cancer.
DESIGN, SETTING, AND PARTICIPANTS: A population-based retrospective economic evaluation was conducted in Ontario, Canada. Patients who received first-line treatments for metastatic breast cancer from January 1, 2008, to March 31, 2018, were identified. Patients were followed up from the start of treatment up to 5 years, with maximum follow-up to March 31, 2019. Patients were identified from the Ontario Cancer Registry and linked to the New Drug Funding Program database to identify receipt of first-line treatment (N = 1158).
Treatment with pertuzumab, trastuzumab, and chemotherapy after public funding (November 25, 2013) compared with treatment with trastuzumab and chemotherapy before funding.
Cost-effectiveness, from a public payer perspective, was estimated from administrative data with a 5-year time horizon, adjusted for censoring, and discounted (1.5%). Incremental cost-effectiveness ratios for life-years gained and quality-adjusted life year (QALY) with bootstrapped 95% CIs were calculated. Sensitivity analysis with price reduction of pertuzumab alone or in combination with trastuzumab was conducted.
A total of 579 pairs of matched patients receiving pertuzumab and controls were included. The mean (SD) age of the matched study cohort was 58 (12.97) years; 1151 were women (99.4%). Pertuzumab resulted in 0.61 life-years gained and 0.44 QALYs gained at an incremental cost of $192 139 (all costs measured in Canadian dollar values, CAD) with an incremental cost-effectiveness ratio of $316 203 per life-year gained and $436 679 per QALY. The main factors associated with cost included the cost of pertuzumab (60%), outpatient cancer treatment delivery (24%), and trastuzumab (15%). With 100% price reduction of pertuzumab, the incremental cost-effectiveness ratio was $174 027 per QALY. When the price of pertuzumab and trastuzumab were both reduced by more than 71%, the incremental cost-effectiveness ratio decreased below $100 000 per QALY.
The findings of this population-based study suggest that pertuzumab may increase survival for patients with metastatic breast cancer but would not be considered cost-effective, even after 100% price reduction, under conventional thresholds.
卫生技术评估机构对帕妥珠单抗用于治疗转移性乳腺癌的初步评估表明,帕妥珠单抗不具有成本效益。在加拿大安大略省,基于显著的临床获益,帕妥珠单抗于2013年11月开始获得资助。迄今为止,缺乏使用真实世界数据对帕妥珠单抗进行成本效益分析的研究。
评估帕妥珠单抗、曲妥珠单抗及化疗联合方案对比曲妥珠单抗及化疗方案用于转移性乳腺癌患者的成本效益。
设计、地点和参与者:在加拿大安大略省进行了一项基于人群的回顾性经济评估。确定了2008年1月1日至2018年3月31日期间接受转移性乳腺癌一线治疗的患者。从治疗开始对患者进行长达5年的随访,最长随访至2019年3月31日。从安大略癌症登记处识别患者,并与新药资助计划数据库相链接,以确定一线治疗的接受情况(N = 1158)。
将2013年11月25日公共资助后使用帕妥珠单抗、曲妥珠单抗及化疗的治疗方案与资助前使用曲妥珠单抗及化疗的治疗方案进行比较。
从公共支付方的角度,利用行政数据在5年时间范围内估计成本效益,并对删失数据进行调整,贴现率为1.5%。计算获得生命年和质量调整生命年(QALY)的增量成本效益比及自抽样的95%置信区间。对仅帕妥珠单抗或帕妥珠单抗与曲妥珠单抗联合降价进行敏感性分析。
共纳入579对接受帕妥珠单抗治疗的匹配患者及对照。匹配研究队列的平均(标准差)年龄为58(12.97)岁;1151例为女性(99.4%)。帕妥珠单抗使患者获得0.61个生命年和0.44个QALY,增量成本为192139加元(所有成本以加元计,CAD),增量成本效益比为每获得1个生命年316203加元,每获得1个QALY 436679加元。与成本相关的主要因素包括帕妥珠单抗成本(60%)、门诊癌症治疗费用(24%)和曲妥珠单抗成本(15%)。若帕妥珠单抗价格降低100%,增量成本效益比为每QALY 174027加元。当帕妥珠单抗和曲妥珠单抗价格均降低超过71%时,增量成本效益比降至每QALY低于100000加元。
这项基于人群的研究结果表明,帕妥珠单抗可能会提高转移性乳腺癌患者的生存率,但按照传统阈值,即使价格降低100%,也不具有成本效益。