Thach Andrew, Kirson Noam, Zichlin Miriam L, Dieye Ibrahima, Pappert Eric, Williams G Rhys
Sunovion Pharmaceuticals Inc.
Analysis Group, Inc.
J Health Econ Outcomes Res. 2021 Nov 17;8(2):82-92. doi: 10.36469/jheor.2021.29488. eCollection 2021.
"On-demand" treatments approved in the United States (US) for "OFF" episodes in Parkinson's disease (PD) include apomorphine hydrochloride injection (SC-APO), apomorphine sublingual film (APL), and levodopa inhalation powder (CVT-301). APL received US approval in 2020, and its cost-effectiveness has not been compared with SC-APO and CVT-301. To develop a cost-effectiveness analysis model comparing APL versus SC-APO and CVT-301 for treatment of patients with PD experiencing "OFF" episodes from a US payer perspective. The model estimated total costs and effectiveness for each comparator arm, informed from the treatments' pivotal studies or literature, over a 10-year horizon. Total and incremental patient costs (in 2020 US dollars), total time spent without "OFF" episode symptoms, and quality-adjusted life years (QALY) gained were summarized and compared. Incremental cost-effectiveness ratios for APL versus SC-APO and CVT-301 were estimated and expressed as incremental patient costs per patient QALY gained and incremental cost per "OFF" hour avoided. Scenario analyses varying inputs and including caregiver costs were also conducted. In the base case, APL had the lowest total "on-demand" treatment costs ($42,095) compared with SC-APO ($276,320; difference: -$234,225) and CVT-301 ($69,577; difference: -$27,482) over the 10-year horizon. APL was also associated with the highest utility, with incremental QALYs of 0.019 versus SC-APO and 0.235 versus CVT-301. APL was dominant over CVT-301 in terms of incremental cost per "OFF" hour, and dominant over both CVT-301 and SC-APO in terms of incremental cost per QALY gained. In all scenario analyses, APL was dominant against both SC-APO and CVT-301, confirming the robustness of the base-case results. APL was dominant compared with both comparator arms, being less costly and more effective on average than SC-APO and CVT-301 in terms of QALYs. For SC-APO, cost-effectiveness of APL was driven by lower "on-demand" treatment costs and adverse event-related disutilities. For CVT-301, cost-effectiveness of APL was driven by lower "on-demand" treatment costs and substantially higher efficacy. From a US payer perspective, APL represents a cost-effective option compared with SC-APO and CVT-301 for treatment of "OFF" episodes in patients with PD.
在美国被批准用于治疗帕金森病（PD）“关”期发作的“按需”治疗药物包括盐酸阿扑吗啡注射液（皮下注射阿扑吗啡，SC - APO）、阿扑吗啡舌下薄膜片（APL）和左旋多巴吸入粉（CVT - 301）。APL于2020年获得美国批准，其成本效益尚未与SC - APO和CVT - 301进行比较。为了从美国医保支付方的角度建立一个成本效益分析模型，比较APL与SC - APO和CVT - 301用于治疗经历“关”期发作的PD患者的情况。该模型根据治疗的关键研究或文献，估计了每个比较组在10年期间的总成本和有效性。总结并比较了患者的总费用和增量费用（以2020年美元计）、无“关”期发作症状的总时间以及获得的质量调整生命年（QALY）。估计了APL与SC - APO和CVT - 301相比的增量成本效益比，并表示为每获得一个患者QALY的增量患者成本以及每避免一小时“关”期的增量成本。还进行了改变输入参数并纳入护理人员成本的情景分析。在基础案例中，在10年期间APL的“按需”治疗总成本最低（42,095美元），相比之下SC - APO为276,320美元（差值： - 234,225美元），CVT - 301为69,577美元（差值： - 27,482美元）。APL还具有最高的效用，与SC - APO相比增量QALY为0.019，与CVT - 301相比为0.235。就每“关”期小时的增量成本而言，APL优于CVT - 301；就每获得一个QALY的增量成本而言，APL优于CVT - 301和SC - APO。在所有情景分析中，APL相对于SC - APO和CVT - 301均占主导地位，证实了基础案例结果的稳健性。与两个比较组相比，APL占主导地位，在QALY方面平均成本更低且效果更好。对于SC - APO，APL的成本效益是由较低的“按需”治疗成本和不良事件相关的负效用驱动的。对于CVT - 301，APL的成本效益是由较低的“按需”治疗成本和显著更高的疗效驱动的。从美国医保支付方的角度来看，与SC - APO和CVT - 301相比，APL是治疗PD患者“关”期发作的一种具有成本效益的选择。
