Bassali Jan, Gould Ian Gopal, Kaye James A, Mladsi Deirdre, Mehta Jyotsna
Global Medical and Scientific Affairs Department , Karyopharm Therapeutics, Newton, MA, 02459, USA.
Research Health Economy, RTI Health Solutions, Research Triangle Park, NC 27709-2194, USA.
Clinicoecon Outcomes Res. 2020 Jun 19;12:317-325. doi: 10.2147/CEOR.S251070. eCollection 2020.
To estimate the budgetary impact of adopting selinexor (XPOVIO; Karyopharm Therapeutics, Inc.) for the treatment of adult patients with penta-refractory multiple myeloma (MM) from the perspective of a third-party payer in the United States (US).
A budget impact analysis was conducted in one-year increments for the first 3 years after the introduction of selinexor for a private payer or Medicare Part D. Total annual treatment costs (2018 US dollars) were calculated as the sum of drug costs, costs of adverse events (AEs; grade ≥3), along with ongoing best supportive care costs. The number of eligible patients was derived from national epidemiology statistics, healthcare databases, and published literature.
In the base-case analysis, selinexor was associated with a per member per month (PMPM) cost of $0.0103 in year 3, assuming a market uptake of 64%, for a hypothetical private payer plan with one million members and four eligible patients. In a scenario analysis with 16 eligible patients with triple-class refractory MM regardless of the line of therapy (this additional scenario analysis was performed with an eligible population that does not fit squarely within the approved label for selinexor but was performed strictly for the purpose of demonstrating the results of the budget impact model when based on a larger pool of eligible patients), the estimated PMPM cost in year 3 was $0.0388. The model showed comparable sensitivity to treatment duration, wholesale acquisition cost for selinexor, and year 1 uptake. The base-case analysis conducted from the perspective of Medicare Part D was associated with a PMPM cost of $0.0078 in year 3 with 159 eligible patients.
The model estimates a small and manageable budget impact of adopting selinexor into a third-party US payer plan, given the low prevalence of penta-refractory MM.
从美国第三方支付方的角度，评估采用塞利尼索（XPOVIO；Karyopharm Therapeutics公司）治疗五重难治性成年多发性骨髓瘤（MM）患者的预算影响。
在塞利尼索引入后的前3年，以一年为增量对私人支付方或医疗保险D部分进行预算影响分析。年度总治疗成本（2018年美元）计算为药物成本、不良事件（AE；≥3级）成本以及持续的最佳支持治疗成本之和。符合条件的患者数量来自国家流行病学统计、医疗保健数据库和已发表的文献。
在基础病例分析中，假设市场占有率为64%，对于一个拥有100万成员且有4名符合条件患者的假设性私人支付方计划，塞利尼索在第3年的人均每月成本（PMPM）为0.0103美元。在一个有16名三重难治性MM符合条件患者的情景分析中（无论治疗线数如何；此额外情景分析是针对不完全符合塞利尼索批准标签的符合条件人群进行的，但严格来说是为了展示基于更大符合条件患者群体的预算影响模型的结果），第3年估计的PMPM成本为0.0388美元。该模型对治疗持续时间、塞利尼索的批发采购成本和第1年的市场占有率表现出类似的敏感性。从医疗保险D部分的角度进行的基础病例分析在第3年有159名符合条件患者时，PMPM成本为0.0078美元。
鉴于五重难治性MM的低患病率，该模型估计将塞利尼索纳入美国第三方支付方计划的预算影响较小且可控。
