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Am J Manag Care. 2019 Aug;25(8):379-386.
To date, breakthrough chimeric antigen receptor (CAR) T-cell therapies, such as tisagenlecleucel, indicated for pediatric acute lymphoblastic leukemia (pALL) and diffuse large B-cell lymphoma (DLBCL), and axicabtagene ciloleucel, indicated for DLBCL, although clinically effective, have been limited by treatment delays. Our study measured the social value of CAR T-cell therapy (CAR T) for relapsed or refractory pALL and DLBCL in the United States and quantified social value lost due to treatment delays.
We used an economic framework for therapy valuation, measuring social value as the sum of consumer surplus and manufacturer profit. Consumer surplus is the difference between the value of health gains from a therapy and its incremental cost, while accounting for indirect costs and benefits to patients.
For 20 incident cohorts of pALL (n = 20 × 400 = 8000) and DLBCL (n = 20 × 5902 = 118,040), we quantified patient value, calculated as the value of additional quality-adjusted life-years gained with CAR T, minus the incremental cost of CAR T compared with standard of care (SOC). We calculated manufacturer profits using a range of production costs given uncertainties in the production process. Patient value and manufacturer profits were summed to obtain total social value. We measured social value lost from treatment delays, assuming that patients received the SOC while awaiting CAR T-cell treatment.
Depending on production costs, as much as $6.5 billion and $34.8 billion in social value was generated for patients with pALL and DLBCL, respectively. However, with 1, 2, or 6 months of treatment delay (assuming $200,000 production costs), the pALL population lost 9.8%, 36.2%, and 67.3% of social value, respectively, whereas the DLBCL population lost 4.2%, 11.5%, and 46.0%, relative to no delay.
The social value of CAR T is significantly limited by treatment delays. Efficient payment mechanisms, adequate capital, and payment policy reform are urgently needed to increase patient access and maximize the value of CAR T.
迄今为止，嵌合抗原受体 (CAR) T 细胞疗法取得了突破性进展，例如tisagenlecleucel 用于治疗儿科急性淋巴细胞白血病 (pALL) 和弥漫性大 B 细胞淋巴瘤 (DLBCL)，以及 axicabtagene ciloleucel 用于治疗 DLBCL，尽管具有临床疗效，但由于治疗延迟而受到限制。我们的研究在美国测量了 CAR T 细胞疗法 (CAR T) 治疗复发或难治性 pALL 和 DLBCL 的社会价值，并量化了因治疗延迟而导致的社会价值损失。
我们使用治疗估值的经济框架，将社会价值衡量为消费者剩余和制造商利润的总和。消费者剩余是治疗带来的健康收益价值与其增量成本之间的差异，同时考虑到患者的间接成本和收益。
对于 20 个 pALL 发病队列（n = 20×400 = 8000）和 20 个 DLBCL 发病队列（n = 20×5902 = 118,040），我们量化了患者价值，计算方法是用 CAR T 治疗获得的额外质量调整生命年的价值减去与标准护理 (SOC) 相比的 CAR T 的增量成本。我们使用生产过程不确定性范围内的一系列生产成本来计算制造商的利润。将患者价值和制造商利润相加以获得总社会价值。我们假设患者在等待 CAR T 细胞治疗时接受 SOC，从而衡量了因治疗延迟而导致的社会价值损失。
根据生产成本的不同，pALL 和 DLBCL 患者的社会价值分别高达 65 亿美元和 348 亿美元。然而，治疗延迟 1、2 或 6 个月（假设生产成本为 20 万美元），pALL 患者分别损失了 9.8%、36.2%和 67.3%的社会价值，而 DLBCL 患者分别损失了 4.2%、11.5%和 46.0%，相对于无延迟。
CAR T 的社会价值受到治疗延迟的显著限制。迫切需要有效的支付机制、充足的资金和支付政策改革，以增加患者获得治疗的机会并最大限度地发挥 CAR T 的价值。
