Cost Effectiveness of Chimeric Antigen Receptor T-Cell Therapy in Multiply Relapsed or Refractory Adult Large B-Cell Lymphoma
Cost Effectiveness of Chimeric Antigen Receptor T-Cell Therapy in Multiply Relapsed or Refractory Adult Large B-Cell Lymphoma
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DOI：
10.1200/jco.18.02079
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发表时间：
2019-08-20
影响因子：
45.3
通讯作者：
Goldhaber-Fiebert, Jeremy D.
中科院分区：
文献类型：
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作者：
Lin, John K.;Muffly, Lori S.;Goldhaber-Fiebert, Jeremy D.
两种抗CD 19嵌合抗原受体T细胞（CAR-T）疗法被批准用于弥漫性大B细胞淋巴瘤，axicabtagene ciloleucel（axi-cel）和tisagenlecleucel;每种疗法的成本为373，000美元。我们评估了他们的cost effectiveness.METHODS我们使用了决策分析马尔可夫模型通知最近的多中心，单臂试验，以评估axi-cel和tisagenlecleucel在多次复发/难治性，成人，弥漫性大B细胞淋巴瘤从美国健康支付人的角度来看，在一生的地平线。在一系列合理的长期有效性假设下，将每种治疗与补救性化学免疫治疗方案和干细胞移植进行比较。主要结果是未贴现生命年、贴现生命周期成本、贴现质量调整生命年（QHMS）和增量成本效益比（3%的年贴现率）。结果在乐观的情况下，假设5年无进展生存期（PFS）为40%，axi-cel将预期寿命延长8.2年，增加129，000美元/QALY（95%不确定区间，90，000美元至219，000美元）。在30%的5年PFS时，预期寿命的改善更为温和（6.4年）和昂贵（159，000美元/QALY增加[95%不确定性区间，105，000美元至284，000美元]）。在乐观的情况下，假设5年PFS为35%，tisagenlecleucel使预期寿命延长4.6年，获得168，000美元/QALY（95%不确定性区间，105，000美元至414，000美元/QALY）。在25%的5年PFS时，预期寿命的改善更小（3.4年），成本更高（增加223，000美元/QALY [95%不确定区间，123，000美元至1，170，000美元/QALY]）。对所有指定患者进行CAR-T治疗将在5年内增加约100亿美元的美国医疗保健成本。价格分别降低至25万美元和20万美元，或仅支付初始完全缓解（按当前价格），将使axi-cel和tisagenlecleucel的成本低于15万美元/QALY，即使在25%PFS.结论在2018年的价格下，两种CAR-T疗法都可能达到低于15万美元/QALY的阈值。这取决于与化学免疫疗法和干细胞移植相比的长期结果，后者尚不确定。广泛采用将大大增加非霍奇金淋巴瘤的医疗保健费用。降价或支付初步反应费用将提高成本效益，即使长期效果不大。
PURPOSE Two anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapies are approved for diffuse large B-cell lymphoma, axicabtagene ciloleucel (axi-cel) and tisagenlecleucel; each costs $373,000. We evaluated their cost effectiveness.METHODS We used a decision analytic Markov model informed by recent multicenter, single-arm trials to evaluate axi-cel and tisagenlecleucel in multiply relapsed/refractory, adult, diffuse large B-cell lymphoma from a US health payer perspective over a lifetime horizon. Under a range of plausible long-term effectiveness assumptions, each therapy was compared with salvage chemoimmunotherapy regimens and stem-cell transplantation. Main outcomes were undiscounted life years, discounted lifetime costs, discounted quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (3% annual discount rate). Sensitivity analyses explored uncertainty.RESULTS In an optimistic scenario, assuming a 40% 5-year progression-free survival (PFS), axi-cel increased life expectancy by 8.2 years at $129,000/QALY gained (95% uncertainty interval, $90,000 to $219,000). At a 30% 5-year PFS, improvements in life expectancy were more modest (6.4 years) and expensive ($159,000/QALY gained [95% uncertainty interval, $105,000 to $284,000]). In an optimistic scenario, assuming a 35% 5-year PFS, tisagenlecleucel increased life expectancy by 4.6 years at $168,000/QALY gained (95% uncertainty interval, $105,000 to $414,000/QALY). At a 25% 5-year PFS, improvements in life expectancy were smaller (3.4 years) and more expensive ($223,000/QALY gained [95% uncertainty interval, $123,000 to $1,170,000/QALY]). Administering CAR-T to all indicated patients would increase US health care costs by approximately $10 billion over 5 years. Price reductions to $250,000 and $200,000, respectively, or payment only for initial complete response (at current prices) would allow axi-cel and tisagenlecleucel to cost less than $150,000/QALY, even at 25% PFS.CONCLUSION At 2018 prices, it is possible that both CAR-T therapies meet a less than $150,000/QALY threshold. This depends on long-term outcomes compared with chemoimmunotherapy and stem-cell transplantation, which are uncertain. Widespread adoption would substantially increase non-Hodgkin lymphoma health care costs. Price reductions or payment for initial response would improve cost effectiveness, even with modest long-term outcomes.