Division of Pharmaceutical Outcomes and Policy, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Division of Pharmaceutical Outcomes and Policy, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Cecil G. Sheps Center for Health Services Research, Chapel Hill, NC, USA.
Value Health. 2020 Oct;23(10):1292-1299. doi: 10.1016/j.jval.2020.05.019. Epub 2020 Aug 7.
Overall survival in chronic myeloid leukemia (CML) in chronic phase is not significantly different by treatment with first-line tyrosine kinase inhibitors (TKIs), but emerging evidence reveals differences in costs and safety profiles. We evaluated the 1-year cost-effectiveness of TKI initiation with imatinib, dasatinib, or nilotinib among a hypothetical cohort of incident patients with CML from a US payer's perspective.
We constructed a decision analytic model to assess quality-adjusted life years (QALYs), healthcare costs, net monetary benefit, and incremental cost-effectiveness of treatment strategies. We used published studies and data from the IBM Watson Health MarketScan database for model parameters. To calculate TKI costs, we used the 2018 Federal Supply Schedule estimates for generic imatinib and branded second-generation TKIs. We evaluated cost-effectiveness under various willingness-to-pay thresholds. We accounted for uncertainty with deterministic and probabilistic sensitivity analyses.
In the base-case analysis, imatinib was favored over dasatinib and nilotinib at a lower cost per QALY gained. Imatinib remained the favored strategy after 1-way variations in TKI costs, TKI switching, QALYs, adverse event risk, and CML progression. When we assessed model uncertainty with prespecified parameter distributions, imatinib was cost-saving compared with dasatinib in 40% of 100 0000 simulations and was favored over all simulations compared with nilotinib. First-line treatment with second-generation TKIs was cost-effective in 50% of simulations at a $200 000/QALY willingness-to-pay threshold.
Generic availability of imatinib provides a more cost-effective treatment approach in the first year compared with other available TKIs for newly diagnosed patients with CML.
慢性期慢性髓性白血病(CML)患者接受一线酪氨酸激酶抑制剂(TKI)治疗的总生存期并无显著差异,但新出现的证据显示其成本和安全性特征存在差异。我们从美国支付者的角度评估了伊马替尼、达沙替尼或尼洛替尼用于初诊 CML 患者的 1 年成本效益。
我们构建了一个决策分析模型,以评估质量调整生命年(QALY)、医疗保健成本、净货币收益和治疗策略的增量成本效益。我们使用了已发表的研究和 IBM Watson Health MarketScan 数据库的数据来获取模型参数。为了计算 TKI 成本,我们使用了 2018 年联邦供应时间表对通用伊马替尼和品牌第二代 TKI 的估计值。我们在不同的支付意愿阈值下评估了成本效益。我们通过确定性和概率敏感性分析来考虑不确定性。
在基础案例分析中,与达沙替尼和尼洛替尼相比,伊马替尼具有更低的成本效益。在 TKI 成本、TKI 转换、QALY、不良事件风险和 CML 进展等方面进行单向变化后,伊马替尼仍然是首选策略。当我们使用预设参数分布评估模型不确定性时,在 1000000 次模拟中的 40%中,伊马替尼的成本效益优于达沙替尼,与尼洛替尼相比,在所有模拟中均占优势。在 200000 美元/QALY 的支付意愿阈值下,二线 TKI 的一线治疗在 50%的模拟中具有成本效益。
与其他可用的 TKI 相比,伊马替尼的通用可用性为新诊断的 CML 患者提供了一种更具成本效益的治疗方法。