Padula William V, Larson Richard A, Dusetzina Stacie B, Apperley Jane F, Hehlmann Rudiger, Baccarani Michele, Eigendorff Ekkehard, Guilhot Joelle, Guilhot Francois, Hehlmann Rudiger, Mahon Francois-Xavier, Martinelli Giovanni, Mayer Jiri, Müller Martin C, Niederwieser Dietger, Saussele Susanne, Schiffer Charles A, Silver Richard T, Simonsson Bengt, Conti Rena M
Affiliations of authors:Department of Health Policy and Management, Johns Hopkins University , Baltimore, MD (WVP); Department of Medicine (RAL) and Departments of Pediatrics and of Public Health Sciences (RMC), University of Chicago, Chicago, IL; Eshelman School of Pharmacy and Gillings School of Global Public Health, Lineberger Comprehensive Cancer Center , and Cecil G. Sheps Center for Health Services Research, University of North Carolina , Chapel Hill, NC (SBD); Department of Haematology, Hammersmith Hospital, Imperial College , London , UK (JFA); Department of Medicine (RH, SS) and Department of Hematology and Oncology (MCM), University of Heidelberg, Mannheim, Germany; Department of Haematology and Oncology, S. Orsola-Malpighi University Hospital (MB), and Department of Hematology, "L. e A. Seragnoli" (GM), University of Bologna , Bologna , Italy ; Department of Haematology and Oncology, University Hospital , Jena , Germany (EE); INSERM Centre d'Investigation Clinique (CIC) 1402, CHU de Poitiers, Poitiers, France (JG, FG); Laboratoire d'Hematologie, Universite Victor Segalen, Bordeaux, Pessac, France (FXM); Department of Internal Medicine, Hematology and Oncology, University Hospital Brno , Brno , Czech Republic (JM); Department of Hematology and Oncology, University Hospital Leipzig , Leipzig , Germany (DN); Barbara Ann Karmanos Cancer Institute, Wayne State University , Detroit, MI (CAS); Department of Medicine, Weill Cornell Medical Center , New York, NY (RTS); Uppsala University , Uppsala , Sweden (BS).
J Natl Cancer Inst. 2016 Mar 4;108(7). doi: 10.1093/jnci/djw003. Print 2016 Jul.
We analyzed the cost-effectiveness of treating incident chronic myeloid leukemia in chronic phase (CML-CP) with generic imatinib when it becomes available in United States in 2016. In the year following generic entry, imatinib's price is expected to drop 70% to 90%. We hypothesized that initiating treatment with generic imatinib in these patients and then switching to the other tyrosine-kinase inhibitors (TKIs), dasatinib or nilotinib, because of intolerance or lack of effectiveness ("imatinib-first") would be cost-effective compared with the current standard of care: "physicians' choice" of initiating treatment with any one of the three TKIs.
We constructed Markov models to compare the five-year cost-effectiveness of imatinib-first vs physician's choice from a US commercial payer perspective, assuming 3% annual discounting ($US 2013). The models' clinical endpoint was five-year overall survival taken from a systematic review of clinical trial results. Per-person spending on incident CML-CP treatment overall care components was estimated using Truven's MarketScan claims data. The main outcome of the models was cost per quality-adjusted life-year (QALY). We interpreted outcomes based on a willingness-to-pay threshold of $100 000/QALY. A panel of European LeukemiaNet experts oversaw the study's conduct.
Both strategies met the threshold. Imatinib-first ($277 401, 3.87 QALYs) offered patients a 0.10 decrement in QALYs at a savings of $88 343 over five years to payers compared with physician's choice ($365 744, 3.97 QALYs). The imatinib-first incremental cost-effectiveness ratio was approximately $883 730/QALY. The results were robust to multiple sensitivity analyses.
When imatinib loses patent protection and its price declines, its use will be the cost-effective initial treatment strategy for CML-CP.
我们分析了2016年非专利伊马替尼在美国上市后,用于治疗初发慢性期慢性髓性白血病(CML-CP)的成本效益。在非专利药上市后的第一年,伊马替尼的价格预计将下降70%至90%。我们假设,在这些患者中先用非专利伊马替尼进行治疗,然后由于不耐受或缺乏疗效而改用其他酪氨酸激酶抑制剂(TKIs),即达沙替尼或尼罗替尼(“伊马替尼优先”),与当前的标准治疗方案相比具有成本效益:医生选择三种TKIs中的任何一种开始治疗。
我们构建了马尔可夫模型,从美国商业医保支付方的角度比较“伊马替尼优先”与医生选择的五年成本效益,假设年贴现率为3%(2013年美元)。模型的临床终点是从临床试验结果的系统评价中得出的五年总生存率。使用Truven的MarketScan索赔数据估算初发CML-CP治疗总体护理成分的人均支出。模型的主要结果是每质量调整生命年(QALY)的成本。我们根据每QALY支付意愿阈值10万美元来解释结果。由欧洲白血病网专家小组监督该研究的实施。
两种策略均达到阈值。与医生选择(365744美元,3.97 QALYs)相比,“伊马替尼优先”(277401美元,3.87 QALYs)使患者的QALYs减少0.10,但为支付方节省了88343美元的五年费用。“伊马替尼优先”的增量成本效益比约为883730美元/QALY。结果在多次敏感性分析中均很稳健。
当伊马替尼失去专利保护且价格下降时,其使用将成为CML-CP具有成本效益的初始治疗策略。