Department of Radiation Oncology, Saitama Medical University International Medical Center, 1397-1 Yamane Hidaka, Saitama, 350-1298, Japan.
Daru. 2013 May 25;21(1):40. doi: 10.1186/2008-2231-21-40.
In Japan, both incidence and mortality rates of cancers have continuously increased and medical costs are growing more rapidly than the overall economy of Japan. However, there is no consensus threshold for cost-effectiveness in medical care, and few studies have investigated cost-effectiveness of medical care in Japan. The present study was to determine the direct costs of molecular-targeting drugs that were recently approved in Japan through simple and quantitative calculations. Thus, we calculated an incremental cost-effectiveness ratio (ICER) and the cost per life-year gained (LYG) by using reported data from randomized clinical trials for various cancers.
Between 2008 and 2011, we reviewed seven molecular-targeting drugs that were approved for treatment of five cancers in Japan. These drugs included Bevacizumab, sorafenib, sunitinib, temsirolimus, Lapatinib, and panitumumab. Direct cost, ICER, and LYG of the drugs were estimated from the randomized phase III clinical trial data referred to in package leaflets. Effectiveness was defined as the prolongation of both median overall survival (OS) and progression-free survival (PFS). Costs were calculated as those of molecular-targeting drugs. Subsequently, ICER was based on 1-month increases in both OS and PFS periods and 1% increases in OS, and LYG was determined.
Direct costs ranged from ¥724,804 ($9,060) to ¥1,506,628 ($18,833). ICERs of the drugs ranged from ¥724,804 ($9,060) to ¥1,506,628 ($18,833) for a 1-month increase in OS. For each month of PFS, ICERs ranged from ¥372,243 ($4,653) to ¥7,399,877 ($92,498). The costs of Bevacizumab and sorafenib for treatment of HCC per 1% increase in OS were ¥376,657 ($4,708) and ¥313,733 ($3,922), respectively. LYG ranged from ¥8,697,650 ($108,721) to ¥18,079,530 ($225,994).
Some molecular-targeting drugs are not cost-effective. Considering ethical and moral issues, we should establish economic endpoints to approve new drugs in Japan.
在日本，癌症的发病率和死亡率持续上升，医疗成本的增长速度超过了日本整体经济的增长速度。然而，对于医疗保健的成本效益，目前还没有达成共识的阈值，而且很少有研究调查日本的医疗保健成本效益。本研究旨在通过简单和定量的计算，确定最近在日本获得批准的分子靶向药物的直接成本。因此，我们使用各种癌症的随机临床试验报告数据计算了增量成本效益比（ICER）和每获得一个生命年的成本（LYG）。
在 2008 年至 2011 年期间，我们回顾了在日本批准用于治疗五种癌症的七种分子靶向药物。这些药物包括贝伐单抗、索拉非尼、舒尼替尼、替西罗莫司、拉帕替尼和帕尼单抗。从包装说明书中提到的随机 III 期临床试验数据中估算了药物的直接成本、ICER 和 LYG。有效性定义为中位总生存期（OS）和无进展生存期（PFS）的延长。成本计算为分子靶向药物的成本。随后，基于 OS 和 PFS 期的 1 个月增加以及 OS 的 1%增加，计算了 ICER，确定了 LYG。
直接成本从 724,804 日元（9,060 美元）到 1,506,628 日元（18,833 美元）不等。药物的 ICER 从 724,804 日元（9,060 美元）到 1,506,628 日元（18,833 美元）不等，OS 增加 1 个月。对于 PFS 的每个月，ICER 从 372,243 日元（4,653 美元）到 7,399,877 日元（92,498 美元）不等。贝伐单抗和索拉非尼治疗 HCC 时，OS 每增加 1%的成本分别为 376,657 日元（4,708 美元）和 313,733 日元（3,922 美元）。LYG 从 8,697,650 日元（108,721 美元）到 18,079,530 日元（185,994 美元）不等。
一些分子靶向药物没有成本效益。考虑到伦理和道德问题，我们应该在日本建立经济终点来批准新药。
