Matsubara Junichi, Mukai Kumi, Muto Manabu
Department of Clinical Oncology, Kyoto University Hospital, 54 Shogoinkawahara-Cho, Sakyo-Ku, Kyoto, 606-8507, Japan.
Int J Clin Oncol. 2025 Jul 18. doi: 10.1007/s10147-025-02809-y.
Comprehensive genomic profiling (CGP)-guided precision medicine enables identification of molecular-based recommended therapy (MBRT), including off-label uses of anticancer drugs for rare genomic alterations. However, in Japan, access to such off-label drugs is limited despite their potential therapeutic benefits. This study aimed to investigate public attitudes toward off-label use of anticancer drugs in Japan.
A nationwide online survey was conducted in Japan from February 15 to 19, 2024, targeting cancer patients (CA), medical professionals (MP), and non-cancer volunteers (non-CA) aged 40 + years. This included explanatory materials on CGP and MBRT, and questionnaires assessing willingness to use off-label drugs in various cost scenarios.
A total of 1,261 responses were analyzed: 419 CA, 430 MP, and 412 non-CA participants (median age: 59, range 40-89). Approximately 80% of MPs reported high comprehension of the explanatory materials (Top-2 box on a 5-point Likert scale), compared with ~ 60% of CA and < 50% of non-CA participants. Willingness to use off-label drugs (Top-2 box) was as follows: "No cost burden": 51% CA, 62% MP, and 50% non-CA; "Cost ¥200,000 per month (approximately $US1,300)": 15% CA, 31% MP, and 15% non-CA; "Cost ¥1,000,000 per month (approximately $US6,700)": 6% CA, 16% MP, and 4% non-CA. Higher comprehension of explanatory materials was associated with greater willingness to use off-label drugs.
Over half of respondents were willing to use off-label anticancer drugs if they were free of charge. However, willingness declined significantly with cost. Policy frameworks are needed in Japan to improve access to CGP-guided off-label therapies.
综合基因组分析（CGP）指导下的精准医疗能够确定基于分子的推荐疗法（MBRT），包括针对罕见基因组改变的抗癌药物的非标签使用。然而，在日本，尽管这些非标签药物具有潜在的治疗益处，但获取此类药物仍受到限制。本研究旨在调查日本公众对抗癌药物非标签使用的态度。
2024年2月15日至19日在日本进行了一项全国性在线调查，目标人群为40岁及以上的癌症患者（CA）、医学专业人员（MP）和非癌症志愿者（非CA）。调查内容包括关于CGP和MBRT的解释材料，以及评估在各种成本情况下使用非标签药物意愿的问卷。
共分析了1261份回复：419名癌症患者、430名医学专业人员和412名非癌症志愿者（中位年龄：59岁，范围40 - 89岁）。约80%的医学专业人员表示对解释材料理解程度高（5分李克特量表中的前两档），相比之下，约60%的癌症患者和不到50%的非癌症志愿者达到这一水平。使用非标签药物的意愿（前两档）如下：“无成本负担”：51%的癌症患者、62%的医学专业人员和50%的非癌症志愿者；“每月成本20万日元（约1300美元）”：15%的癌症患者、31%的医学专业人员和15%的非癌症志愿者；“每月成本100万日元（约6700美元）”：6%的癌症患者、16%的医学专业人员和4%的非癌症志愿者。对解释材料理解程度越高，使用非标签药物的意愿越强。
超过一半的受访者表示如果抗癌药物免费，愿意使用非标签药物。然而，随着成本增加，意愿显著下降。日本需要政策框架来改善获取CGP指导下的非标签疗法的机会。
