ReviewInternational journal of clinical oncology2025
The nationwide survey of Japanese public opinion about off-label use of anticancer drugs recommended by comprehensive genomic profiling.
Review in International journal of clinical oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Authors and funding
3 authors.
Funding
Abstract
backgroundComprehensive 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.
methodsA 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.
resultsA 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.
conclusionsOver 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.
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Registered trials
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