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Background <p>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.</p> Methods <p>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.</p> Results <p>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 &lt; 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.</p> Conclusions <p>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.</p>

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The nationwide survey of Japanese public opinion about off-label use of anticancer drugs recommended by comprehensive genomic profiling

  • 期刊论文

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  • 发表日期：

    2025年7月18日

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  • Junichi Matsubara,
  • Kumi Mukai,
  • Manabu Muto

摘要

Background

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.

Methods

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.

Results

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.

Conclusions

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.