Approval gap of pharmacogenomic biomarkers and in vitro companion diagnostics between the United States and Japan.
Approval gap of pharmacogenomic biomarkers and in vitro companion diagnostics between the United States and Japan.
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DOI：
10.1111/jcpt.12129
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发表时间：
2014-04
影响因子：
2
通讯作者：
Ikeda M
中科院分区：
文献类型：
--
作者：
Shimazawa R;Ikeda M
体外伴随诊断器械（CDx）提供了药物基因组学生物标志物（PGBM）的信息，从而能够安全有效地使用靶向药物进行个性化治疗。这些设备需要在科学证据和经济负担之间取得平衡的具体法规。目的是比较PGBM和CDx在美国和日本的批准情况，并帮助为当前关于个性化医疗的讨论提供信息。我们分析了来自美国和日本的CDx和PGBM的已发表文件，这些文件由美国食品药品监督管理局（FDA）列出。评价的方面是目的、批准状态和治疗领域。还调查了日本国民健康保险的覆盖范围。截至2013年3月，FDA表格中列出了38种PGBM。在美国，目标是55%（21/38）的有效性。最大的治疗领域是肿瘤学（39%，15/38）。53%（20/38）的PGBM有相应的CDx批准。在FDA表格中的38种PGBM中，有6种在日本没有批准的药物;在其余32种PGBM中，有16种的目的是有效性。最大的治疗领域是肿瘤学（34%，11/32）。在32例PGBM中，15例与获批和/或涵盖的CDx相关，仅11例具有获批的CDx。4种PGBM在日本未经事先批准的情况下具有涵盖的CDx。我们的研究证实，日本和美国之间在PGBM和CDx的批准方面仍存在巨大差距。然而，国家健康保险对未经批准的CDx的补充覆盖正在将这两个国家的获得提高到类似的水平。由于昂贵的个性化药物和CDx的数量正在增加，患者可及性将继续成为所有国家医疗保健系统的一个重要挑战。
In vitro companion diagnostic devices (CDx) provide information on pharmacogenomic biomarkers (PGBMs) to enable the safe and effective use of targeted agents for personalized therapy. These devices require specific regulations that strike a balance between scientific evidence and financial burden. The aims were to compare approval of PGBMs and CDx in the USA and Japan and to help inform current discussions on personalized medicine. We analysed published documentation from the USA and Japan for CDx and PGBMs, listed by the US Food and Drug Administration (FDA). Aspects evaluated were aim, approval state and therapeutic area. Coverage by the National Health Insurance in Japan was also investigated. Thirty-eight PGBMs were listed in the FDA table as of March 2013. In the USA, the aim was efficacy in 55% (21/38). The largest therapeutic area was oncology (39%, 15/38). Fifty-three per cent (20/38) of the PGBMs had a corresponding CDx approved. Of the 38 PGBMs in the FDA table, six had no approved drug in Japan; in 16 of the remaining 32 PGBMs, the aim was efficacy. The largest therapeutic area was oncology (34%, 11/32). Of the 32 PGBMs, 15 were associated with an approved and/or covered CDx, with only 11 having an approved CDx. Four PGBMs had a covered CDx without prior approval in Japan. Our study confirms that there is still a substantial gap in the approval of PGBMs and CDx between Japan and the USA. Complementary coverage of unapproved CDx by the National Health Insurance, however, is raising access to a similar level in both countries. Because the number of expensive personalized medicines and CDx is increasing, patient access will continue to be an important challenge to healthcare systems in all countries.
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影响因子：
2
作者：
Shimazawa, R.;Ikeda, M.
通讯作者：
Ikeda, M.
DOI：
10.1111/j.1365-2710.2011.01311.x
发表时间：
2012-06-01
影响因子：
2
作者：
Shimazawa, R.;Kusumi, I.;Ikeda, M.
通讯作者：
Ikeda, M.
影响因子：
2.9
作者：
Tobinai, Kensei;Takahashi, Takeshi;Akinaga, Shiro
通讯作者：
Akinaga, Shiro
影响因子：
3.5
作者：
Ozeki, Takeshi;Mushiroda, Taisei;Nakamura, Yusuke
通讯作者：
Nakamura, Yusuke
影响因子：
2.8
作者：
Cohen, J.;Wilson, A.;Manzolillo, K.
通讯作者：
Manzolillo, K.