Safety and Evidence of Off-Label Use of Approved Drugs at the National Cancer Center Hospital in Japan
Safety and Evidence of Off-Label Use of Approved Drugs at the National Cancer Center Hospital in Japan
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DOI：
10.1200/op.20.00131
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发表时间：
2021-03-01
影响因子：
4
通讯作者：
Fujiwara, Yasuhiro
中科院分区：
文献类型：
--
作者：
Bun, Seiko;Yonemori, Kan;Fujiwara, Yasuhiro
在日本，药品要纳入公共医疗保险，其有效性和安全性必须首先在临床试验中得到确认。据我们所知，这项研究是第一次在日本的一个高容量癌症治疗中心对药品的标签外使用进行调查。本研究的目的是通过调查东京国立癌症中心医院药品标签外使用的频率和适应症，探索肿瘤学领域未来药物开发和监管批准所需的框架，日本.材料和方法根据已提交给内部审查委员会要求超说明书使用的申请，对2003年至2015年在国家癌症中心医院日常实践中的标签进行回顾性检查。研究期间共收到390份申请。最常用的证明文件是II期试验的结果，其次是病例系列和III期试验。最常使用标签外药物治疗的癌症是肉瘤（15.1%），其次是泌尿系癌症（9.2%）和GI癌症（7.6%）。结论如以往研究所报道，药品通常用于治疗罕见癌症的标签外，大规模的临床试验是很难进行的。有必要继续讨论指导药物开发所需的框架类型。(c)2020年美国临床肿瘤学会
PURPOSE In Japan, for pharmaceutical products to be covered by public medical insurance, their efficacy and safety must first be confirmed in clinical trials. To our knowledge, this study is the first investigation into the off-label use of pharmaceutical products at a high-volume cancer treatment center in Japan. The objective of this study is to explore the framework necessary for future pharmaceutical development and regulatory approval in the field of oncology by surveying the frequency of and indications for off-label use of pharmaceutical products at the National Cancer Center Hospital in Tokyo, Japan.MATERIALS AND METHODS The pharmaceutical products used off-label in daily practice from 2003 to 2015 at the National Cancer Center Hospital were retrospectively examined based on applications that had been submitted to an internal review committee requesting off-label use.RESULTS A total of 1,390 applications were submitted during the study period. The most frequently used supporting documents were the results of phase II trials, followed by case series and phase III trials. The cancer most frequently treated with off-label drugs was sarcoma (15.1%), followed by urologic cancer (9.2%) and GI cancer (7.6%).CONCLUSION As reported in previous studies, pharmaceutical products were generally used off-label for the treatment of rare cancers, for which large-scale clinical trials are difficult to conduct. Continued discussion of the types of frameworks that are needed to guide pharmaceutical development is necessary. (c) 2020 by American Society of Clinical Oncology