The drug lag and associated factors for orphan anticancer drugs in Japan compared to the United States
The drug lag and associated factors for orphan anticancer drugs in Japan compared to the United States
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DOI：
10.1007/s10637-018-0612-y
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发表时间：
2019-10-01
影响因子：
3.4
通讯作者：
Tsukamoto, Katsura
中科院分区：
文献类型：
--
作者：
Nakayama, Hiroki;Matsumaru, Naoki;Tsukamoto, Katsura
日本孤儿抗癌药物获批增加以满足较高社会需求药品滞后，即新药审批滞后，在日本被认为是一个社会问题。我们调查了日本和美国之间的审批滞后及其组成部分，提交滞后和审查滞后，以揭示审批滞后是否仍然存在，并确定可能有助于缩短审批滞后的潜在因素。利用公开信息对2004年4月至2017年11月期间在日本批准的抗癌药物进行了调查。结果显示，2016年-2017年孤儿抗癌药审批滞后中位数为727.0天(四分位数区间，IQR为310.0-1054.3)。审批滞后与提交滞后显著相关(相关系数=1.00，P<；0.001)，而与审查滞后无关(相关系数=-0.16，P=0.22)。孤儿抗癌药物的提交延迟显著长于非孤儿药物(中位数，712.5天[智商，186.0-1448.3]对387.0天[92.8-1096.0]，P=0.023)。药物开发的外部合作与较长的提交延迟相关(系数=762.1，P=0.017)，而美国的突破性治疗指定与较短的提交延迟(系数=-832.8，P=0.035)相关。综上所述，我们揭示了2016-2017年间孤儿抗癌药的审批滞后现象仍然存在。孤儿抗癌药的提交滞后是影响审批滞后的主要因素，且比非孤儿药的提交滞后时间长。药物开发方面的外部合作可能是减少孤儿抗癌药物提交滞后的一个潜在因素。
The approval of orphan anticancer drugs in Japan has increased to meet high social demand. Drug lag, namely the approval lag of new drugs, is recognized as a social issue in Japan. We investigated the approval lag and its components, submission lag and review-time lag, between Japan and the United States (US) to reveal whether an approval lag still exists, and to identify potential factors that may contribute to reducing the approval lag. Anticancer drugs approved in Japan between April 2004 and November 2017 were investigated using publicly available information. Results showed that the median approval lag of orphan anticancer drugs in 2016-2017 was 727.0 days (interquartile range, IQR, 310.0-1054.3). The approval lag was significantly correlated with the submission lag (correlation coefficient = 1.00, P < 0.001) but not with the review-time lag (correlation coefficient = -0.16, P = 0.22). The submission lag was significantly longer for orphan anticancer drugs than non-orphan drugs (median, 712.5 days [IQR, 186.0-1448.3] vs. 387.0 days [92.8-1096.0], P = 0.023). External collaboration in drug development was associated with a longer submission lag (coefficient = 762.1, P = 0.017), while breakthrough therapy designation in the US was associated with a shorter submission lag (coefficient = -832.8, P = 0.035). In conclusion, we revealed that an approval lag for orphan anticancer drugs still existed in 2016-2017. A submission lag for orphan anticancer drugs was the main component affecting the approval lag, and was longer than that for non-orphan drugs. External collaboration in drug development may be a potential factor in reducing the submission lag for orphan anticancer drugs.