Global Regulatory Science, Gifu Pharmaceutical University, Gifu, Japan.
Department of Regulatory Affairs, Pfizer R&D Japan G.K, Tokyo, Japan.
J Clin Pharm Ther. 2022 Sep;47(9):1395-1401. doi: 10.1111/jcpt.13677. Epub 2022 Apr 30.
Regulatory authorities in several regions have introduced a number of expedited programs (EPs) to promote the development of innovative drugs for patients in their own countries. The EPs in the United States (US), alone or in combination, have been successful in shortening the clinical development time in the US. We examined whether US-EPs, as well as other related factors, have an impact on the clinical development time in Japan to obtain new insights for more efficient drug development.
In total, 168 drugs approved as new molecular entities (NMEs) in Japan and approved in the US between 2012 and 2019 were surveyed. We compared the clinical development time in Japan for those drugs with or without US-EPs. We also examined the impact of overlapping designations of US-EPs on clinical development time in Japan. Multiple regression analysis was performed to identify associated factors related to clinical development time in Japan, including US-EPs.
The clinical development time in Japan was significantly shorter at 37.4 [Interquartile range, IQR, 28.7-48.9] months for Accelerated Approval (AA), 42.2 [30.0-53.6] months for Breakthrough Therapy (BT), 42.3 [29.3-56.4] months for Fast Track (FT), 44.5 [30.7-60.0] months for US Priority Review, and 45.2 [31.3-61.8] months for US Orphan Designation. Multiple regression analysis revealed that AA (p = 0.008), FT (p = 0.013), Japan Priority Review, and the difference in development initiation dates between the US and Japan were significant factors related to a decrease in the clinical development time in Japan, whereas Japan Orphan Designation and the development of anticancer drugs were significant factors linked to an increase in the clinical development time.
US-EPs were associated with a decrease in the clinical development time in Japan for the drugs that were approved as NMEs in Japan and approved in the US. This association was not restricted to particular therapeutic areas or development strategies. Stakeholders involved in drug development, including the drug developers and regulatory authorities in Japan, should realize these effects for efficient drug development.
几个地区的监管机构已经推出了多项加速计划（EPs），以促进本国患者创新药物的发展。美国（美国）的 EPs 单独或联合使用，成功缩短了美国的临床开发时间。我们研究了美国 EPs 以及其他相关因素是否对日本的临床开发时间产生影响，以期为更有效的药物开发提供新的见解。
共调查了 2012 年至 2019 年在日本批准为新分子实体（NME）并在美国批准的 168 种药物。我们比较了这些药物在日本有无美国 EPs 的临床开发时间。我们还研究了美国 EPs 的重叠指定对日本临床开发时间的影响。进行了多变量回归分析，以确定与日本临床开发时间相关的相关因素，包括美国 EPs。
在加速审批（AA）、突破性治疗（BT）、快速通道（FT）、美国优先审查和美国孤儿药指定的情况下，日本的临床开发时间分别为 37.4 [四分位距（IQR），28.7-48.9]个月、42.2 [30.0-53.6]个月、42.3 [29.3-56.4]个月、44.5 [30.7-60.0]个月和 45.2 [31.3-61.8]个月，显著缩短。多变量回归分析显示，AA（p=0.008）、FT（p=0.013）、日本优先审查以及美国和日本开发启动日期之间的差异是与日本临床开发时间减少相关的重要因素，而日本孤儿药指定和抗癌药物的开发是与临床开发时间延长相关的重要因素。
美国 EPs 与在日本被批准为 NME 并在美国获得批准的药物在日本的临床开发时间缩短有关。这种关联不限于特定的治疗领域或开发策略。药物开发的利益相关者，包括药物开发商和日本监管机构，应该意识到这些对于高效药物开发的影响。
