Kobayashi Kojiro, Inaba Yuina, Hashimoto Mayu, Maeda Hideki
Department of Regulatory Science, Graduate School of Pharmaceutical Science, Meiji Pharmaceutical University, Tokyo, Japan.
Daiichi Sankyo Co. Ltd, Tokyo, Japan.
Ther Innov Regul Sci. 2025 Apr 8. doi: 10.1007/s43441-025-00771-5.
Investigations on the relationship between the designated systems for drug development and regulatory processes in Japan and those in the United States over an extended period of time are limited.
We aimed to comprehensively investigate the special regulatory pathways for approved drugs in Japan focusing on the types of drugs receiving SRPs, the benefits of SRP in terms of review periods, a comparison between Japan and the United States, and the investigation of mutual influences in obtaining SRPs in both countries over 20 years.
All drugs approved in Japan between September 1999 and December 2022, and those that underwent various special regulations based on publicly available information were investigated. A comparison between Japan and the United States was conducted using publicly available information.
In total, 2,309 drugs were approved in Japan between September1999 and December 2022, of which 819 drugs received special regulatory pathways. The number of drugs receiving special regulatory pathways increased annually, from 10 from September to December in 1999 to 90 in 2022. Anticancer drugs accounted for one-third of all drugs. Orphan drugs (432 drugs, 52.7%), expedited reviews (257 drugs, 31.4%), and priority reviews (135 drugs, 16.5%) were the most common regulatory pathways. For drugs receiving special regulatory pathways, 315 (38.5%) were approved within 6 months and 699 (85.3%) were approved within 12 months (the mean review period in Japan). Designations of priority review, expedited review, and orphan drug in Japan mutually influence the designation of these special regulatory pathways. In addition, designation of expedited review and orphan drug in Japan was influenced by designation of breakthrough therapy in the US, and expedited review in Japan was influenced by fast track in the US. In the US, special regulatory pathways are not significantly overlapped each other in the US.
In the drugs approved in Japan, the Japanese specific special regulatory pathways are mutually influenced. Expedited review and orphan drug in Japan are significantly impacted by the specific special regulatory pathways of the US. Additionally, special regulatory pathways of the US did not influence each other.
长期以来，关于日本和美国药物研发指定体系与监管流程之间关系的研究有限。
我们旨在全面调查日本获批药物的特殊监管途径，重点关注获得特殊审评程序（SRP）的药物类型、SRP在审评周期方面的益处、日本与美国之间的比较，以及20年来两国在获得SRP方面的相互影响。
对1999年9月至2022年12月期间在日本获批的所有药物，以及根据公开信息接受各种特殊监管的药物进行调查。利用公开信息对日本和美国进行比较。
1999年9月至2022年12月期间，日本共批准了2309种药物，其中819种药物获得了特殊监管途径。获得特殊监管途径的药物数量逐年增加，从1999年9月至12月的10种增加到2022年的90种。抗癌药物占所有药物的三分之一。孤儿药（432种，52.7%）、加速审评（257种，31.4%）和优先审评（135种，16.5%）是最常见的监管途径。对于获得特殊监管途径的药物，315种（38.5%）在6个月内获批，699种（85.3%）在12个月内获批（日本的平均审评周期）。日本的优先审评、加速审评和孤儿药指定相互影响这些特殊监管途径的指定。此外，日本的加速审评和孤儿药指定受到美国突破性疗法指定的影响，日本的加速审评受到美国快速通道的影响。在美国，特殊监管途径之间没有明显重叠。
在日本获批的药物中，日本特定的特殊监管途径相互影响。日本的加速审评和孤儿药受到美国特定特殊监管途径的显著影响。此外，美国的特殊监管途径之间没有相互影响。
