A cross-sectional study on the first-in-human trials of anticancer drugs in Japan and the United States and the probability of approval.

IF 2.8 3区医学 Q3 ONCOLOGY

International Journal of Clinical Oncology Pub Date : 2025-10-01 Epub Date: 2025-08-06 DOI:10.1007/s10147-025-02849-4

Akari Mukaida, Hideki Maeda

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引用次数: 0

Abstract

Background: This study aimed to examine the characteristics of First-in-human (FIH) trials conducted in Japan and the US and whether the probability of approval for pharmaceuticals that had undergone FIH trials differs in the two countries.

Methods: FIH trials of anticancer drugs initiated between 2007 and 2017 were investigated in this study. The trials were searched using ClinicalTrials.gov.

Results: There were 22 FIH trials conducted in Japan and 261 in the US. Of these, six drugs (27.2%) were approved in Japan and 27 (10.3%) were approved in the US, indicating that the probability of approval was significantly higher for FIH trials conducted in Japan than in the US. Comparison of the characteristics of FIH trials between Japan and the US, showed that 81.8% of the FIH trials conducted in Japan were sponsored by the top 20 pharmaceutical companies, whereas 55.6% in the US were sponsored by non-top 20 companies (P = 0.003, Chi-square test). The number of patients was higher in Japan than in the US (P = 0.044). Further, all of the trials conducted in Japan were multiregional clinical trials in collaboration with other countries such as Europe and the US, whereas 49.0% of the trials in the US were conducted in the US alone (P < 0.001).

Conclusion: We inferred that the FIH trials conducted in Japan are multiregional clinical trials by major pharmaceutical companies with Europe and the US, and are conducted with drugs that are expected to have a high probability of successful approval.

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日本和美国抗癌药物首次人体试验及获批概率的横断面研究。

背景：本研究旨在研究在日本和美国进行的首次人体试验（FIH）的特点，以及在这两个国家进行FIH试验的药物获得批准的概率是否不同。方法：本研究对2007年至2017年间启动的抗癌药物的FIH试验进行调查。使用clinicaltrials .gov检索试验结果：在日本进行了22项FIH试验，在美国进行了261项试验。其中，6种药物（27.2%）在日本获得批准，27种药物（10.3%）在美国获得批准，这表明在日本进行的FIH试验获得批准的概率明显高于美国。日本和美国FIH试验的特征比较显示，日本进行的FIH试验中，有81.8%是由排名前20的制药公司赞助的，而美国有55.6%是由排名前20的制药公司赞助的（P = 0.003，卡方检验）。日本的患者数量高于美国（P = 0.044）。此外，在日本进行的所有试验都是与欧美等其他国家合作进行的多区域临床试验，而在美国进行的试验中有49.0%是单独在美国进行的(P结论：我们推断在日本进行的FIH试验是与欧美主要制药公司进行的多区域临床试验，并且所使用的药物预计有很高的成功批准概率。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

International Journal of Clinical Oncology 医学-肿瘤学

CiteScore

6.80

自引率

3.00%

发文量

175

审稿时长

2 months

期刊介绍： The International Journal of Clinical Oncology (IJCO) welcomes original research papers on all aspects of clinical oncology that report the results of novel and timely investigations. Reports on clinical trials are encouraged. Experimental studies will also be accepted if they have obvious relevance to clinical oncology. Membership in the Japan Society of Clinical Oncology is not a prerequisite for submission to the journal. Papers are received on the understanding that: their contents have not been published in whole or in part elsewhere; that they are subject to peer review by at least two referees and the Editors, and to editorial revision of the language and contents; and that the Editors are responsible for their acceptance, rejection, and order of publication.