Kubota Yosuke, Narukawa Mamoru
Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo, 108-8641, Japan.
Development, Astellas Pharma Inc., Tokyo, 103-0023, Japan.
Ther Innov Regul Sci. 2022 Jul;56(4):659-666. doi: 10.1007/s43441-022-00404-1. Epub 2022 Apr 27.
High-quality evidence is often not obtained in the clinical trials of rare diseases because these trials tend to be smaller in size and non-controlled. We investigated the potential factors associated with the need for randomized controlled trials (RCTs) in the clinical data package for new drug applications for rare diseases in Japan.
This study focused on 130 drugs with orphan drug designation approved in Japan between April 2004 and March 2020.
Multivariable regression analysis showed that the prevalence (odds ratio [OR] 3.21, 95% confidence interval [CI] 1.18-8.6) and the type of primary endpoint (OR 6.66, 95% CI 2.41-18.37) were associated with the need for RCTs in the clinical data package in Japan.
Our findings highlight the importance of adequate understanding of the target disease in new drug development for rare diseases.
在罕见病的临床试验中，往往无法获得高质量证据，因为这些试验规模通常较小且无对照。我们调查了与日本罕见病新药申请临床数据包中随机对照试验（RCT）需求相关的潜在因素。
本研究聚焦于2004年4月至2020年3月期间在日本获批的130种孤儿药指定药物。
多变量回归分析显示，患病率（优势比[OR] 3.21，95%置信区间[CI] 1.18 - 8.6）和主要终点类型（OR 6.66，95% CI 2.41 - 18.37）与日本临床数据包中RCT的需求相关。
我们的研究结果凸显了在罕见病新药研发中充分了解目标疾病的重要性。
