Randomized Controlled Trial Data for New Drug Application for Rare Diseases in Japan.

Abstract

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.