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

Background: 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. Methods: This study focused on 130 drugs with orphan drug designation approved in Japan between April 2004 and March 2020. Results: 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. Conclusions: Our findings highlight the importance of adequate understanding of the target disease in new drug development for rare diseases. [ABSTRACT FROM AUTHOR]