This study focused on 233 drugs with orphan drug designations approved in the US between April 2001 and March 2021. Univariable and multivariable logistic regression analyses were conducted to investigate the association between the presence or absence of RCTs in the clinical data package for new drug applications.
Thursday, June 8, 2023
Randomized controlled trial data for successful new drug application for rare diseases in the United States
Kubota, Y., Narukawa, M. Randomized controlled trial data for successful new drug application for rare diseases in the United States. Orphanet J Rare Dis 18, 89 (2023). doi:10.1186/s13023-023-02702-9
