Results
ODD
REGRESSION
A total of 335 drugs with ODD containing new active substances approved in the US between April 1, 2001, and March 31, 2021 were identified. Among these, 233 drugs with ODD were selected for the analysis (Fig. 
Flow diagram of drug selectionThe characteristics of the selected orphan drugs are summarized along with the design of the pivotal trial, RCT, or SAT (Table 
Characteristics of orphan drugsTable 
Results of univariable and multivariable logistic regression analyses*