LAUSR

OBJECTIVE For approved expedited program and orphan drugs, the safety populations used for premarket drug risk assessments are often limited in size. The study's objective was to determine whether large safety populations, with the capacity for excluding uncommon adverse drug effects, influenced approval of these drugs for patients use. STUDY DESIGN AND SETTING The study included expedited program and orphan drugs for which the Food and Drug Administration (FDA) reviewed the drug applications for approval decisions from 2007 to 2014. From the series of 100 fast track and orphan drugs, approved or non-approved drug applications were compared. RESULTS Among the fast track and orphan drugs, large safety populations having 1,500 or more subjects were associated with increased odds of drug approval (adjusted odds ratio, OR, 10.75; 95% CI, 1.52-133.43). Only a third of the approved drugs had large safety populations. CONCLUSION Approved expedited and orphan drugs are more likely than the non-approved to have been assessed using large safety populations.