February, 2003
© 2000, 2001, 2003 Scott S. Emerson, M.D., Ph.D.
Phase III: Common
scenarios
Establish efficacy
of new treatment
·superiority
over no intervention
·superiority
over existing treatment
Establish
equivalence with current treatment
·Two-sided
equivalence: bioequivalence
·One-sided
equivalence: noninferiority
-perhaps
superior on secondary endpoint
Establish harm of
existing treatment