February, 2003
© 2000, 2001, 2003 Scott S. Emerson, M.D., Ph.D.
Phase III:
Establishment of efficacy
Goals:
·Obtain
measure of treatment’s efficacy on disease process
·Incidence of
major adverse effects
·Therapeutic
index
·Modify
clinical practice (obtain regulatory approval)
Methods
·Relatively
large number of participants from true target population
(almost)
·Clinically
relevant outcome