February, 2003
© 2000, 2001, 2003 Scott S. Emerson, M.D., Ph.D.
Phase II: Preliminary
evidence of efficacy
Goals:
·Screening
trial to look for any evidence of treatment efficacy
·Incidence of
major adverse effects
·Decide if
treatment is worth studying in larger samples
Methods
·Relatively
small number of participants
·Participants
closer to true target population
·Outcome
often a surrogate
·Sometimes no
comparison group (especially in cancer)