February, 2003
© 2000, 2001, 2003 Scott S. Emerson, M.D., Ph.D.
Regulatory agencies
(cont.)
Usually require 2 -
3 independent phase III studies
·Concurrent
control group to assess efficacy and rates of common adverse
experiences
·
Usually
require experience treating some minimal number of patients in order to
put upper bounds on rates of serious adverse experiences that
went unobserved
·Rule of 3: If
no events were observed in N patients, the upper 95% confidence
bound is asymptotically 3 / N (4.6 / N for 99% bound)