A statistical framework is presented for examining cost and effect dat
a on competing interventions obtained from an RCT or from an observati
onal study. Parameters of the joint distribution of costs and effects
or a regression function linking costs and effects are used to define
cost-effectiveness (c-e) measures. Several new c-e measures are propos
ed that utilize the linkage between costs and effects on the patient l
evel. These measures reflect perspectives that are different from thos
e of the commonly used measures, such as the ratio of expected cost to
expected effect, and they can lead to different relative rankings of
the interventions. The cost-effectiveness of interventions are assesse
d statistically in a two stage procedure that first eliminates clearly
inferior interventions. Members of the remaining admissible set are t
hen rank ordered according to a c-e preference measure. Statistical te
chniques, particularly in the multivariate normal case, are given for
several commonly used c-e measures. These techniques provide methods f
or obtaining confidence intervals, for testing the hypothesis of admis
sibility and for the equality of interventions, and for ranking interv
entions. The ideas are illustrated for a hypothetical clinical trial o
f antipsychotic agents for community-based persons with mental illness
. (C) Elsevier Science Inc.