Randomized phase III clinical trials provide important information on
the efficacy of new pharmaceutical agents for cancer patients. Policy
makers are showing increased interest in both economic and clinical da
ta on new agents in order to approve pharmaceuticals for widespread us
e, and clinical trials have been viewed as the proper setting to evalu
ate these outcomes for new agents. With the recent approval of new pha
rmaceutical agents that have high costs, early assessments of economic
benefit have taken on larger importance to physicians and patients. T
he integration of economic and clinical analysis in phase III clinical
trials raises methodological and practical issues related to study de
sign, collection of data on resource utilization, and generalizability
of data to other settings. In this paper, we review these issues and
discuss their relationship to clinical trials for new pharmaceutical p
roducts.