Economic analysis in phase III clinical cancer trials

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 data on new agents in order to approve pharmaceuticals for widespread use, and clinical trials have been viewed as the proper setting to evaluate these outcomes for new agents. With the recent approval of new pharmaceutical agents that have high costs, early assessments of economic benefit have taken on larger importance to physicians and patients. The integration of economic and clinical analysis in phase III clinical trials raises methodological and practical issues related to study design, 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 products.