ECONOMIC-ANALYSES OF PHASE-III COOPERATIVE CANCER GROUP CLINICAL-TRIALS - ARE THEY FEASIBLE

Both economic and clinical evaluations of new pharmaceutical agents ar e important to physicians who practice in the current health care envi ronment. While cooperative cancer groups carry out large-scale phase I II clinical evaluations of these agents, few cooperative group studies incorporate economic analyses because of concerns over overburdening of data management, investigators, and statistical center personnel. I n this study, we describe the results and operational considerations o f one of the first completed economic analyses of a phase III cooperat ive group trial of the Eastern Cooperative Oncology Group (ECOG). We d eveloped an economic model estimating economic benefits of yeast-deriv ed granulocyte-macrophage colony-stimulating factor (GM-CSF) as adjunc t therapy for adult patients (56-70 years) with acute myelogenous leuk emia. Clinical data were based on prospectively collected information from a recently reported double-blind phase III multi-institutional st udy carried out by ECOG. Retrospective economic data were obtained fro m financial information systems at our hospital, one of the study site s. The cost-minimization analyses were based on the perspective of a t hird-party payer. Indirect costs related to loss of earnings by patien ts and caregivers as well as quality-of-life adjustments were not inco rporated into the model. Clinical trial results indicated that patient s treated with GM-CSF had shorter times to recovery of absolute neutro phil count of 500 cells/mm(3) and 1000 cells/mm(3) and fewer serious i nfections than patients who received placebo following induction chemo therapy, while no significant differences were noted in red blood cell and platelet transfusion dependency, toxicities, and duration of hosp italization. The economic model estimated that the group treated with GM-CSF was estimated to have lower costs of care, associated with lowe r frequencies of serious infections and lower overall infection-relate d costs. Sensitivity analyses indicated that these results held true o ver a wide range of estimates of costs and infection rates. Prospectiv e economic analyses of phase III cooperative cancer group clinical tri als have not been completed to date. Strategies that are not likely to overburden data managers and statistical center personnel are possibl e to devise. However, these studies require careful planning and coord ination between clinical trialists, economists, and health services re searchers.