GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR AS ADJUNCT THERAPY IN RELAPSED LYMPHOID MALIGNANCY - IMPLICATIONS FOR ECONOMIC-ANALYSES OFPHASE-III CLINICAL-TRIALS

With the increasing concern over the high cost of health care, policy makers have incorporated economic analyses into phase III clinical tri als as the randomized clinical trials can provide important informatio n on the efficacy and potential cost-effectiveness of new pharmaceutic al agents. Economic analyses of single-hospital experience during phas e III trials of granulocyte-macrophage colony-stimulating factor (GM-C SF) as adjunct therapy for high dose chemotherapy with autologous stem cell support found significant shortening of neutropenia with GM-CSF at each hospital, but shortened hospitalization (and lower costs) at o nly two of three hospitals. In this study, we added data from three ad ditional hospitals and found that the 103 patients who received GM-CSF had, on average, 5.7 days shorter durations of severe neutropenia tha n the 95 patients who received placebo (p < 0.0001) and 3.4 days short er in hospitalization (p = 0.06). However, the duration of hospitaliza tion, the primary determinant of health care costs, was shorter for GM -CSF patients in only four of the six centers and the duration of hosp italization of placebo patients was shorter at the other two centers. Careful analyses must be carried out when phase III clinical trial res ults are used to derive estimates of cost-effectiveness of new pharmac eutical agents. The interpretation of economic analyses of phase III c linical trials raises issues related to the perspective of the investi gators, study design, collection of data on resource utilization, lear ning curve effects and generalizability of the results to other settin gs.