Recombinant granulocyte-macrophage colony-stimulating factor (rGM-CSF): an appraisal of its pharmacoeconomic status in neutropenia associated with chemotherapy and autologous bone marrow transplant.

Recombinant granulocyte-macrophage colony-stimulating factor (rGM-CSF) expedites neutrophil recovery in cancer patients receiving chemotherapy with or without autologous bone marrow transplant (ABMT). The limited cost analyses available in patients undergoing ABMT support a cost reduction of about 25 to 35% with rGM-CSF therapy, relative to placebo, generated primarily by decreases of 20 to 30% in hospitalisation costs reflecting reductions in length of hospitalisation. Results of 1 trial show equivalent cost savings of 40% versus placebo with either rGM-CSF or recombinant granulocyte colony-stimulating factor (rG-CSF) in patients with chemotherapy-induced febrile neutropenia. Whether reduced infection rates seen with rGM-CSF may lessen costs of antimicrobial therapy is undetermined; however, a 16% decrease in this cost factor was reported in 1 evaluation of high dose chemotherapy with ABMT. No analyses have assessed the cost effectiveness of rGM-CSF as prophylaxis in patients receiving chemotherapy. Survival rates have increased in patients treated with rGM-CSF after bone marrow graft failure. In contrast, with the exception of one small trial, improvements in mortality or relapse rates have not occurred with rGM-CSF used prophylactically with chemotherapy, despite favourable effects on neutrophil recovery and facilitation of dose-intensified chemotherapy regimens. Similarly, survival has not increased in patients undergoing ABMT. The long term economic impact of rGM-CSF in these indications is thus unknown. Other factors predicted to produce modest cost savings include possible reductions in expenditure related to treating mucositis, and lowered transfusion requirements in some patients. Whether rGM-CSF may provide benefits in other areas that can be expressed in economic terms, such as quality of life, also remains to be established. On the whole, rGM-CSF has a good tolerability profile, obviating the need for costly monitoring procedures. Like other expensive biotechnology products, its cost effectiveness will be aided by implementation of appropriate prescribing techniques and protocols to minimise wastage. Thus, at present rGM-CSF therapy appears to offer a means of reducing hospitalisation costs, and therefore a substantial component of treatment expenditure, in patients undergoing ABMT or with chemotherapy-induced febrile neutropenia.