HOECHST-ROUSSEL’s GM-CSF COST-BENEFIT ANALYSIS BY BATTELLE

HOECHST-ROUSSEL's GM-CSF COST-BENEFIT ANALYSIS BY BATTELLE will be submitted to FDA in support of Hoechst's pending PLA. The company said it has received preliminary results from a retrospective cost-effectiveness study by the Seattle-based Battelle research and consulting firm in patients receiving GM-CSF therapy after bone marrow transplants. The use of GM-CSF to enhance recovery from bone marrow transplantation had a "major impact" on hospital length of stay and led to a "major savings" overall, Hoechst said. In addition to duration of hospital stay and room charges, the retrospective analysis looked at reduction in antimicrobial agents, I.V. solutions, and professional fees or physicians services. Hoechst did not conduct a cost-benefit analysis of GM-CSF treatment of patients with engraftment failure because of the complexity of analyzing the 38 centers that were involved. Hoechst and co-developer Immunex have been granted a Treatment IND for the engraftment indication ("The Pink Sheet" Oct. 1, T&G-6), Hoechst is also planning prospective cost-effectiveness studies to run concurrently with its Phase III trials in patients receiving chemotherapy without bone marrow transplantation. One of the first cost analyses will be of breast cancer patients treated with high doses of chemotherapy and GM-CSF compared with patients receiving regular doses. Study endpoints will include time to return to work and frequency of hospitalizations. The study will also look at quality of life, although those specific endpoints have yet to be determined. At an American Medical Association seminar on colony stimulating factors funded by Hoechst-Roussel, Stephanie Williams, MD, University of Chicago Medical Center, said that "G-CSF or GM-CSF may allow us to successfully dose escalate therapy during cycles or treatment for diseases such as small lung cancer, lymphomas, bladder cancer and in particular, breast cancer." More intensive chemotherapy could lead to "higher response rates in patients with these malignancies as well as decrease the toxicity associated with these treatments," she noted, adding that "it may mean less supportive care and less cost for supportive care such as hospitalization, antibiotic therapy and blood product transfusion." However, there may be cost increases in other areas. "One of the major possibilities that is offered through the use of CSFs is the possibility of giving more doses of intensive therapy," said James O'Reilly, M.D., Memorial Sloan-Kettering Cancer Center. But "as you go for the cure, unfortunately, if increased dose intensity is correct, then it is going to increase that [hospital] cost." O'Reilly noted that if "insurance companies are looking at this from the context of a cost of hospitalization," while time in the hospital will be shortened, "your cost per diem is going to be higher." In the area of bone transplantation -- the current focus of PLAs filed by Immunex (Prokine) and Schering/Sandoz (Leucomax) -- "colony stimulating factors in this setting may well decrease the time in hospital, as some studies have suggested," Williams noted. At the University of Chicago Medical Center "the average cost of a patient undergoing an autologous bone marrow transplant is $150,000 for an average five-week stay in the hospital." Williams pointed out that studies by John Nemunaitis, MD, Fred Hutchinson Cancer Research Center, who has conducted some of the Hoechst/Immunex trials with GM-CSF, have shown that there is a decrease in the number of days in the hospital by about one week. Schering is reportedly completing an analysis of a prospective cost-effectiveness study of its GM-CSF product. Amgen's Neupogen GM-CSF cost-benefit study, conducted by Washington, D.C.-based consulting group Health Technology Associates, in chemotherapy-induced neutropenia is also near completion. The researchers are planning to publish the results in a major medical journal within six to 12 months.