GUSTO cost-effectiveness analysis sets new standard for integrating cost in clinical trials -- NEJM editorial.

GUSTO COST-EFFECTIVENESS ANALYSIS SETS NEW STANDARD FOR CLINICAL TRIALS to include cost outcomes in drug development, Thomas Lee, MD, Brigham & Women's Hospital, asserted in an editorial accompanying an article on the cost of the thrombolytic agent in the May 25 issue of the New England Journal of Medicine. "In important ways, this article sets a new standard for the integration of cost-effectiveness research into randomized trials," Lee maintained.

Daniel Mark, Duke University, et al., report in the NEJM that the cost of Genentech's Activase (alteplase or tPA) per year of life saved is $32,678 based on a cost-effectiveness analysis from the Global Utilization of Streptokinase and Tissue Plasminogen Activator for Occluded Coronary Arteries (GUSTO) study.

The cost-effectiveness analysis used a price range for Activase of $2,216 to $2,750 and $270 to $320 for streptokinase. The incremental cost of annual tPA therapy, $2,845 per patient, was weighed against an increase in life expectancy of .14 year for each patient receiving tPA based on an absolute 1% mortality difference between accelerated tPA and SK in the 41,021-patient study. The analysis also included the cost of hospitalization, procedures and physician visits.

"The cost effectiveness of treatment with accelerated tPA rather than streptokinase compares favorably with that of other therapies whose added medical benefit for dollars spent is judged by society to be worthwhile," the authors concluded. They compared the incremental cost of tPA to the cost of dialysis for chronic renal failure patients and estimated that treating all 250,000 eligible patients who have heart attacks annually with tPA would add $500 mil. to health care costs but would also save 3.5 mil. years of patient life after myocardial infarction.

In his editorial, Lee noted that "cost effectiveness is a continuous, not a yes-or-no, variable" that must be measured against other life-saving treatments. "Calling a therapy cost effective implies that there is agreement on the frame of reference."

Lee added that cost effectiveness should "focus on strategies, not drugs," noting that "the cost effectiveness of tPA depends on how the drug is administered and to whom it is given." Accelerated tPA is better than standard SK but "a faster tPA regimen now under investigation -- the so-called double-bolus approach -- may prove to be even more effective and therefore more cost effective," Lee said. The GUSTO results were first reported in April 1993 and the accelerated regimen of tPA was approved in April of this year ("The Pink Sheet" April 10, T&G-3).