Boulevard of Broken Dreams: Drug Approval for Older Adults With Acute Myeloid Leukemia

weeks) plus supportive care or supportive care alone; almost all patients randomly assigned to the control arm (88%) received LDAC. The primary end point was overall survival. With 485 patients enrolled, the hazard ratio for death for decitabine therapy compared with that for control therapy at the time of the preplanned end point was nonsignificant (0.85; 95% CI, 0.69 to 1.04; P .11), although the complete remission rate in the decitabine arm was more than double that in the control arm (16% v 7%), and the observed median overall survival time was numerically higher in the decitabine arm (7.7 v 5.0 months). Why did decitabine join the ranks of tipifarnib (Zarnestra; Johnson Genzyme, Cambridge, MA), and laromustine (Onrigin; Vion Pharmaceuticals, New Haven, CT) in failing to convince an FDA panel of its favorable efficacy and safety profile in a population of older and frailer patients with AML who so clearly need additional therapies? There were, of course, factors specific to the negative decision for each of these drugs.