MCNA didn't fail...the design of the trial did. Nearly all panelists agreed that MCNA appeared safe and well tolerated, which for some offered a sufficient reason to provide an alternative treatment in NMIBC, a disease that has not seen a new therapy approved in nearly three decades. The most widely used drug, BCG, has its problems. As a live, attenuated strain of M. bovis, it cannot be administered immediately postop and it's been plagued by drug shortage issues in recent years. Meanwhile, a substantial percentage of patients – estimates put the figure at roughly 40 percent – relapse on or are refractory to BCG. For those patients, often the only option is cystectomy, or bladder removal which, as Monica Smith, of the Bladder Cancer Advocacy Network, noted during the public hearing part of the meeting is a "major life change for patients."
Her comments were echoed by panel member Thomas Griffith, microbiology associate professor and department of urology member at the University of Minnesota, one of the six yes votes. "I think a lot of the deficiencies raised were clearly there, but I felt the weaknesses were outweighed by a number of strengths," particularly the fact that there are few options. "We need something that is not so much of a radical procedure" for patients.
A few panelists said they would vote in favor of MCNA if the label indicated use only for those patients who were not eligible or who refused cystectomy. For Michael Menefee, assistant professor in the division of hematology and oncology at the Mayo Clinic, however, even that modification would elicit a no vote. "I think the problem lies in that the study was not well designed to answer the question . . . with the confidence we would like to have," he said.