Concerns About Provenge Simmer as CMS Ponders Coverage
Authors: M. Goozner
Journal: JNCI Journal of the National Cancer Institute (2011)

A Medicare advisory committee in mid-November expressed mild support for the evidence behind the use of sipuleucel-T (Provenge), approved by the U.S. Food and Drug Administration for asymptomatic, metastatic, castration (hormone)-resistant prostate cancer. The 14-member Medicare Evidence Development and Coverage Advisory Committee rated the quality of evidence supporting the vaccine at 3.6 of a possible 5 for its FDA on-label use and at 1.2 of 5 for off-label use. But in an unusual move, committee members also suggested that the Centers for Medicare and Medicaid Services (CMS) collect postapproval outcomes data through its coverage-with-evidence-development program. That could help physicians and patients identify which patients are most likely to benefi t from the therapy. Medicare has previously used registries to track use of procedures such as imaging, but never drugs. An expanded registry for sipuleucel-T could monitor outcomes such as mortality, time to progression, and side effects. “Many members of the committee felt strongly there should be additional information collected either through clinical trials or through registries,” said committee member Robert Stein brook, M.D., a professor at Dartmouth and a physician at the White River Junction VA Medical Center, Vermont. The suggestion highlighted the questions that continue to hover over sipuleucel-T. The fi rst therapeutic cancer vaccine to reach the market, it’s made through a process called leukapheresis, in which white blood cells are removed from an individual patient, activated with a recombinant fusion protein made from prostatic acid phosphatase and granulocyte – macrophagecolony-stimulating factor, and then infused back into that patient. Researchers theorize that the vaccine stimulates the immune system to generate cancer-fi ghting antibodies. CMS’s decision to conduct a national coverage analysis, unusual for a cancer drug, was based on requests from regional Medicare carriers shortly after the FDA approved the new treatment.The analysis will determine whether CMS will cover the drug; a notice of its proposed ruling is due March 30 and a fi nal ruling by June 30. Meanwhile, 14 of 15 regional Medicare payment contractors now cover sipuleucel-T for on-label use, as do many private payers, including CIGNA, HealthNet, UnitedHealthcare, and Wellpoint, according to sipuleucel-T manufacturer Dendreon’s most recent Securities and Exchange Commission fi ling. One source of uneasiness for many is the cost of the vaccine (although neither FDA nor CMS can consider cost in their decisions). Dendreon announced the $93,000 price tag at the time of FDA approval last April, setting a record for prostate cancer treatments. Even clinicians who participated in the clinical trials are divided over its value. Daniel Petrylak, M.D., a professor of medicine at Columbia University who spoke in favor of the treatment at the CMS meeting, called the price “not an order of magnitude greater than [that of] drugs that are out there now.” Five cycles of one common chemotherapy regimen costs about $80,000, he said. But Michael Neuss, M.D., past chair of clinical practice for the American Society of Clinical Oncology — whose Cincinnati practice, Oncology Hematology Care, also received reimbursement for recruiting patients for Dendreon’s trials — pointed out that most patients who received sipuleucel-T eventually received chemotherapy as well. “The problem is that you wind up paying for more treatment later,” he said.