For nearly a decade, many men with prostate cancer have followed the slow progress of the U.S. Food and Drug Administration's approval process for Provenge. In April it became the first therapeutic cancer vaccine approved by the FDA after clinical trials showed it added, on average, four months to recipients' life expectancy.
But because of a host of issues surrounding the drug -- including its high cost, relatively short improvement in life expectancy and a limited supply for the next six months -- the Centers for Medicare and Medicaid Services, or CMS, are going to review the drug on Wednesday.
Exactly what CMS intends to review and do is not clear. Will it try to pay less than the $93,000 its Seattle-based maker, Dendreon, is charging for the three needed treatments? Will Medicare restrict who'll get the drug?
With 217,000 new cases of prostate cancer diagnosed annually, and 32,000 men dying from it every year, patients, their families and doctors alike are waiting to see what CMS decides.
"They will set the standard in terms of how much they're willing to pay and who gets to get the drug," said Ralph Miller, a urologist and director of the Allegheny General Hospital Prostate Center. "They have tremendous power in this."
What CMS decides will likely affect what private insurers decide to pay and whom they approve to take Provenge, too. Several Pittsburgh-area doctors said they weren't aware of any private insurers that excluded coverage because of the drug's cost nor of any eligible patient not getting it because of shortages. Dendreon said it could only produce enough doses for 2,000 patients in the first year.
Provenge works by boosting the immune system to fight prostate cancer, rather than attacking the cancer itself. The immune-boosting approach has long been theorized, and with Provenge's approval it is touted as just the first of a new line of cancer drugs.
For each of three treatments, blood is drawn, the white blood cells are separated out and then incubated with a protein and an immune system booster, and then the treated cells are transfused back into the patient.
But the drug still only extends life by an average of four months -- although a third of men who took it in trials were still alive three years later -- and it isn't the long-dreamed of cure that researchers are working toward.
"I think of it as evolutionary, as opposed to revolutionary," Miller said. "But this is unique, and it is a legitimately hopeful thing."
Like many drugs, though, it is problematic, said Joel B. Nelson, head of the University of Pittsburgh Medical Center's urology department.
"There is no way to assess response and no way to know necessarily who will benefit and who won't," he said. "So, from a drug company point of view, it's the perfect drug because you have to give it" to whoever is medically eligible.
John Lech, a urologist with West Penn Allegheny Health System, was part of the clinical trials for Provenge. His practice was among the first in the country to have access to the drug. "This is not a cure," he said, "but compared to chemo, it's a heck of a lot better."
For most men, Provenge has almost no side effects, unlike those associated with chemotherapy: nausea, hair loss, fatigue.
Prostate cancer patients have been asking their doctors about the drug nonstop since its approval.
"Some guys with bone pain, which makes them too weak from the cancer, that tends to exclude people," Lech said. "And this is not for everyone with prostate cancer, (but) only those with advanced disease. And that's my job: to weed out who is and who isn't eligible. That's the hard part."