As the Centers for Medicare & Medicaid Services (CMS) deliberated earlier this year over whether to cover Provenge (sipuleucel-T), an expensive but potentially gamechanging new treatment for prostate cancer, many in healthcare policy, patient advocacy, and oncology circles held their breath. Would the agency reject a cancer treatment approved by the FDA?
In late March, CMS ended the speculation by announcing its intention to pay for onlabel uses of the drug, which costs $93,000 for a 3-dose regimen, while leaving off-label coverage decisions to its regional contractors. More recently, the agency issued a final National Coverage Determination that requires Medicare contractors to cover Provenge’s use in treating asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment.
Despite the outcome, CMS watchers called the controversial review significant, with possible implications for policy decisions down the road.
J. Leonard Lichtenfeld, MD
Is It the Cost?
J. Leonard Lichtenfeld, MD, deputy chief medical officer for the American Cancer Society, described it as “the preliminary round in a heavyweight match-up between Uncle Sam and the pharmaceutical companies.”
“One wonders why they went through the process if it were not about cost,” he said. “From a purely practical point of view, this was a straightforward decision. The drug had shown benefits, had the approval of the FDA, and so it would have flown in the face of decisions the agency had made previously to deny coverage.”
But he noted that the agency had already signaled its sensitivity to costs by refusing to pay for cancer tests.
Indeed, Lichtenfeld said that CMS “threw down the gauntlet” in 2009 in denying coverage of CT colonography for the detection of colorectal cancer. The agency made it clear following that decision, he said, that assessing the value of treatments and their impact on the Medicare budget were within the scope of reviews.
“The agency declared an aggressive public policy intended to put the brakes on approvals, and the decision was written in a way that suggested it was within their purview to ask [for] more details. I thought this time might be the time CMS would exercise that newfound authority,” he said, noting however that he agreed with the decision to cover on-label uses of Provenge.
One wonders why they went through the process if it were not about cost. From a purely practical point of view, this was a straightforward decision. The drug had shown benefits, had the approval of the FDA, and so it would have flown in the face of decisions the agency had made previously to deny coverage. ”
–J. Leonard Lichtenfeld, MD
“But I think we missed an opportunity to take on questions about off-label use,” he added. “I expected CMS to say ‘no’ to off-label use, except as part of clinical trials. That did surprise me.” Lichtenfeld commented on his blog that such an open-ended policy could subject the local carriers to a deluge of men “seeking this treatment for all the wrong reasons: very advanced symptomatic disease all the way to men newly diagnosed with their cancer, or whose cancer has spread but still responds to hormone treatment.”
While the decision ultimately backed existing healthcare policy, it infused uncertainty into the process that is unlikely to dissipate soon, said Allen S. Lichter, MD, chief executive officer of ASCO.
“I don’t know whether we’ll see this again or will get it with every single new cancer agent,” Lichter said, adding that such ambiguities in the approval process pose a threat to both care and research. “If you’re a drug developer, the possibility must be factored in as a risk of making the investment. It injects uncertainty no matter what you do, and this is the specter this process has raised.”
Allen S. Lichter, MD
A Controversial Review
When CMS announced the review last year, ASCO urged the agency to withdraw from it on principle, noting a lack of “clarity regarding the purposes underlying this action” and asserting ASCO’s belief that the Social Security Act requires CMS to cover drugs and biologics for FDA-approved indications used in anticancer chemotherapeutic regimens.