Can Medicare afford the next generation of marginally effective cancer drugs, whose price tags are approaching $100,000 a year?
That issue wasn’t on the agenda last week when an outside advisory panel told the Center for Medicare and Medicaid Services that the medical evidence was strong enough to justify the agency paying for the prostate cancer drug Provenge. But it was clearly on the advisers’ minds since paying for men eligible for the drug could wind up costing the agency $2 billion a year.
Several commented near the end of the meeting that Medicare should begin measuring how well the $93,000 immune system enhancer treatment actually works – just in case its real world efficacy is less than what has been proven so far through clinical trials run by the manufacturer. The evidence submitted to Food and Drug Administration by Seattle-based Dendreon Corp. showed sipuleucel-T (that is Provenge’s generic name) extended the average lifespan of people with metastatic prostate cancer, which is currently about two years, by an average of four months.
Medicare has the power to order companies to collect outcomes data after a therapeutic approach is approved for reimbursement. The so-called “coverage with evidence development” (CED) program was initiated by Mark McClellan, who ran the agency in the mid-2000s under President George W. Bush.
But the 10 CEDs that CMS has ordered to date haven’t involved the efficacy of FDA-approved drugs (they’ve mostly involved imaging, devices and genetic testing). Starting it off with Provenge is sure to provoke a firestorm of opposition.
Some background. Provenge has been the most controversial new cancer drug to hit the market since Imclone System’s Erbitux landed Martha Stewart in jail. Patient advocates, many of whom owned stock in the start-up company, engaged in a massive lobbying campaign to win FDA approval for the drug after it was initially turned down for lack of evidence. The drug was approved on its second-go-round earlier this year.
Advocates are convinced the process – it’s not really a drug, but involves removing a patient’s white blood cells, enhancing them with cancer-fighting antigens, and injecting them back into the patient’s body in a series of injections – is a god-send for the mostly elderly prostate cancer patients whose tumors have metastasized and are resistant to chemical castration drugs. While it may increase stroke risk, Provenge has none of the nasty side effects associated with the other drugs.
Despite last week’s press accounts suggesting the advisory committee vote amounted “to a recommendation that Medicare pay for Provenge,” the evidence reviews on its performance were mixed. A technology assessment provided CMS by the Agency for Healthcare Research and Quality suggested Provenge’s benefits for prostate cancer patients were “modest.” The National Comprehensive Cancer Network clinical practice guidelines, which can be used by physicians to claim reimbursement for the off-label use of drugs, endorsed Provenge only for its on-label use and even then said it was “not recommended for patients with a life expectancy of six months.”
The review by the Medicare Evidence Development and Coverage Advisory Committee last week wasn’t much better. The committee gave it a 3.6 out of 5 rating for its on-label use and just 1.2 out of 5 for its off-label use, which would make it available to many more of the 200,000 men diagnosed with prostate cancer each year. Only about 20,000 of that group have tumors resistant to chemical castration drugs – the on-label indication.
When used on-label, Provenge is already being paid for by several insurers and most regional Medicare carriers. Medicare patients pick up 20 percent of the cost – about $18,600 a year.
“The base of evidence upon which this therapy rests is of moderate strength; it’s not really wide, it’s not really deep,” said committee chairman Clifford Goodman, a senior vice president at the Lewin Group, which does consulting for both insurance companies and health care providers. “Much work is needed to help collect more data to make it broader and deeper.”
Committee member Robert Steinbrook, a professor at Dartmouth and a clinician at the White River Junction Veterans Administration hospital, echoed that sentiment in a telephone interview. “Many members of the committee felt strongly there should be additional information collected either through clinical trials or through registries,” he said.
The FDA has already ordered Dendreon to construct a 1,500-person outcomes registry to see if it increases stroke risk, which was hinted at in the three clinical trials submitted to the agency. Expanding that registry to include all patients given the treatment with Medicare funding would be far more costly for the company, and require tracking mortality and quality-of-life outcomes, not just stroke data.
That’s not something manufacturers like to do once they have FDA approval since there’s no upside. The best that can happen is that the registry ratifies the FDA’s decision. The downside is a narrowing of its use, or possibly even a decision by Medicare to discontinue reimbursement if patients on Provenge in the real world fare worse than those given other therapies.
A Dendreon spokeswoman would not comment when asked if the company would support constructing a comprehensive outcomes registry. Yet with $2 billion a year in the offing, it’s the least it can do. The tens of thousands of elderly men who face this life-threatening illness every year deserve to know if their and the taxpayers’ money is being wasted.
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