Medicare, with little public debate, has expanded its coverage of drugs for cancer treatments not approved by the Food and Drug Administration.
Cancer doctors had clamored for the changes, saying that some of these treatments, known as off-label uses, were essential if patients were to receive the most up-to-date care. But for many such uses there is scant clinical evidence that the drugs are effective, despite costing as much as $10,000 a month. Because the drugs may represent a patient’s last hope, though, doctors are often willing to try them.
The new Medicare rules are the latest twist in a protracted debate over federal spending on off-label drugs — drugs prescribed for uses other than those for which they have been specifically approved.
Proponents of the changes say such spending not only helps patients, but can also enhance medical understanding of which treatments work against various forms of cancer.
But opponents argue that the new approach may waste money and needlessly expose patients to the side effects of drugs that may not help them. They also raise the possibility of conflicts of interest, because the rules rely on reference guides that in some cases are linked to drug makers.
The new policy, which took effect in November, makes it much easier to get even questionable treatments paid for, critics of the changes say. Medicare is providing “carte blanche in treatment for cancers,” said Steven Findlay, a health policy analyst for Consumers Union. He said overly expansive coverage encourages doctors to use patients as guinea pigs for unproved therapies.
Because Medicare officials canceled a cost analysis of the changes, it is hard to predict how much spending will increase beyond the $2.4 billion Medicare paid in 2007 for cancer drugs. But cancer doctors and other experts say the new policies, adopted in the final months of the Bush administration, seem almost certain to raise the federal drug bill, while making it more difficult for the new administration to rein in spending on unproven medical treatments.
Although President Obama has made a goal of controlling health care costs, a spokesman for the Obama administration declined to comment on the Medicare changes.
One of the many drugs whose use is likely to expand is the Eli Lilly product Gemzar, which costs $2,500 to $5,000 a month. The F.D.A. has approved it to treat only four types of cancer. But the new rules will virtually guarantee that Medicare will pay for its use for about a dozen other cancers, including advanced cervical cancer — even though the evidence supporting Gemzar for that use is “inconclusive,” according to one of the reference guides Medicare will now be consulting.
In the case of Genentech’s Avastin, one of the world’s most expensive and widely used cancer drugs, Medicare rejected in 2007 nearly all of the estimated $16 million in requests from doctors’ offices to cover its off-label use for ovarian cancer, according to claims specialists who work with Medicare data but declined to be identified because of the controversy over the topic. Under the new rules, Avastin will be routinely covered for ovarian cancer — as will at least some other off-label uses, including for brain and kidney cancer.
It is unclear how much precedent Medicare’s new rules might have on private insurers, which often follow the agency’s lead on paying for drugs.
Medicare officials defend the new policies, saying they respond to cancer doctors’ concerns that the agency has been too slow to recognize promising new off-label treatments. Dr. Steve Phurrough, who has overseen coverage for the agency since 2003, noted that a 1993 federal law gave Medicare specific authorization to cover some unapproved uses of cancer drugs.
“Congress wanted a lesser level of evidence,” Dr. Phurrough said. The question of what is adequate evidence is “not a line in the sand,” he said. “It’s a broad stripe in the sand.”
The American Society of Clinical Oncology, which represents cancer doctors, has hailed the new rules, saying they will ensure that the appropriate off-label uses are covered.
But some specialists say that being able to offer off-label drugs can also let physicians avoid hard discussions with patients about a grim prognosis.
“It makes it easier to give drug after drug,” said Dr. Andrew Berchuck, director of gynecologic oncology at Duke University, “and keep the fantasy alive.”
The new rules expand the number of reference guides — or compendiums — that Medicare relies on for determining which off-label uses of cancer drugs to cover. The writers and editors of these compendiums, who work completely outside the federal government, scan the medical literature and evaluate the evidence in making their recommendations.
In 1993, Congress had authorized three compendiums for Medicare, all published by not-for-profit organizations. But by 2007 two had stopped publishing, leaving Medicare with a single compendium. Having selected three additional guides last year, the agency plans to review its choice of guides every year.
Under the old rules, Medicare representatives were supposed to consult the compendiums but also use their own discretion in interpreting the guides’ recommendations. The new rules essentially delegate the decision to guides Medicare has selected, even when there is little clinical evidence behind a particular recommendation. As long as at least one of them recommends a cancer treatment, Medicare is essentially obliged to pay for it — unless one of the other guides specifically advises against it.
And some of these new compendiums have close financial ties to the drug industry, according to the draft of a report Medicare commissioned last year after Congress raised questions about possible conflicts of interest. The draft was completed in October, with a final version to be released soon.
The draft criticizes the new rules for essentially taking most decisions about off-label cancer drugs out of Medicare’s hands, even when the agency is aware of potential conflicts. The guide’s recommendation, the report says, “becomes the final word.”
For some experts, the bigger concern about using some cancer drugs off-label without adequate evidence is that they may not only be useless — they may cause dangerous side effects.
“We have very little faith that those indications that make it into the compendia are safe, let alone effective,” said Dr. Allan M. Korn, the chief medical officer for the Blue Cross and Blue Shield Association, who added that Medicare should cover off-label drugs only if the results of their use are carefully tracked afterward. There is no such requirement in the new Medicare guidelines.
There have been three different top Medicare administrators since the off-label rule changes were set in motion a few years ago. The second of them, Leslie V. Norwalk, chose to select the compendiums through a streamlined and internal administrative process, instead of the more elaborate and public process that Medicare often uses for broad coverage decisions.
“I did not see it as a significant step in coverage,” said Ms. Norwalk, who left Medicare in 2007.
Drug makers say they welcome the Medicare changes. A spokesman for the Pharmaceutical Research and Manufacturers of America, the industry’s main trade group, said the new rules ensured “that cancer patients have access to the treatments they need.”
Many oncologists say they needed greater flexibility in using cancer drugs because it can take months or years for a new use to be approved by the F.D.A. They cite the example of Celgene’s drug thalidomide, now a mainstay treatment for multiple myeloma, which was prescribed only off-label for years before the F.D.A. formally approved it for that use.
And in the case of rare types of cancer, there may be so few potential patients that companies have little financial incentive to undergo the formal F.D.A. process for approving a drug for expanded use. Only two drugs have been approved by the F.D.A. for brain cancer, for example, and cancer doctors say they need the ability to try other drugs or other combinations of treatments.
“To arbitrarily stop after two drugs to me is ludicrous,” especially for younger patients, said Dr. Virginia Stark-Vance, a solo practitioner in Dallas and Fort Worth. She said one of her brain cancer patients had been kept alive for 10 years by off-label use of irinotecan, a colon cancer drug that was the ninth drug the patient tried.
Medicare seems to have ignored some concerns raised by a group of outside researchers whom the agency had asked to survey a half-dozen compendiums, including the four that Medicare has now adopted. That report, completed in 2007, found that the six guides “cited very little of the available evidence,” said Dr. Amy P. Abernethy, a Duke oncologist who led the study.
The study also found great variability among the guides, in terms of what uses were recommended — or discussed at all.
Despite her study’s findings, Dr. Abernethy says she does not oppose Medicare’s new rules.
“I think the addition of the new compendia this year is an important increase in the bandwidth,” she said.
Critics say the agency also seems to have played down the potential financial conflicts of interests between the drug industry and the producers of the compendiums. The draft study that was completed in October notes that one of the new guides is published by the National Comprehensive Cancer Network, a group of 21 leading cancer centers that routinely employs experts who have financial ties to the drug industry.
William T. McGivney, the network’s chief executive, said each committee of reviewers had 20 to 30 members, which “diminishes the opportunity for dominance of one person’s opinion,” regardless of any ties to drug makers.
Then there is the American Hospital Formulary compendium, the one that Medicare was using before the November changes and will continue to consult. It has long been published by the nonprofit American Society of Health-System Pharmacists. But last year the society forged a financial relationship with a foundation linked to drug companies and some cancer doctors’ private practices.
A drug company can apply to that foundation, the Foundation for Evidence-Based Medicine, and pay a $50,000 fee to have new uses of its drug reviewed by the compendium within 90 days. The foundation was started in 2007 by the Association of Community Cancer Centers, which represents oncology practices, and says it received about $200,000 in initial funding from drug makers.
Gerald K. McEvoy, the guide’s editor in chief, said the application fee was meant to raise money to pay for additional researchers, to address previous criticism that the publication was too slow to vet new evidence. The foundation insulates the guide’s staff from industry pressure, he said, and fewer than one-third of the reviews under the new arrangement have resulted in a positive recommendation in the compendium.
Medicare officials acknowledge that some of the potential conflicts need to be addressed. But they say they have confidence in the guides they have chosen. “We had significant conversations with all the companies,” Dr. Phurrough said.