First published 6/30/11 in The Fiscal Times:
How much should you, your insurer or the government pay for an extra month of life? How much is an extra month of hope worth, even if doesn’t extend life?
Those questions never came up during this week’s Food and Drug Administration appeal hearing on the agency’s decision to withdraw approval from the Roche-Genentech drug Avastin for advanced metastatic breast cancer. But they will be front and center in the years ahead as medical science and the drug industry continue to bring advanced cancer therapeutics to market that have only marginal effects on a devastating disease that takes half million lives a year, most of whom are elderly and receive treatment through Medicare.
The FDA is not allowed to consider costs, only benefits and risks, when deciding whether to approve or withdraw approval for drugs or other medical devices. Yet cost was clearly the backdrop as the FDA’s Oncology Drugs Advisory Committee on Wednesday voted 6-0 to reaffirm last December’s FDA decision to withdraw approval for the $88,000-a-year drug, which only stops breast tumors from progressing for a few months and doesn’t extend life. Genentech had appealed the decision. FDA Commissioner Margaret Hamburg will make the final determination sometime later this year.
Avastin, one of the new targeted therapies that a decade ago were heralded as a major advance in cancer therapeutics, has turned out to be much less effective than originally hoped. Although it has been approved for colon and lung cancer, its effects on those common cancers are also limited.
In 2008, the FDA granted Avastin so-called “accelerated approval” for metastatic breast cancer based on a single clinical trial showing the drug, when combined with standard chemotherapy, delayed tumor progression by 5 ½ months compared to chemo alone. There was also a hint that it might prolong life.
The accelerated approval program, which was created in the early 1990s as a way of rushing promising AIDS drugs to market, requires companies to conduct additional clinical trials to confirm the promise shown in the earlier test. However, when the new trials came in last year, they not only didn’t show any increase in overall survival, they failed to fully replicate the delay in tumor progression seen in the original trial.
That scientific evidence was of little interest to the dozens of breast cancer patients, their families, physicians and advocacy groups who showed up at the hearing to offer emotional testimony in favor of the drug. Shannon Morgan of Charlotte, N.C., who has survived a diagnosis of metastatic breast cancer for over two years, insists she is alive today because she was treated with Avastin (the median time of survival in the original trial was 24.8 months on chemotherapy alone, compared to 26.5 months on chemo plus Avastin; statistically there was no difference, meaning the results could have been by chance). She’s afraid her insurance company will stop paying for the drug if the FDA withdraws approval
“You may be rich but I am not,” she testified. “If you take Avastin for breast cancer off the label, how many of us successful users will die during that time? Do you want that on your conscience?”
The fears and hopes of patients like Morgan lie at the heart of the nation’s emerging struggle with the rising cost of cancer care, which is one of the major drivers of rising Medicare costs. A study that appeared earlier this year in the Journal of the National Cancer Institute estimated the U.S. paid about $125 billion for treating the 16 most common forms of cancer in 2010. By 2020, that tab is likely to grow to $173 billion because of the aging of the population and the rising cost of new drugs and other therapeutics.
But so far patient fears that the government or insurance companies might ration care by using cost-benefit analysis like Great Britain’s National Institute for Clinical Excellence has not come to pass. Medicare and most insurers continue to pay for any drug listed in guidelines written by the National Comprehensive Cancer Network, an alliance of the nation’s largest academic cancer centers.
In the wake of the FDA’s announcement last December that it planned to remove the breast cancer indication from Avastin’s label, NCCN announced it wouldn’t follow suit. Many oncologists who use the guidelines insist the drug provides improved quality of life for their patients, since every month the tumor doesn’t progress is a month they can continue believing they may defeat the disease. They can continue to prescribe Avastin for breast cancer patients “off label” since it is still available for other indications. In addition, 11 of the 33 physicians on the NCCN breast cancer guideline writing panel have financial ties to Genentech, including the committee’s chairman Robert Carlson of Stanford.
“If the NCCN recommends it, regardless of what the FDA does, we wouldn’t make any changes,” said Lee Newcomer, senior vice president for oncology at UnitedHealthcare, the nation’s largest private insurer with 25 million covered lives. “We have lots of off-label indications from the NCCN that we pay for. We wanted to have those decisions made outside the company to avoid any impression that we would deny drugs simply for costs.”
But that precludes insurers like United from bargaining for a lower price based on the drug or other intervention’s limited efficacy. “We are bound to cover an FDA-approved drug by insurance regulators and at United, by our own policy, we are bound to use NCCN recommendations,” Newcomer said. “The manufacturer is well aware of that. They know we have no leverage at the negotiating table. Whatever price they set, we’re forced to pay.” The same is true for Medicare.
Merrill Goozner is an independent health care journalist and a regular contributor to the Fiscal Times.