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On June 28, the Food and Drug Administration (FDA) will hold a hearing to decide the fate of Avastin, a drug taken by thousands of women fighting late-stage breast cancer. Many of these women have pleaded for continued access to the drug, which they consider a matter of life and death.
But this case is really about what will guide decisions on treatment options—the best judgment of doctors and their patients, or the policy preferences of the FDA.
Last year, the FDA began the process of revoking Avastin's approval for breast cancer. Some leading oncologists applauded the decision, arguing that, for the average patient, Avastin doesn't work very well and has significant side effects.
Patient advocates and thousands of women who credit their survival to Avastin argue that it's unfair for the FDA to remove one of the few available options for patients diagnosed with terminal cancer. They're right.
Avastin originally hit the market in 2004 to treat other cancers, and in 2008 the FDA conditionally approved it for breast cancer. Initial testing showed that, on average, Avastin didn't lengthen patients' overall survival time. But it did slow tumor growth, giving many patients a longer "progression-free" survival. What this means is that dying patients get a precious few extra months of quality time they can spend with family and friends, travel rather than being confined to a bed, or get their personal effects in order.
Scientist Stephen Jay Gould lived another 20 years after being diagnosed with lung cancer.
A small percentage of patients taking Avastin have been cured of their breast cancer. But the drug's permanent approval hinged on the results of two additional clinical studies focusing on the progression-free survival end-point experienced by the majority of Avastin users. As before, neither study found an increase in overall survival, but they did record modest gains in progression-free survival—about five and a half months longer than those on the alternative treatment. That wasn't enough for the FDA, so the agency moved to revoke Avastin's approval for breast cancer last July.
The weakness of the FDA's reasoning here is that averages ignore that individual patients respond differently to treatments. Particularly with life-threatening illnesses, where the downside of any treatment is relatively small, average or median survivability too often masks the fact that some patients respond very well.
When well-known scientist Stephen Jay Gould was diagnosed with a rare form of lung cancer in July 1982, he was told the diagnosis meant a median survival time of just eight months. His doctor gave up on him. But he lived another 20 years.
"Means and medians are the abstractions," he wrote in Discover magazine in 1985. "Therefore, I looked at the mesothelioma statistics quite differently—and not only because I am an optimist . . . but primarily because I know that variation itself is the reality."
Like Gould's doctor, the FDA and its technocratic supporters are giving up on breast cancer patients because of their slavish obsession with median response rates. Everyone can agree that, on average, Avastin does not extend most patients' life expectancy. But some patients have responded incredibly well, living years longer than expected. The medical community calls them "super responders." Statisticians might describe them as "outliers." But they're real people, alive because of Avastin.
In a recent letter to USA Today, Shannon Morgan of Charlotte, N.C., wrote: "I've been on Avastin nearly three years, am able to work full-time and lead an active life. I'm not alone. Every drug has side effects. The FDA seems to forget that the primary side effect of Stage 4 cancer is death."
Avastin is expensive: $56,000 to $96,000 per year. The expected value of treatment for any given patient—the abstract "median"—seems small, so a private health plan may understandably balk at paying such a tab. But cost-effectiveness calculation isn't the FDA's job, and in fact the law forbids the agency from considering anything but a product's safety and effectiveness while it's evaluated.
What is the logic of keeping terminally ill patients from potential treatments? Can't they at least go down fighting?