The Public Policy
FDA Decision Dooms Cancer Patients
Opponents of medical progress are celebrating the decision of a Food and Drug Administration panel to revoke approval of the use of Avastin in treating advance breast cancer.
Not so fast.
As Scott Hensley, a health care reporter for NPR, wrote:
The day after a panel of experts advised the Food and Drug Administration to go ahead with plans to revoke approval of Avastin to treat breast cancer, European authorities moved in the opposite direction.
The European Medicines Agency (EMA) gave the OK to an expansion of Avastin's approval to include using the drug in combination with Xeloda, a chemotherapy drug, to treat metastatic breast cancer, Genentech parent company Roche said Thursday.
The Europeans considered the same information that hasn't proved persuasive to U.S. regulators. Among other things, a study called RIBBON 1 found Avastin plus Xeloda increased progression-free survival or PFS -- (living longer without tumor growth) of women by 2.9 months over women getting Xeloda alone.
This improvement seems small but is actually quite significant. Gains in PFS among patients with cancers that spread through the body are hard to achieve because when the disease spreads it is considered incurable. And over time, the small victories over incurable cancers extend and improve life.
The EMA believes 2.9 months is a great beginning. The FDA sees nothing less than 6 months to be worth it and worked hard to make sure Avastin would never hit that mark.
The FDA's Richard Pazdur, who heads up FDA's Office of Cancer Drugs, had approved Avastin's use in breast cancer on the condition that future studies confirmed the improvement in PFS. Genentech conducted three studies that hit that mark. But then Pazdur claimed that PFS was not enough. He wanted evidence that median overall survival increased.
While an increase in overall survival has been considered the gold standard for cancer drugs, progression-free survival has been used as a benchmark for longer and better quality of life.
That's because a standard like overall survival only tells you what the median survival is for all patients -- it doesn't take into account individual differences in response based on genetic, clinical and other factors.
Yet when results of additional studies this summer concluded that Avastin doesn't extend life on average, an FDA advisory committee suddenly decided that Avastin should be measured by overall median survival -- not progression-free survival.
Before this measurement switch, the FDA admitted that the new studies showed a statistically significant improvement in PFS with Avastin. But then it said, "the magnitude of treatment effect, as commonly assessed by clinicians based on differences in median PFS, was marginal."
Pazdur seems to want to toughen standards based on a one-size-fits-all measure that requires longer and more expensive clinical trials. In doing so he has been supported by such left-leaning and anti-innovation entities as the Center for Science in the Public Interest's Merrill Goozner and Public Citizen, a Naderite group that has warned against most new medicines for cancer and diabetes since the 1970s. These and other Avastin opponents -- who have long regarded the drug as a symbol of corporate greed -- have lobbied for median survival as the key standard. They know that applying that standard would harm the perception of Avastin's effectiveness.
Goozner -- who has no medical background -- was appointed to an FDA advisory committee on pharmaceutical science. Two senior Public Citizen operatives, Peter Lurie and Larry Sasich, now set policy for the FDA. Fran Visco, the head of the National Breast Cancer Coalition, applauded the FDA decision after lobbying for it over the past year. Visco, a Democrat, is also on Experts Advisory Panel for the Universal Health Insurance Program at the New America Foundation, a left-wing think tank supporting Obamacare. The NBBC also supported the administration's decision not to cover mammograms for women under 50 though many breast cancers grow faster and earlier in African-American women.
To these groups, the FDA decision was a triumph. But their effort to manipulate the FDA will backfire. The EMA and every major group of cancer providers support Avastin's use. Cancer patients moblilized spontaneously to keep Avastin's label. They will take on the anti-innovation establishment and the FDA with greater intensity and vigor.
Medicare and some health plans will try to stop paying for Avastin. But Congress will hold oversight hearings on the Avastin decision en route to amending FDA's statute through one of many bills calling for faster access to new treatments.
The administration will have to respond to show it's not using FDA to ration care or slow innovation.
In the long term this is all well and good. But meanwhile many women dying of breast cancer will have to go to Europe to get a drug that could keep them alive.
Robert M. Goldberg is vice president of the Center for Medicine in the Public Interest and founder of Hands Off My H ealth, a grass roots health care empowerment network. His is new book, Tabloid Medicine: How the Internet is Being Used To Hijack Medical Science For Fear and Profit, was published last month by Kaplan.
