A Food and Drug Administration panel took a major step Wednesday toward ending use of the best-selling drug Avastin for treating advanced breast cancer in the United States, despite appeals from patients and the company manufacturing the drug.
The six-member FDA advisory committee unanimously concluded the drug was harming women more than it was helping them. FDA Commissioner Margaret Hamburg must make a final decision, but agency commissioners usually follow advisory panel recommendations.
The fate of the drug for breast cancer has been closely watched by patients, oncologists, women's health advocates, health care policymakers, politicians and the pharmaceutical industry.
The case has triggered strong reactions from advocates of easier access to new treatments and from critics fearing health care rationing. It is seen as one of the most visible medical examples of scientific evidence winning out over public outcry. Ultimately, clinical trials failed to support the drug's early promise for breast cancer treatment.
"I think we all wanted Avastin to succeed, but the reality is that these studies did not bear out that hope," said Natalie Compagni Portis, who represented patients on the panel.
If the FDA revokes Avastin's approval for treatment of breast cancer, the drug would remain available for other cancers, enabling doctors to prescribe it "off-label" for breast cancer patients. But the decision would probably prompt insurers to stop paying for the drug -- which costs about $88,000 a year per patient -- for breast cancer treatment. Breast cancer patients also would lose eligibility for a program sponsored by the drugmaker, Genentech, that caps the annual cost at about $57,000 for women making less than $100,000 a year.
"A terrible tragedy has occurred," said Frank Burroughs of the Abigail Alliance for Better Access to Developmental Drugs, which is among the groups that has been pressing the FDA to maintain Avastin's approval on the grounds that it benefits some patients.
But the decision was praised by others who said it was important for the agency to base its decisions on the scientific evidence, not emotional appeals, especially when lives are at stake and controlling health care costs is so crucial to the nation's economy.
"Now we must focus attention and resources on drugs that will truly make a difference for women suffering from breast cancer. Avastin does not," said Fran Visco of the National Breast Cancer Coalition.
The FDA is not supposed to acknowledge cost in drug approvals, and officials have stressed that the price was irrelevant. But coming amid a continuing national debate over President Obama's health care overhaul, the Avastin fight has become caught in the politically sensitive struggle over medical spending and effectiveness.
Avastin is among the costliest of a new generation of anti-cancer medications that appear to give patients a few extra months of life. It was the first drug designed to fight cancer using a new strategy -- inhibiting blood flow to tumors -- and was approved for treating several malignancies.
The agency moved in December to revoke its 2008 approval of Avastin for breast cancer, based on a July 20 advisory committee conclusion that new studies had not shown that the drug extends life and had indicated that it slowed tumor growth for far less time -- perhaps as little as a month.
"The FDA has stubbornly put itself between patients and their doctors by severely limiting access to a life-extending drug, and has even cited cost as a criterion," Sen. David Vitter, R-La., said Wednesday.
"We are very disappointed by the committee's recommendation," Hal Barron, Genentech's chief medical officer, said.
Immediately after the final vote, patients in the audience erupted in shouts against the FDA and its experts. "What do you want us to take? We have nothing else!" shouted Christi Turnage, of Madison, Miss.
FDA officials said existing data indicated Avastin provided little or no benefit and put women at risk for potentially life-threatening complications, including high blood pressure, hemorrhages and heart attacks.