The Food and Drug Administration on Friday revoked the approval of the best-selling drug Avastin for treating advanced breast cancer in the United States, despite appeals from distraught patients and the company that manufactures the drug.
FDA Administrator Margaret Hamburg issued a 69-page decision outlining her decision, which was based on the recommendation of a six-member FDA advisory committee that unanimously concluded in June that the drug was harming women more than it was helping them.
“This was a difficult decision. FDA recognizes how hard it is for patients and their families to cope with metastatic breast cancer and how great a need there is for more effective treatments. But patients must have confidence that the drugs they take are both safe and effective for their intended use,” Hamburg said.
“After reviewing the available studies it is clear that women who take Avastin for metastatic breast cancer risk potentially life-threatening side effects without proof that the use of Avastin will provide a benefit, in terms of delay in tumor growth, that would justify those risks. Nor is there evidence that use of Avastin will either help them live longer or improve their quality of life,” she said.
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 also being seen as one of the most visible medical examples of scientific evidence winning out over an animated public outcry. Ultimately, clinical trials failed to support the drug’s early promise for breast cancer treatment.
Despite the revocation, Avastin will 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 drugmaker Genentech that caps the annual cost at about $57,000 for women making less than $100,000 a year.
Charlotte Arnold, a spokeswoman for Genentech, said, “We are disappointed with this outcome. We remain committed to the many women with this incurable disease and will continue to provide help through our patient support programs to those who may be facing obstacles to receiving their treatment.”
The decision was criticized by some advocates for patients. But it was praised by others, including several leading breast cancer advocacy groups and experts 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.
“We have all debated this issue for some time now. The science shows us that Avastin does not save lives and that it harms women. We support the FDA and Commissioner Hamburg’s decision. It is the right one,” said Fran Visco of the National Breast Cancer Coalition.
“We should be using our time and resources to find drugs that work. It is unfortunate that Avastin does not. Marketing campaigns and appeals by the public devastated by breast cancer cannot change that. These women deserve drugs that well-designed research tells us will prolong their lives. Let’s focus our attention on making that happen,” Visco said.
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 fight about Avastin has become entangled 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.
Its 2008 approval for breast cancer was controversial from the start. Only one study had found that the drug appeared to slow the growth of an advanced breast tumor, delaying progression by about 5 1 / 2 months. It remained unclear whether patients lived longer or enjoyed a better quality of life. So the FDA authorized Avastin under a special program designed to make advances in treatment available to patients quickly. The deal was that Genentech must validate the risk-benefit ratio with additional studies.
The agency moved in December to revoke the approval based on a July 20 advisory committee conclusion that the 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.
Genentech immediately challenged that decision. The FDA’s two-day meeting in June was marked by unusually tense exchanges between representatives of Genentech and agency officials. They clashed repeatedly over issues including the interpretation of studies evaluating Avastin and whether the agency had shifted its criteria for approving the drug.
The company vowed to start a new study of Avastin in combination with the drug paclitaxel in previously untreated metastatic breast cancer patients to “evaluate a potential biomarker that may help identify which people might derive a more substantial benefit from Avastin,” she said.
Before the FDA announced plans to revoke Avastin’s approval for breast cancer in December, the drug was being prescribed annually to about 17,500 U.S women with breast cancer. Globally, Avastin had annual sales of $6.8 billion in 2010, making it the world’s best-selling cancer drug and top-selling product for Genentech and Roche, its Swiss owner. Genentech has waged an unprecedented campaign to save U.S. approval of the drug, which alone brings in nearly $1 billion a year.
