Federal regulators are considering taking the highly unusual step of rescinding approval of a drug that patients with advanced breast cancer turn to as a last- ditch hope.
The debate over Avastin, prescribed to about 17,500 women with breast cancer each year, has become entangled in the politically explosive struggle over medical spending and effectiveness that flared during the battle over health care reform: How should the government balance protecting patients and controlling costs without restricting access to often-costly cutting-edge treatments?
The Food and Drug Administration is reviewing the recommendation of influential scientific advisers to revoke authorization of the drug to treat metastatic breast cancer. Contrary to initial research, new studies indicate that the benefits of the expensive drug, which costs $8,000 a month, do not outweigh its risks, the advisory panel concluded.
Cost plays role
Citing a dearth of evidence of the drug’s effectiveness, its potential toxic side effects and its high cost, many cancer experts, patient advocates and others are welcoming the prospect that Avastin’s authorization for breast cancer might be repealed. But the possibility is alarming other cancer specialists, women taking the drug, some members of Congress and advocates for giving patients as much access to as many treatments as possible.
The FDA is not supposed to consider costs in its decisions, but if the agency were to rescind approval, insurers are likely to stop paying for treatment.
“It’s hard to talk about Avastin without talking about costs,” said Eric Winer, director of the Breast Oncology Center at the Dana-Farber Cancer Institute in Boston. “For better or worse, Avastin has become in many ways the poster child of high- priced anti-cancer drugs.”
Avastin is the best-selling cancer drug in the world, with global sales of $5.4 billion, and it is the best-selling product for Roche, whose Genentech unit makes it. Its use to treat breast cancer brings in about $8.5 million in revenues in the United States a year.
Avastin was the first drug designed to fight cancer by blocking blood flow to tumors, which has been hailed as one of the first significant innovations in decades in the war on cancer. But Avastin is also one of the most expensive of a new generation of anti-cancer medications that eke out only a few extra months of life.
The FDA endorsed Avastin for advanced breast cancer in 2008 despite divided opinion about its usefulness for that purpose. Only one study found that the drug appeared to delay an advanced breast tumor from growing by about five months. It remained unclear whether patients actually lived longer or experienced an improved quality of life.
But the FDA approved Avastin under a special program designed to make new treatments available as quickly as possible, with the caveat that pharmaceutical companies conduct follow- up studies validating their drug’s effectiveness.
The agency has been criticized for failing to adequately review such approvals. Only one of at least 90 drugs that have received accelerated approval has been pulled.
An FDA advisory committee voted 12-1 on July 20 to withdraw Avastin’s authorization for advanced breast cancer based on two new studies that the advisers concluded had failed to show that the drug extends life.
Not only that, the committee concluded that the studies indicated the drug slowed tumor growth for even less time — perhaps as little as about a month.
“The vast majority opinion of the committee was that the drug was not doing very much, and what it was doing was more than offset by the negative,” said Wyndham Wilson of the National Cancer Institute, who chaired the committee.
Avastin can cause a variety of potentially serious side effects, including blood clots, bleeding and heart failure.
Drugmaker disagrees
The recommendation has been praised by many cancer experts and by advocates for breast-cancer patients.
“The FDA should never have approved Avastin for breast cancer to begin with,” said Fran Visco of the National Breast Cancer Coalition. “We don’t see evidence of benefit, but we do see evidence of harm.”
But the company disputes the committee’s interpretation of the studies, saying they demonstrated that the drug reduced the risk of a woman’s cancer progressing or her risk of death from the cancer by between 31 percent and 52 percent.
“It’s our opinion that when we look at the data, they are clinically meaningful,” said Sandra Horning, who heads cancer research for Genentech. “Avastin should continue to be an option for women.”
The FDA is not required to follow the recommendation, but the agency usually does. A decision is expected by Sept. 17.
Avastin is approved for use in treating several cancers, including those of the colon, lung, kidney and brain. So doctors could continue to write prescriptions for it for breast cancer, as an “off-label” use.
But in addition to prompting insurers including Medicare to stop paying for Avastin, an FDA revocation of approval for its use in breast-cancer treatment might mean that patients would lose eligibility for a program in which Genentech caps the annual cost of the drug at $57,000 for women with annual incomes of less than $100,000.
That prospect has prompted despair among women taking the drug, some of whom have started petition drives to persuade the FDA to ignore the advisory committee’s recommendation.
“I’m very upset,” said Leslie Twohig, 48, of Lothian, Md., who has been taking Avastin for eight months and credits the drug with helping her survive.
