Federal regulators took the unusual step Thursday of moving to revoke approval of a drug that women with advanced breast cancer turn to in a last-ditch effort to save their lives.
The decision intensifies a politically charged debate over costly cancer drugs that appear to produce modest benefits - if any.
The Food and Drug Administration concluded that four studies indicate that the blockbuster drug Avastin, the world's best-selling cancer drug and among the most expensive, does not help patients with metastatic breast cancer live longer or provide other benefits that outweigh its potentially life-threatening risks.
"Today's decision was a difficult one for the agency," said FDA official Janet Woodcock, who stressed that the decision was made by officials with experience treating cancer patients, including some who had been "personally touched by the disease."
Woodcock added, "FDA is responsible for ensuring the products we approve for patients both effective and safe."
The FDA gave Genentech, which makes Avastin, 15 days to seek a hearing to review the decision, and the company immediately said it would make the request. That would mark the first time a company has challenged the FDA on such a decision. Genentech maintains Avastin does help some patients, noting that the European Medicines Agency had affirmed the drug's benefits the same day.
FDA Commissioner Margaret A. Hamburg must decide whether to grant company's request or proceed with the withdrawal. Until the final FDA decision, Avastin, which is prescribed to about 17,500 breast cancer patients a year, remains approved for breast cancer.
The FDA's step, which was recommended July 20 by an agency advisory committee, was based on new studies that officials concluded had not shown that the drug extends life and indicated that the drug slowed tumor growth for perhaps as little as a month. At the same time, Avastin can cause serious, potentially life-threatening side effects, including very high blood pressure, hemorrhages, heart attacks and heart failure.
Avastin was the first drug designed to fight cancer by blocking blood flow to tumors. But it is also one of the costliest of a new generation of anti-cancer medications that appear to eke out only a few extra months of life. It has global sales of $5.8 billion, and it is the top-selling product for Roche, whose Genentech unit makes it. Its use to treat breast cancer brings in about $855 million a year in revenue in the United States.
If the agency follows through with the revocation, the drug would remain available to treat cancer of the brain, colon, kidney and lung, and doctors could continue to prescribe it for breast cancer as an "off-label" use.
But the loss of FDA approval for breast cancer would probably prompt insurers to stop paying for Avastin for metastatic breast cancer. Avastin costs about $8,000 a month. Medicare was awaiting the outcome of the FDA process before making a decision on whether to continue paying for Avastin, Woodcock said.
Breast cancer patients also would lose eligibility for a program in which Genentech caps the annual cost of the drug at about $57,000 for women making less than $100,000 a year.
Eric P. Winer, chief scientific adviser for the breast cancer advocacy group Susan G. Komen for the Cure, also expressed concern that cost might have influenced the agency.
"Though the FDA is not focusing on cost, I think it's hard, particularly in 2010, not to pay attention to the fact that this is a very expensive drug," said Winer, director of the Breast Oncology Center at the Dana-Farber Cancer Institute in Boston.
The decision comes as Medicare is conducting an unusual review to determine whether to pay for the first vaccine approved to treat cancer for men with prostate cancer. The vaccine, called Provenge, costs about $93,000 per patient.
The possibility of Avastin losing authorization for breast cancer prompted anguish among some breast cancer advocates, doctors and patients, including some who started petition drives to persuade the FDA to retain the drug's authorization.
"This is horrific," said Pat Howard, 65, of Summerfield, Fla., who has been taking Avastin for five years and credits the drug with putting her cancer in remission. "This is like saying, 'We don't value your life.' I'm shocked. I'm totally shocked. Without Avastin, I'm going to die."
Internet chat rooms for women with breast cancer lit up with similar expressions of alarm.
Several experts and advocates argued that a some breast cancer patients clearly do benefit from Avastin, and they urged insurance companies to continue paying for the drug.
But other experts and consumer and patient advocates welcomed the announcement, saying the weight of the evidence was that Avastin was not beneficial and potentially harmful.
"We know how difficult it is to deal with breast cancer. We understand how painful it is that we do not know how to cure metastatic disease," said Fran Visco of the National Breast Cancer Coalition. "We need to focus advocacy, public policy and resources on saving lives and doing more good than harm."
The FDA approved Avastin for advanced breast cancer in 2008 despite divided opinion about its usefulness for that purpose. Only one study had found that the drug appeared to slow the growth of an advanced breast tumor, producing a delay of about five months. It remained unclear whether patients lived longer or experienced an improved quality of life.
The FDA authorized Avastin under a special program designed to make new treatments available as quickly as possible, with the understanding that pharmaceutical companies would conduct follow-up studies validating the effectiveness of the drugs.