Food and Drug Administration Commissioner Dr. Margaret Hamburg said on Friday she is revoking approval for doctors to use the world’s best-selling cancer drug Avastin to treat breast cancer, concluding that it is not safe or effective for that use.
Avastin will remain approved for certain types of colon, lung, kidney, and brain cancer, the FDA said. Some breast-cancer advocates have been fighting to retain approval to use the drug, which starves tumors.
“As a doctor, as a woman, and as a parent I recognize how frightening and difficult it is to receive the diagnosis of metastatic breast cancer,” Hamburg said on a conference call. “But despite the hopes of investors, patients, industry and even the FDA itself, the results of vigorous testing can be disappointing. That is the case with Avastin for metastatic breast cancer.”
Avastin is not only expensive – costing up to $88,000 a year – it can also cause serious side effects including severe high blood pressure; bleeding and hemorrhaging; heart attack or heart failure; and the development of holes in the nose, stomach, intestines, and elsewhere.
In June, the Centers for Medicare and Medicaid Services said it would continue to pay for Avastin to treat breast cancer if a doctor prescribed it, even if FDA did revoke approval.
CMS spokesman Don McLeod said the agency is not changing that policy anytime soon.
“Medicare will continue to cover Avastin. CMS will monitor the issue and evaluate coverage options as a result of action by the FDA but has no immediate plans to change coverage policies," McLeod said in an e-mail.
Advisers to FDA said that evidence clearly shows that the drug does not help breast-cancer patients live any longer and recommended that the agency revoke its approval for that indication.
Advocates and some breast-cancer patients complained -- and even demonstrated outside the FDA -- arguing that the decision would take away what they believe to be a life-saving drug. The CMS statement suggests that such fears are unfounded. Private insurers often follow CMS's lead in deciding what drugs to cover. Because it’s an approved drug, any doctor can prescribe Avastin for any patient--a practice called off-label prescribing.
Hamburg acknowledged that in a conference call.
“Outside of our mandate, various insurers and payers will no doubt look at our decision and will make their own decisions,” Hamburg said.
The battle illustrates the fine line that Washington regulators must walk as they seek to balance patient pressures with the medical evidence. Last July, Sen. David Vitter, R-La., wrote to FDA to accuse its advisers of taking cost into consideration--something the agency denies.
“Throughout this process, I expressed my deep concerns and objections to restricting access to Avastin,” Vitter said in a statement Friday. “Sadly, the FDA has decided to take that option away from them.”
An aide to Vitter said the senator would continue to monitor coverage of the drug, particularly at CMS.
Hamburg said the agency could not identify a specific sub-set of women who benefited from using Avastin to treat breast cancer.
“I hope Genentech will pursue that research,” Hamburg said. Avastin is made by Genentech, a California company that is now a subsidiary of Swiss drug giant Roche. The drug was cleared for use against breast cancer in 2008 under an accelerated approval process that required Roche to do studies to confirm that the drug helped breast cancer patients live longer.
The studies did not show any benefits.
American Cancer Society Deputy Chief Medical Officer Dr. Leonard Lichtenfield said he hoped insurance companies already covering Avastin for women who appeared to be benefiting would continue.
"The full impact of this decision is difficult to determine at this time," Lichtenfield wrote on an ACS blog. "At the least, we would hope that insurers will continue to cover treatment with Avastin in those women with breast cancer currently on the drug and who are showing a benefit from its use."
The National Breast Cancer Coalition welcomed the FDA decision.
"We are gratified that the FDA has continued to support science and put what is best for patients above political, public and industry pressures," the group said in a statement. "The decision on Avastin supports patients' trust that the drugs they are given have been proven to give them benefit and not expose them to greater harm."