June 30, 2011 — FDA's Oncologic Drugs Advisory Committee on Wednesday unanimously voted that the drug Avastin is neither safe nor effective for the treatment of advanced breast cancer and recommended that the agency revoke approval of the medication for treatment of the disease, CQ HealthBeat reports. FDA Commissioner Margaret Hamburg will make the final decision on whether the drug will remain approved to treat breast cancer (Bunis, CQ HealthBeat, 6/29).
The two days of hearings were marked by emotional testimony from a few patients who said the drug had benefited them and should remain on the market. Genentech had requested that FDA retain approval while it conducted one more trial to try to prove that the drug works. However, the panel said trials to date failed to show any scientific evidence that Avastin extended or improved women's lives (Pollack, New York Times, 6/29). Rather, the drug exposed patients to potentially life-threatening risks, the panel said (Mundy, Wall Street Journal, 6/30).
Avastin was approved for treatment of breast cancer under FDA's accelerated approval process, which hastens approval of drugs to treat serious diseases if the company conducts further studies. FDA in December 2010 announced it had started the process of revoking the approval of Avastin to treat metastatic breast cancer after several studies found that the drug does not extend women's lives and carries significant risks, such as high blood pressure and internal bleeding. In February, the agency granted a request from Genentech, the drug's maker, for a hearing allowing it to defend the medication (Women's Health Policy Report, 6/29).
Avastin also is approved as a treatment for other types of cancer. Even if approval for breast cancer is withdrawn, doctors will be able to prescribe Avastin "off-label" to treat the disease. However, insurers might refuse to pay for the treatment, which can cost $88,000 annually (New York Times, 6/29).
In a statement, the panel wrote, "The commissioner's decision related to breast cancer will not affect Avastin's approved indications for use in colon, lung, kidney and brain cancers. That is, regardless of the final decision on metastatic breast cancer approval, Avastin will remain on the market" (CQ HealthBeat, 6/29).
Natalie Compagni Portis, the patient representative on the panel, said, "I think we all wanted Avastin to succeed, but the reality is that these studies did not bear out that hope" (Stein, Washington Post, 6/29).
Panelist Ralph Freedman, a gynecologic oncologist at M.D. Anderson Cancer Center, said, "The agency has to look at protecting a larger number of patients," adding, "Sometimes they have to make a decision that doesn't favor individual patients, but it's on the basis of the whole" (New York Times, 6/29).
Panelist Wyndham Wilson of the National Cancer Institute said, "Reasonable people would agree that a month or so [of] progression-free survival is not a meaningful benefit" compared to the mortality risk (Wall Street Journal, 6/30). He added, "We have a standard and we shouldn't be changing that standard unless we have a very good reason" (New York Times, 6/29).
Officials from FDA and Genentech have until July 28 to submit additional comments to Hamburg before she makes her final decision. Karen Midthun, director of FDA's Center for Biologics Evaluation and Research and panel leader, said, "I cannot tell you at this point when the decision will be finalized and issued," adding, "But we all do recognize the importance of resolving these issues expeditiously" (CQ HealthBeat, 6/29).
Genentech officials said in a statement, "We are very disappointed by the committee's recommendation and hope the commissioner does not decide to remove an important medicine for women with this incurable disease who already have too few treatment options," adding, "We remain ready to collaborate with the FDA to find a solution that is in the best interest of patients who need Avastin" (CQ HealthBeat, 6/29). Edward Lang, a Genentech spokesperson, said the company would propose additional compromises, such as restricting the drug's use to aggressive cancers or changing its label. Lang also said there is a possibility that the company would challenge an unfavorable decision in court (New York Times, 629).
Some breast cancer advocates have applauded the decision. Fran Visco, president of the National Breast Cancer Coalition, said the panel voted for "scientific evidence in the face of intense public and media pressure," adding that women "deserve drugs that save lives and significantly improve quality of life. We need to focus on finding therapies that will make a real difference" (Szabo, USA Today, 6/30).