SAN FRANCISCO (AP) - For the past 2½ years, Terry Kalley's wife has battled breast cancer with Avastin, the world's bestselling cancer drug. About a year ago, he found out that federal regulators were considering pulling their approval of the drug for that use.
Kalley quit his job and has dedicated himself to making sure that doesn't happen. But the odds don't look good.
A large California health insurer's recent decision to stop covering Avastin for breast cancer comes as the most visible sign yet that the drug patient advocates like Kalley believe are keeping their loved ones alive may soon be much harder to come by.
Blue Shield of California will stop covering new Avastin prescriptions for breast cancer patients starting Oct. 17. Patients already on the drug will still be covered, and exceptions could be made for individual cases reviewed by a panel of cancer experts.
The not-for-profit insurer cited an unprecedented vote by a U.S. Food and Drug Administration panel that found the drug was not effective enough against breast cancer to risk its potentially dangerous side effects.
"Blue Shield's decision follows an FDA advisory panel's unanimous determination in June that Avastin is ineffective and unsafe for advanced breast cancer," the insurer said in a statement.
The FDA has not made a final decision on whether to follow the panel's advice and withdraw the fast-track approval the agency granted the drug in 2008 as a treatment for breast cancer that has spread to other parts of the body.
With 3.2 million members, Blue Shield appears to be the largest health insurer so far to stop covering Avastin for breast cancer. Three smaller insurers also no longer cover Avastin for breast cancer, according to a spokeswoman for Genentech Inc., which developed and makes the drug as a unit of Swiss pharmaceutical giant Roche AG.
Roche is asking the FDA not to pull its approval while the company conducts another study to prove its effectiveness when used along with another chemotherapy drug. Avastin, Roche's bestselling drug, brought nearly $6.5 billion in sales last year. The company stands to lose an estimated $1 billion if the FDA follows the panel's recommendation.
Medicare still covers the drug for breast cancer. Avastin can cost about $100,000 per year including fees, making it one of the world's most expensive drugs.
"We still believe the medicine is an important option to women with breast cancer," said Genentech spokeswoman Charlotte Arnold. "We still hope that the commissioner may consider keeping it approved for people with the greatest need."
Avastin also has federal approval for use as a treatment for colorectal and kidney cancer, as well as some forms of lung cancer and brain tumors. Those uses are not being questioned.
Less than a month after the FDA panel's 6-0 vote, a group representing the country's leading cancer centers voted 24-0 to maintain its position recommending Avastin for breast cancer when used with the chemotherapy drug paclitaxel.
Avastin is an "appropriate therapeutic option for metastatic breast cancer," the expert panel of the National Comprehensive Cancer Network found.
The FDA granted Avastin's initial approval for breast cancer after one study showed the drug slowed tumor growth for about five months longer when paired with paclitaxel, compared to using paclitaxel alone. Doctors had prescribed Avastin off-label for breast cancer before that approval, but two later studies showed Avastin had little impact. None of the studies showed patients lived longer on Avastin.
Side effects of Avastin in some patients can include potentially fatal bleeding, surgery and wound healing problems and holes in the stomach, small intestine and colon.
Not all patients suffer severe side effects, just as some appear to improve while on Avastin. Others see little or no effect on their cancer.
Researchers don't have a good sense of why Avastin affects patients differently, and Arnold said Genentech hoped the FDA would keep the drug's approval while its scientists explored that question.
Kalley testified at the FDA panel's June hearing and formed the group Freedom of Access to Medicines to keep Avastin available and help lobby for access to experimental treatments for severely ill patients. He and other patient advocates argue that until more is known about who will respond well to Avastin and who won't, patients likely to die soon anyway should have the option to give the drug a try.
In the meantime, he fears for patients whose insurers followed Blue Shield's lead.
"It's tough enough having the uncertainty of life and death," said Kalley, "much less having the uncertainty of wondering whether the drug you think is working for you will be available or not."