Concluding a rare, two-day hearing, an FDA panel has voted 6-to-0 to revoke the breast cancer indication for Avastin, dealing a huge blow to Roche and its Genentech unit, which now stand to lose an estimated $1 billion in annual sales. The drugmaker had appealed a decision last December by the agency to yank the indication, prompting a heated debate over the veracity of the FDA accelerated approval program.
A final decision, however, rests with FDA commish Margaret Hamburg. Meanwhile, the FDA and Roche's Genentech unit will provide additional written submissions by July 28, and the docket will remain open for public comment until then (see this).
At the conclusion of the meeting, some patients were particularly vocal. “What do you want us to take!? We have nothing else!” shouted Christi Turnage, of Madison, Mississippi, whose cancer has been undetectable for more than two years since starting therapy with Avastin. Steve Walker of the Abigail Alliance, which lobbies for patient access to experimental medicine, tells the Associated Press the hearing was a "kangaroo court. There wasn’t one dissenting thought up there, let alone one dissenting vote.”
In explaining their initial decision, FDA officials cited the results of clinical studies and determined the data indicate Avastin does not prolong overall survival in breast cancer patients or provide a sufficient benefit in slowing disease progression to outweigh significant risks. These include severe high blood pressure; bleeding and hemorrhage; development of perforations in the body, including in the nose, stomach, and intestines; swelling of the brain, and heart attack or heart failure (read the memo).
Since then, the accelerated approval program has been scrutinized, especially as industry execs and some members of Congress say the FDA is too strict. Many patients and their families also complained that a useful treatment would be unfairly removed (look here). However, others have pointed out that FDA credibility is at stake and the accelerated approval program would not be effective if the FDA is unwilling to act in the face of evidence that an indication is no longer warranted (see this).
Recently, the FDA’s Oncologic Drugs Advisory Committee urged the FDA to require drugmakers to submit at least one controlled trial for approval for their cancer meds. That’s because more than half of the drugs passing through accelerated approval do so by relying on a single-arm trial. And the FDA panel was concerned that drugmakers are failing to conduct required post-marketing studies to verify surrogate endpoints on a timely basis, undermining the accelerated approval program (read this).
In a statement, Genentech says: "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. We remain ready to collaborate with the FDA to find a solution that is in the best interest of patients who need Avastin."
Avastin, of course, remains available for treating bowel, lung, brain and kidney cancers. And docs can continue to prescribe the med for breast cancer, but insurers are unlikely to pay $50,000 to $100,000 a year. The Centers for Medicare & Medicaid Services, meanwhile, will not act on coverage until the appeals process has played out, although many insurers are expected to drop coverage.