SILVER SPRING, MARYLAND—At an emotional hearing here today, the U.S. Food and Drug Administration (FDA) argued that a drug that it approved just 3 years ago for breast cancer, Avastin, doesn't help patients after all and should be withdrawn. It was the first day of an unprecedented 2-day hearing granted to Avastin's maker, Genentech of South San Francisco, California, giving it the chance to basically beg FDA to rescind the withdrawal decision of 6 months ago and permit Avastin to remain easily available to breast cancer patients.
Day 1 was FDA's reiterating its case against Avastin; tomorrow will be Genentech's turn to present the defense. The judge and jury is a six-member advisory committee that offers input on cancer drugs, although FDA will have the final say. Avastin is approved for a number of other cancers, so no matter what's decided, the drug will stay on the market. But being stripped of its approval for breast cancer would mean that many insurance plans likely wouldn't pay for it, and few patients can afford its annual price tag of around $90,000. Avastin is supposed to work by inhibiting the growth of tumor blood vessels.
Early this morning, picketers clustered outside FDA's White Oak Campus here, clad in pink and holding banners declaring, "I am more than a statistic." Members of the public, including breast cancer patients who were taking Avastin, had a chance to tell their stories. A handful of anti-Avastin speakers were booed.
Avastin was approved under a "fast-track" at FDA called accelerated approval. Accelerated approval can be given to drugs that treat life-threatening diseases, often cancer, and appear to help patients. Because the data underlying accelerated approval is often less compelling, Genentech had to repeat the trial that got Avastin approved in the first place, showing that its benefits outweighed its risks.
And there, FDA officials argued today, Genentech fell short. The trial that swayed FDA showed that breast cancer patients had an extra 5.5 months of "progression-free survival," time when their tumors did not grow. But two subsequent trials showed a much more modest benefit; in one trial, the advantage was less than a month. Furthermore, there were lots of side effects, including deaths FDA attributed to the drug. "The legal standard has been met" to strip Avastin of its approval, said FDA Associate Chief Counsel Abby Brandel. No trial has shown that Avastin can help breast cancer patients survive longer.
Asked by an attorney for Genentech, Paul Schmidt, whether "there are patients for whom Avastin provides meaningful benefit," FDA's answer was not encouraging. "No, we do not agree," said Patricia Keegan, who works on oncology products at FDA's Center for Drug Evaluation and Research.
But Genentech argued that FDA had never been clear about what it really wanted to see from Avastin. Why wasn't buying patients a month of progression-free survival enough? What number would be?
FDA officials didn't answer, and they admitted that they'd never said before that a month was insufficient. Avastin appears to have a tough road to climb tomorrow if it wants its blessing for breast cancer back.