Which way society tips may impact the lives of untold numbers of individual patients and their families as well as the sales, marketing and, potentially, development of many cutting-edge pharmaceuticals.
At the center of the controversy this week is Avastin, a drug used with mixed results by metastatic breast-cancer patients. A Food and Drug Administration (FDA) panel is considering whether to continue to approve the drug's use for breast cancer treatment. Its decision, yay or nay, will have no effect on a doctor's ability to prescribe the drug but it will have a huge impact on who can afford to buy it. If the panel rules against Roche, in a vote scheduled for today, Medicare and private insurers could stop reimbursing patients for treatments that cost about $88,000 annually. FDA Commissioner Margaret Hamburg, however, will have the final say.
"The proceedings emphasized a conflict that has bedeviled pharmaceutical regulation and other efforts to control the practice of medicine -- one between cold statistics from clinical trials about overall populations and the often emotional experiences of individual patients who say a therapy has, or might, work for them," writes Andrew Pollack in the New York Times.
"I never thought that in the U.S., I would have to beg for a drug that's keeping me alive," Patricia Howard testified yesterday. "What if I was your wife? What if I was your grammy?" Pointing out that five of the six panel members are male, Time's Melinda Henneberger writes that they all "looked elsewhere as she spoke." Henneberger, herself a breast-cancer survivor, continues:
"A man pleaded, 'Please just leave my dear wife alone.' His mic was switched off as he said, 'This hearing is a death trial.' His allotted three minutes were up, but as a metaphor, the locked-in time limit did not work in the agency's favor."
Avastin is approved commonly used to treat colon, lung, brain and kidney cancers but FDA officials cite studies that show the drug delays tumor growth only for a month or so in most women with metastatic disease, doesn't extend lifespan and can have serious side effects. The FDA's Center for Drug Evaluation and Research recommended rescinding Avastin's approval for breast cancer in December but, aware of the potential backlash of the decision, agreed to the unprecedented two-day open appeal that's underway.
While most of the testimony yesterday was in favor of Avastin, not all was, Alicia Mundy reports in the Wall Street Journal. "The women who didn't do well taking this drug can't testify today," said Helen Schiff, who reported that an acquaintance who took Avastin suffered a hemorrhage and "was bleeding from every orifice in her body," and died about a month later.
Christine Brunswick, vp of the National Breast Cancer Coalition, who herself has metastatic breast cancer, testified that studies have not proven that the drug is safe or effective as a treatment for the disease, reports Brenda Goodman of WebMD Health News. "We must accept and act on evidence or we will never make the needed progress we so desperately want," she said. "We now know that women died because of this drug."
"Emotions reigned ... ," writes Mundy, "with tearful stories, rowdy protests and some booing of witnesses." She also quotes a spokesman for Roche's Genentech unit: "These examples are important for people to know about, but you also saw examples this morning of women who are living." Ed Lang also said one study suggests Avastin may delay tumor growth for five months or more.
Another implication for all pharmaceutical marketers in the Avastin case is the future of the FDA's accelerated approval program. "The program is designed to get new medicines on the market faster," Mundy writes, "but the FDA is concerned that it will have trouble revoking the approvals if later data don't support the drugs."
In a front-page story on Monday, The New York Times' Pollack wrote about the controversy surrounding three new drugs -- Dendreon's Provenge, Johnson & Johnson's Zytiga and Sanofi's Jevtana -- that have demonstrated in clinical trials that they could "prolong the lives and relieve the symptoms of men with advanced prostate cancer, but could also add billions of dollars to the nation's medical bills."
"What a great time it is in prostate cancer," said Dr. Daniel J. George of the Duke Cancer Institute at a medical conference recently. "And it's a great time for the drug makers," writes Pollack, pointing out that there are potentially billions of dollars to be made.
Although "cost-containment' is not supposed to factor into the FDA's consideration of whether to approve a drug, administrators have ears. "The price of these drugs has already stirred concerns about the costs of care among patients, providers and insurers," writes Pollack. "For example, Provenge costs $93,000 for a course of treatment, while Zytiga costs about $5,000 a month."
Back at the Avastin hearings, Time's Henneberger points out that the Obama administration will be accused of putting economics above compassion if the FDA denies Roche's appeals.
"I covered [commissioner] Hamburg when she was New York City's Health Commissioner in the mid-1990s, and put the likelihood that she would decide on that basis at zero, give or take," she writes. "She won't be swayed by emotional appeals from survivors, either, myself included; her job is to evaluate the science. But can 17,500 women really be anecdotal?"