FDA Delay In Cancer Therapy Is Attacked
Friday, July 6, 2007
Oncologists do not usually need bodyguards when they present scientific data at a medical symposium.
But when Howard I. Scher of the Memorial Sloan-Kettering Cancer Center and Maha Hussain of the University of Michigan spoke at a recent meeting of the American Society of Clinical Oncology, they were in fear for their safety.
The two doctors have been at the center of an unusually bitter debate over an experimental therapy for prostate cancer, ever since they helped persuade the Food and Drug Administration to delay approving it, enraging both patients and investors. The first-of-its-kind therapy, called Provenge, is a "vaccine" designed to extend the lives of patients with advanced prostate cancer by stimulating their immune systems.
The debate over Provenge illustrates the highly charged atmosphere that often surrounds new treatments as the desperation of deathly ill patients increasingly converges with the high-stakes intensity of biotech investing in the anything-goes forum of the Internet. The result in this case has been anonymous threats, accusations of conflicts of interest, Capitol Hill protests, congressional lobbying and vitriolic postings on blogs, Web sites and MySpace pages.
"This case may be different and all the more controversial because it's at the intersection of patient advocacy and the nervous world of biotech investors," said Daniel P. Carpenter, who studies the politics of health care at Harvard University. "It makes for a much more volatile politics."
A panel of experts recommended in March that the FDA approve Provenge. But in May, the agency instead asked for more evidence that the vaccine works after specialists, including Scher and Hussain, questioned its effectiveness.
Scher and Hussain told the FDA that Dendreon Corp., the small Seattle biotech company that developed Provenge, submitted a study to win approval for the drug that was so small that the apparent benefit it showed could have been the result of chance.
The FDA's surprise decision unleashed a spasm of criticism by prostate-cancer patients, advocacy groups and investors in Dendreon.
After the price of Dendreon's stock quadrupled and then plummeted, irate investors wrote hundreds of letters to the FDA and Congress, posted blistering critiques in Internet chat rooms, and created Web sites and MySpace pages denouncing the FDA, Hussain and Scher. They alleged various motives for the decision, including internecine rivalries within the FDA and pressure from larger rival drug companies.
"Why else would they object?" said Ray Vestal, a Huntsville, Ala., investor. His "Approve Provenge Now" MySpace page asks, "Hey, Hey, FDA, How Many Dads Did You Kill Today?" as images of Hussain and Scher flash across a backdrop of crooked crosses and Mozart's "Requiem" plays. "Perhaps there's something else behind the scenes," he said.
Patients and advocacy groups, meanwhile, borrowing strategies from AIDS activists and breast-cancer advocates, mounted an orchestrated lobbying effort. They launched a letter-writing campaign and Web sites, staged a Capitol Hill rally June 4, and demanded and got a meeting with FDA Commissioner Andrew C. von Eschenbach the same day. They began lobbying to amend FDA legislation moving through Congress to allow easier access to experimental treatments.
"The prostate-cancer community has probably been awakened for the first time. We're clearly upset about what has happened," said Thomas A. Farrington of the Prostate Health Education Network, which created the ProvengeNow.org Web site. "The true victims are the prostate-cancer patients whose lives could be saved. We're talking about terminally ill men, many of whom have no other options."
Scher and Hussain, meanwhile, began receiving anonymous e-mails, phone calls and letters attacking and sometimes threatening them.
"We have been harassed and trashed for giving our opinion in an area of our expertise," Hussain said. "It's been a nightmare, to say the least."
The pair requested and received extra security at the cancer meeting in Chicago after they heard that Provenge supporters were planning a protest June 3.
"This was concerning, especially since I was scheduled for a podium presentation that day," said Hussain, who has wondered whether to continue as an FDA adviser.
"When this sort of thing interferes with my patients and/or family, I might have to say 'sorry' and pull out," she said. "Right now, I believe I have an obligation to my patients to stay and continue to do the right thing, no matter how difficult it might be."
A handful of protesters did try to enter the meeting, but they left peacefully after being turned away by guards.
Farrington and other patient advocates, and several vocal investors, including Vestal, dispute Hussain and Scher's arguments but deny any involvement in the threats.
"I don't believe terminally ill men are getting up off their beds to threaten anyone," Farrington said.
Dendreon CEO Mitchell Gold said the company has not orchestrated or financed any of the efforts by patients or investors. He also condemned the attacks.
"We don't condone those kinds of threats," he said. "But we morally support what the patients and advocates are going through and their desire to live longer and better lives."
The FDA said it continues to work with the company as it completes more research on Provenge.
"Additional scientific data is required before a definitive decision can be made," spokeswoman Kristine B. Mejia wrote in an e-mail.
The campaign for Provenge has been seized on by other groups who want to make it easier for patients to get experimental therapies.
"It's certainly glaring proof of what we've been talking about and the need for change," said Frank Burroughs of the Abigail Alliance, which advocates expanded access to developmental drugs and is suing the FDA in federal court. "What we're saying is that when you have a drug like Provenge, you should let people have access to it who have run out of options."
But that argument worries those who say the FDA needs to be more vigilant, not less, about ensuring the safety and effectiveness of new treatments to avoid another Vioxx debacle.
"I certainly understand the desperation that patients feel," said Nancy Davenport-Ennis of the National Patient Advocate Foundation. "But at the end of the day, our society is protected through the system that we have and the process that assures us that when a drug is approved by the FDA, adequate testing has been completed to assure safety and efficacy."
The Provenge controversy comes amid revelations that a widely used diabetes drug, Avandia, may increase the risk of heart attacks. That triggered a similarly intense debate, including angry criticism of cardiologist Steven Nissen of the Cleveland Clinic, who sounded the alarm about the drug.
The stakes can be especially high for small biotech companies, because their future is often riding on a handful of products -- and sometimes just one. At the same time, patient groups are vying more for scarce research dollars, experts say.
"Something has changed," said Marie Hojnacki, who studies patient advocate groups at Penn State University. "It's become an increasingly competitive atmosphere, and both patient advocacy groups and others, including pharmaceutical companies, are doing what they feel like they need to do, which means being very aggressive about getting what they want."