The Food & Drug Administration has come under heavy fire from many quarters in recent years for approving drugs that later proved to have serious and sometimes deadly side effects. But every drug pulled from the market has a group of advocates more dedicated even than the company that makes it: the patients who want access to a treatment, whatever the danger. In their view, the benefits far outweigh the risks.
This week, such patient-advocates can chalk up a win for their side. After listening to the tearful pleading of some two dozen multiple sclerosis sufferers, a FDA advisory panel recommended on Mar. 8 that Tysabri, a novel MS drug pulled from the market in February, 2005, be allowed to return. Sales had been halted just two months after Tysabri won FDA approval because it was linked to a rare but deadly brain infection in three patients, two of whom died (see BW Online, 3/1/05, "Biogen: How Deep a Wound Is Tysabri?").
PLEASED PATIENTS. Investors responded to the panel's action by sending shares of Tysabri's two developers, Biogen Idec (BIIB) and Elan Corp. (ELN), soaring. But patients seeking the drug had greater cause for glee. "Whaoo!!," and "GRRRRRRRREAT NEWS HERE!!!!" were typical of the e-mails that sailed back and forth between MS patients on the news.
If the FDA follows the panel's recommendation -- and it almost always does -- Tysabri could be available in a month, making it only the second drug to return to the market after being halted over safety concerns. The first one was GlaxoSmithkline PLC's (GSK) Lotronex, for irritable bowel syndrome, a painful but non life-threatening condition. That drug was suspended in 2000 after being linked to a gastrointestinal illness that killed five people. But thousands of patients sent letters demanding its return, and the FDA allowed the drug back in 2002, albeit with many restrictions.
MS patients were just as fervent -- and more desperate. Although the deadly brain infection turned up in three of 3,000 patients who took the drug after its approval -- an unusually high one-in-1,000 incidence for infection -- the patients who lobbied the FDA didn't care.
'WORTH THE RISK.' Heather Smith, a 36-year-old from South Bend, Ind., cried during her testimony before the panel on Mar. 7. Diagnosed with MS 7-1/2 years ago, she is now on full disability. Existing therapies do not work for her and she often cannot even hold her 2-1/2 year old son. She had two infusions of Tysabri when it first came on the market in Jan. 2005 and "my energy level went from basically zero to where I could do almost everything I wanted," she says. "For me, when I looked at my own risk/benefit equation, I felt the drug was worth the risk," she says.
FDA advisors who listen to such pleas say they usually give more weight to the data, but there is no question that patient testimony can be quite moving. Smith held up a photo of her son during her testimony, and when she finished, she says an FDA-affiliated doctor knelt by her chair and thanked her for testifying. "He didn't have to do that," Smith says. "I think that showed I was getting through to them."
Patients have been trying to get through to the FDA for months. Michael Barron, an MS patient from Phoenix, started a Web site to rally fellow sufferers and raised some $25,000 to help pay the expenses of patients who wanted to come to Washington to testify before the panel. Last July he and David Kaplan, a Washington-area attorney, met with two FDA officials for two hours to discuss Tysabri. "They told us they had never had direct input from an MS patient before," says Kaplan. "I think it had tremendous impact."
LOBBYING'S OTHER SIDE. Patients don't always lobby in favor of a drug, however. Some parents of children on antidepressants fervently urged the FDA to study whether the drugs can raise the risk of suicide in children. The agency confirmed that there may be a problem, and recently required that the labels of the drugs carry warnings of the risk.
The consumer advocacy group Public Citizen, which has lobbied for bans of a number of drugs over safety concerns, just called for the recall of the pain reliever Darvon, a generic that has been on the market since the 1950s. The group says the drug can cause heart problems and has been linked to accidental overdoses.
Plenty of patients are sure to disagree, however, as they have with almost every problem drug. FDA panels have listened to patients demanding Merck's arthritis drug Vioxx, pulled in 2004 because it was linked to fatal heart problems, because they feel it is the only medicine that will ease their pain. There is no shortage of women who wanted silicon breast implants back on the market, despite reports of leakage -- this for a device meant for cosmetic surgery, not disease treatment.
The FDA often tries to come down somewhere in the middle, accommodating patients by allowing the drug, but strengthening label safety warnings. In the case of Tysabri, the panel recommended that the drug be closely monitored and that all patients taking it be registered with the company. But the panel's chairman, Dr. Karl D. Kieburtz of the University of Rochester, said at the end of the two-day meeting that there will probably be additional cases of the brain infection. "It is likely there will be deaths from it," he said. Plenty of MS patients say they want to decide for themselves whether that's a risk worth taking.