When Patients Say: Don't Ban My Drug

Emotional lobbying can keep a treatment on the market despite serious side effects

Anyone who thinks Food & Drug Administration forums are eye-glazing recitations of scientific data should read the transcripts of the Apr. 10 hearings on silicone gel breast implants. The U.S. banned the implants for most uses in 1992, and the FDA is considering whether to allow them back on the market. An advisory panel considered the inconclusive scientific data and then listened to more than 12 hours of emotional testimony, both pro and con, from some 150 women who have tried the implants. The next day the panel members voted by a narrow 5 to 4 margin to recommend against approval.

Now the FDA must weigh in with a final decision. Dr. Caroline A. Glicksman, a plastic surgeon in Sea Girt, N.J. who wants the ban lifted, called on the agency to base its decision "on facts, not politics and emotion." In an ideal world that might happen. But outspoken patients can be a potent force, heavily influencing whether a drug or medical device stays or is pulled from the market. Hundreds or even thousands of individuals frequently lobby the FDA for a given product -- even one with life-threatening side effects -- because they believe it is the only thing that works for them.

That puts the agency in a difficult spot. Although it's under pressure from Congress to be more diligent about policing serious drug side effects, the FDA is also beseiged by patients eager to take such risks. Besides visits from silicone-implant proponents, FDA panels have recently heard from patients who want Cox-2 arthritis drugs such as Celebrex or Vioxx, which can hike the risk of heart disease, because they think the drugs are the only way to ease their pain. Others want access to AstraZeneca PLC' (AZN )s lung-cancer drug, Iressa, believing it will shrink their tumors, though it has limited benefit and is linked to a deadly lung infection.

AT ODDS WITH THE WATCHDOGS

FDA advisers who listen to such pleas say they usually give more weight to the data. Still, notes Dr. Alastair J.J. Wood, associate dean at Vanderbilt University School of Medicine in Nashville: "Patient testimony can really be quite moving." Wood chaired the FDA panel that considered whether the Cox-2 drugs Bextra, Vioxx, and Celebrex should be pulled. The panel recommended that they be left on the market, but with much stronger warning labels -- a common way of balancing safety and patient demands. Only Pfiizer Inc.'s (PFE ) Bextra was pulled from pharmacy shelves, on Apr. 7.

Patients keep lobbying, meanwhile, because they know it can work. The classic case is GlaxoSmithKline PLC's (GSK ) Lotronex, for irritable bowel syndrome (IBS), a painful but non-life-threatening condition. The drug was suspended in 2000 when it was linked to a gastrointestinal illness that killed five people. After receiving thousands of letters from patients demanding Lotronex' return, the FDA allowed it in 2002, albeit with many restrictions. "It was clear from the letters we got that Lotronex was life-altering for these patients," says Theresa A. Toigo, director of the FDA's Office of Special Health Issues.

Such lobbying often puts patients at odds with watchdog organizations meant to look after their interests. The consumer group Public Citizen labeled the Lotronex decision "a serious public health mistake," and it is just as opposed to the Cox-2 drugs, Iressa, and silicone implants. "Patient pleas are of interest, but they have to be put in the perspective of other data," says Dr. Sidney M. Wolfe, director of Public Citizen's Health Research Group. On the other side, says Michael Paranzino, head of patient-advocacy group Psoriasis Cure Now: "I err on the side of patient empowerment."

When the data backing up a drug aren't very compelling, patient empowerment can be the deciding factor. Lung-cancer victims who testified at the initial Iressa hearings made a big impact, even though the drug shrinks tumors in only 10% of patients. Iressa was conditionally approved in 2003, but follow-up studies failed to show it extends life.

The FDA is expected to make a final decision about both Iressa and silicone implants soon. After that, patients are likely to lobby for other drugs, including Tysabri, a powerful multiple-sclerosis treatment recently suspended because three patients developed a deadly brain infection. The FDA's mailroom workers had better brace themselves for a flood of letters.

Corrections and Clarifications "When patients say: Don't ban my drug" (News: Analysis & Commentary, Apr. 25) should have noted that while the Food & Drug Administration decided that Merck & Co.'s (MRK ) Vioxx could return to the market after the company voluntarily withdrew it last September, Merck is not reintroducing the drug at this time.

By Catherine Arnst in New York

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