The Magazine

Patient Power

The FDA doesn't have to decide everything.

May 8, 2006, Vol. 11, No. 32 • By JOHN E. CALFEE
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THOUSANDS OF MULTIPLE SCLEROSIS (MS) patients got an all-too-familiar message recently: Their lives are ruled by experts. The issue was their access to Tysabri, a breakthrough biotech drug with a unique ability to slow down the debilitating progression so feared by MS patients. The manufacturers pulled Tysabri from the market after two patients died from an unexpected side effect. Now the FDA has to decide whether to let the drug come back after new research has verified its potency against MS while also suggesting a 1 in 1,000 chance of the fatal side effect.

The FDA did what it usually does in these situations. It convened an advisory committee of experts. They got it half right. They unanimously recommended putting the drug back on the market. But they split on the crucial question of whether to recommend using the drug as a first-line therapy instead of waiting until patients fail on the current crop of drugs. And the committee strongly suggested that the FDA put together a potent "risk management" scheme to make sure Tysabri is used the way the FDA thinks it should be used.

Then the FDA's own experts took over. They are expected to reach a decision in the next month or so. No one thinks they will keep the drug off the market--but the FDA still has to decide which MS patients it will allow to use Tysabri and which patients it will keep from using Tysabri.

What about the patients, you ask? Well, a bunch of them testified at that advisory committee meeting, shuffling about with canes and wheelchairs, and almost all of them begged the FDA to bring Tysabri back. But as a cable news anchor said, the FDA decides on the basis of science, not emotion. Experts only, please.

In this instance, however, science doesn't have the answer. The issue, after all, is not whether the drug works or whether it sometimes kills people. It is whether the trade-off is worth it: Is a 1 in 1,000 chance of dying a risk worth running in order to reduce the much-feared "relapses" associated with MS by two-thirds or to diminish the formation of brain lesions by 83 percent? The experts on these questions are the patients. They are the only ones who can balance quality-of-life against the risk of death. Their views should be absolutely paramount.

Critics of patient power can correctly point out that people who testify at FDA hearings are not representative. MS sufferers who are happy enough without Tysabri, for example, are unlikely to travel to suburban Maryland to talk for three minutes before a panel of silent doctors and bureaucrats.

But, as it happens, we know quite a lot about what MS patients in general think of Tysabri. In addition to the usual clinical trial evidence on safety and efficacy, we have exceptionally sound survey data. In order to discover the preferences of MS patients, I worked with survey researchers at Roper Public Affairs to conduct a representative survey funded by Biogen Idec, one of the manufacturers of Tysabri. What we found is spectacular.

Roughly half of MS patients want to take their chances with Tysabri and its potentially fatal side effects. This feeling is consistent throughout the MS population regardless of level of disability. In other words, patients who are still early in the progression towards severe disability are about as interested in Tysabri as those who are already using canes and wheelchairs.

The patients also have strong feelings about FDA regulation. Seventy-one percent agreed with the statements, "If a drug has safety concerns, the FDA should warn people, but I should be free to decide with my doctor whether to use those drugs or not," and "I am capable of making my own treatment choices, based on the information and advice I get from my doctor." Patients want FDA expertise, but not FDA fiat putting valuable drugs out of reach.

This is not to say the patients want to decide everything all by themselves. Far from it. Three-fourths of our sample had seen their neurologist at least four times in the previous two years and usually talked about drug side effects. Eighty-one percent said they would be willing to visit their neurologist more often in order to get a better but riskier drug (only 2 percent disagreed; the rest agreed "somewhat").