Calfee argues that the FDA's approach to risk management for a promising MS treatment should focus on giving patients the information they need to make informed choices, not make those choices for them.
Thousands of multiple sclerosis (MS) patients got an alltoofamiliar 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 firstline 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 marketbut the FDA still has to decide which MS patients it will allow to use Tysabri and which patients it will keep from using Tysabri...
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&150;off is worth it: Is a 1 in 1,000 chance of dying a risk worth running in order to reduce the muchfeared "relapses" associated with MS by twothirds 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 qualityoflife against the risk of death. Their views should be absolutely paramount.