March 2, 2005

Post-Vioxx Twilight Zone

Apologies to Dow Jones, but I must post (steal is such a charged word) a Wall Street Journal Editorial in its entirety today. Subscribers who want to read it legally may click here

Still don't think political frenzies have consequences? Consider the weird and troubling inversion this week regarding the promising new treatment for multiple sclerosis, Tysabri.

The biotech partners Biogen and Elan pulled their drug from the U.S. market following just two reports (including one fatality) of serious complications possibly linked to the compound. Meanwhile, officials at the FDA went out of their way to defend Tysabri. Steve Galson of the agency's Center for Drug Evaluation and Research said the FDA "continues to believe Tysabri offers great hope to MS patients." Added drug evaluator Douglas Throckmorton, "It makes meaningful differences in people's lives."

Welcome to the twilight zone of pharmaceutical development post-Vioxx, in which the natural hyper-caution of regulators is exceeded only by the hyper-caution of terrified drug makers. Perhaps Biogen and Elan possess some information that the rest of us don't. But on the face of it the Tysabri withdrawal appears to be an overreaction that will harm MS patients in the near term, and all of us who depend on a healthy drug-development culture over the long run.

Tysabri had received accelerated approval from the FDA just three months ago because clinical trials had shown it to be twice as effective as alternative therapies in preventing flare-ups of MS, which is a degenerative and eventually fatal disease. Tysabri is also easier to take than alternative treatments, and tolerated by a subset of MS patients who can't take the others at all.

But for the indefinite future everyone will have to do without because two of the thousands of patients who've received Tysabri developed a rare neurological disorder. Those two patients happened to also be on another immuno-suppressive MS treatment called Avonex. There is no reason to believe that Tysabri has caused this disorder when used alone.

There's plenty of blame to go around here, starting with the trial lawyers and their climate of fear. Congressmen who demagogue about non-existent FDA safety "lapses" aren't much better. But we're also disappointed with CEOs who imagine they're doing patients and shareholders a favor with such rash decisions. In retrospect, Merck CEO Ray Gilmartin only strengthened the hand of the lawyers by withdrawing Vioxx when the FDA would have been content with relabeling.

Biogen and Elan seem not to have learned Merck's lesson and paid for it by having $17 billion wiped off their market caps on Monday. Litigation is in fact less of a risk in the smaller MS market than it was to Merck. But there's also political risk, and the last thing Biogen and Elan probably wanted was to have their names dragged before Capitol Hill cameras by Ted Kennedy or Chuck Grassley. On the other hand, withdrawal only attracts more media attention than the more sensible move of product relabeling.

Whatever the corporate motivations, it's obvious that no one benefits from this climate of drug-development fear. Just about everyone agrees that Tysabri will return to the market at some point, to be taken by many patients whose MS will have progressed further than it would have otherwise. Meantime, another bad business precedent has been set. Sick patients everywhere should stand up and cheer for the next CEO who keeps a beneficial product on the market, despite news of a troubling side effect.

L'audace! guys, l'audace! Timidity will not help your shareholders, will not score points with regulators -- and it woun't do a lot of good for us MS patients, either!

(For those keeping score, I am taking no medication at present. My neurologist suggests that steroid treatments might help but I am overdue for a return visit.)

Pharmaceuticals Posted by John Kranz at March 2, 2005 10:57 AM