April 7, 2006; Page A12

"Every day I'm a little bit worse," Audrey Greenfield tells us. The former New York law partner used to travel the country representing real estate developers. But the multiple sclerosis that she first noticed 15 years ago when she lost coordination in her right foot has now progressed to the point she needs a wheelchair. And she isn't comforted by the fact that she's being denied an effective treatment by the Food and Drug Administration, which is once again harming patients with overcaution.

The therapy in question is Tysabri, and for many MS patients it appears to halt progression of the degenerative neurological disorder. But a year ago -- just months after it was approved -- corporate partners Biogen and Elan "voluntarily" withdrew their drug because of FDA pressure and litigation fear after two patients developed a rare brain infection. That infection might have been linked to Tysabri, since the drug works by suppressing parts of the immune system. But these patients also had other immuno-suppressive therapies, and in any case the MS patients were almost all willing to run the risk.

Just a few weeks ago prospects were looking better for Ms. Greenfield. She and scores of other MS patients had made their voices heard at an FDA advisory panel, which voted unanimously to return Tysabri to the market. The agency was due to rule on the issue by late last month.

But shortly before the deadline, the FDA announced it would take a 90-day extension until the end of June because of concerns over Tysabri's "risk management" plan -- a preposterous excuse given that Tysabri is delivered by injection and only under the supervision of doctors. With its delay, the FDA is denying very-well-informed doctors and patients the freedom to "manage" the risks of untreated MS. "I haven't had any treatment in over a year," says Ms. Greenfield, who got one injection of Tysabri before it was withdrawn.

Bartira Tibertius of Chicago faces the same predicament. She received Tysabri for a full 28 months as part of a clinical trial. "I was doing great. I even forgot that I was sick," Ms. Tibertius, a language teacher and translator, tells us. "But now I'm getting very, very scared. It's deteriorating and I know that," describing numbness and tingling in her hands, arms, feet and face. As for any possible risks from the drug, she says, "I'm more scared of not having Tysabri than having Tysabri. If you told me the Tysabri would shorten 10 years of my life I would do it. I want quality of life, not quantity."

Her risk-benefit analysis is backed by the latest scientific evidence. Several recent studies reported in the New England Journal of Medicine conclude that, among other benefits, Tysabri appears to cut the number of new or expanding brain lesions in MS patients by 83%, and for many induces near-complete remission. Yet the FDA was unable to get its act together and meet its March deadline. It's still likely that Tysabri will return to the market, but the agency's apparent nonchalance in the face of progressive neurological damage is a sad spectacle.

One lesson is that Tysabri should never have been withdrawn in the first place, when a "Dear Doctor" warning letter would have sufficed. But the possible side effects came to light at the height of the Vioxx panic (another relatively small risk, by the way), and then-acting FDA chief Lester Crawford wasn't going to take any difficult stands while he was awaiting confirmation. We hope Andrew von Eschenbach, the latest nominee to head the FDA, won't follow Mr. Crawford's lead. The FDA badly needs public leadership to explain that almost all live-saving and life-improving drugs do have some risks.

The Tysabri story is also further evidence of the need for Congress to pass "Kianna's Law" -- legislation introduced by Senator Sam Brownback (R., Kan.) that would require the FDA to move faster on drugs for terminal and degenerative disease. The tendency of FDA bureaucrats is always to delay decisions on drugs that might turn out to have some nasty side effects. But as Audrey Greenfield, Bartira Tibertius and thousands of other MS patients have had to learn over the past year, too much caution can have nasty side effects too.

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4 Comments:

This really is analagous to the notion of informed consent to surgery. Bad things happen in a small percentage of operations, but people give informed consent to the procedure having been informed of the risks.

Why not have Tysabri users sign a similar form, and waive certain tort rights they might otherwise have pursued?

By Cardinalpark, at Fri Apr 07, 10:12:46 AM

I can tell you why: because some clever attorney will allege their "consent" was not really informed or was made under duress or strain of illness.

This is what aggravates me so about the legal profession: we have made it so easy to sue that the ease of litigation is now backfiring on plaintiffs.

It is madness. Adults should be presumed to be capable and informed unless there is some pretty damned strong evidence otherwise: the BOP should be on the plaintiff in this case to prove why their "consent" was not informed or voluntary or why they were incompetent, and that ought to be a very, very steep evidentiary mountain to climb.

Otherwise, medical decisions are taken out of the hands of patients and their families and placed in the hands of government. Do we really want that? I know I don't.

By Cassandra, at Fri Apr 07, 10:20:54 AM

...almost all life-saving and life-improving drugs do have some risks.

Exactly. I doubt there is a drug that is absolutely safe in all instances. Aspirin would be considered a wonder drug if introduced today, and yet one wonders what the FDA would make of its link to Reye syndrome or its tendency to promote bleeding.

Using a drug provides benefit in the face of some risk. We accept the risk for the benefit. The choice of treatment should be left to doctors and patients, not to the vagaries of bureacratic meddling.

http://tigerhawk.blogspot.com/2006/04/fda-tysabri-conundrum-and-our-cultural.html