A cancer drug proves its usefulness. Will the FDA now do the same?

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In the wake of last year's Vioxx panic, it would be easy to imagine that unexpected news about new drugs will be mostly bad. But two stories last week remind us why that's not the case, and that an excess of caution--whether due to regulation, litigation or fear--can itself be harmful to public health.

The first story concerns Erbitux, the ImClone cancer drug that became infamous after its initial rejection by the Food and Drug Administration in December 2001 led to an insider trading scandal that counted Martha Stewart as a victim. We said from the start that the FDA had made a bad call, and Erbitux was eventually approved to treat colon cancer.

Well, it turns out Erbitux works for other cancers too. Last Wednesday the FDA approved the drug for a new indication--head and neck cancer--based on data showing a whopping 20-month survival advantage over traditional chemotherapy alone. In fact, Erbitux represents the first significant advance for head and neck tumors since the 1950s.

That Erbitux worked in head and neck tumors was fairly obvious from early trial results even at the time it was first rejected for colon cancer. Dr. Mark Thornton, one of the FDA medical reviewers who finally helped push Erbitux through in 2004, tells us there was "extremely compelling" data on Erbitux for head and neck cancer as early as 2000. He adds that "it was hard to argue against providing it to patients" at the time it was first rejected.

Another man for whom the latest news on Erbitux is bittersweet is Frank Burroughs, who founded the Abigail Alliance for Better Access to Developmental Drugs in honor of his daughter, who died of head and neck cancer while trying to obtain Erbitux and other treatments. "This news serves as yet another reminder that the biggest 'ImClone scandal' of 2001 had nothing to do with Martha Stewart, but instead resulted from the FDA's failure to approve a drug that had been proven safe and effective for thousands of patients," the Alliance said in a statement.

In short, Erbitux is a perfect example of why it's important to get active drugs with reasonable safety profiles out before all the efficacy data is refined to the 10th decimal place, as the FDA always tries to insist. Such data are never going to be complete anyway. Think of all the new benefits that still keep being discovered for humble aspirin.

The second good-news story last week concerns Tysabri, the multiple sclerosis treatment that was voluntarily withdrawn last year (read: lawsuit panic) after three patients on the drug developed a rare neurological infection. This possible side effect wasn't entirely surprising, given that MS is a degenerative neurological disorder caused by immune system dysfunction and Tysabri works by depressing parts of the immune system. But it was also clear, as we editorialized at the time, that the patients in question had been treated with other immuno-suppressive drugs too, and that the risks of untreated or poorly treated MS probably outweighed the risks of taking Tysabri.

Well, now several new studies reported in the New England Journal of Medicine appear to support the point that Tysabri's risks are small and its potential benefits big. Tysabri appears to cut the rate of clinical relapses by 68%, and reduce by a whopping 83% the number of new or expanding brain lesions found in MS patients.

And remember that since MS is a degenerative disease there's no way to undo the damage in patients denied the right to weigh Tysabri's risks and benefits for themselves. This week an FDA panel will meet to consider Tysabri's re-introduction to the market and MS patients are expected to be there in force. "This drug worked for me, and I want to be able to have the choice to make an informed decision," said Cheryl Bloom, who also told Bloomberg news service she will be paying her own travel expenses from Idaho to have her voice heard.

A common theme in the story of these two different drugs is the issue of informed consent. Our philosophy is that patients and doctors dealing with fatal or degenerative diseases ought to have the maximum possible autonomy in treatment decisions. We now know that the FDA could have extended a lot of lives had it followed that principle with Erbitux from the start. And it has an opportunity to extend and improve many more if it decides to help facilitate Tysabri's return to the market.