Different River

”You can never step in the same river twice.” –Heraclitus

November 25, 2005

Will the FDA kill Brian White?

Filed under: — Different River @ 2:54 pm

Brian White, of Fairfax, Virginia, is hoping to survive long enough for the FDA to allow him to be treated. Here’s an excerpt of the story in The Fairfax Times:

Battling a rare disease

By Frank Mustac
11/22/1005

His breathing is shallow and deliberate, but, despite a constant struggle to fill his lungs, Brian White is determined to keep up with his young sons playing at the basketball hoop outside his home in Fairfax’s Kings Park West neighborhood.

But with each breath White takes, the slender-framed 42-year-old husband and father of two is even more determined to stay healthy long enough to receive a yet-unavailable but promising new treatment for the rare neuromuscular disorder he has.

In 2002, after two and a half years of misdiagnoses, White was finally diagnosed correctly at Johns Hopkins in Baltimore with Pompe disease, a disorder that affects only about 5,000 people in the United States.

Pompe is caused by a deficiency of an enzyme responsible for breaking down glycogen, a form of sugar stored in the muscle cells throughout the body. This buildup inside cells causes muscle fibers to expand and leak, leading to muscle weakness that becomes more severe over time and can often be fatal.

Now, along with his job as a regulatory expert for a gas pipeline company, White has kept busy as a vocal advocate, despite the sound of his voice, doing all he can as a patient to help win approval by the Food and Drug Administration for a new enzyme replacement therapy for the treatment of Pompe.

Developed by Genzyme, a Massachusetts-based company, the firm earlier this year submitted an application to the FDA for both the adult and infant therapies, White said. A response from the FDA is expected sometime next year.

What is the reason for the delay here? Every day the FDA delays action, Bryan White and 5,000 other people are one day closer to being so sick they can’t be treated. No doubt many of these people will die, untreated, waiting for FDA approval.

This is not like people who die waiting for organ transplants, or people who die waiting for a treatment to be invented. These people are dying waiting for a signature on a piece of paper.

Why is this? In 1962, Congress passed the Kefauver-Harris Amendments, which changed the Federal Food, Drug, and Cosmetic Act to prohibit the sale of any drug that had not been determined by the FDA to be “safe and effective.” The law didn’t say how the FDA was to determine that, so the FDA prescribed a long, complex, and expensive process that companies must go through before the FDA approved their drugs. Prior to 1962, the requirement was the that the FDA find the drug “safe” — and furthermore, the FDA has 180 days to act on a drug application, after which, if it was not unsafe, it could be sold. The 1962 amendments removed the 180-day limit, added the “effective” requirement, and set the stage for thousands of people to die while the FDA spends years processing each new drug application. The FDA’s own history of itself calls this, “a milestone advance in medical history.”

One can only ask what kind of a “milestone” this is when applied to a treatment for a fatal disease for which no other treatment is available. What is “effective”? Without treatment, the patient dies. There is no treatment other than the one current candidate. How could any treatment be less effective than this? If the treatment saves 5% of the patients, is that “effective”? Surely not, by the FDA’s standards — nor anyone else’s. But if the alternative is to save 0% of the patients by having no treatment available, should the FDA stand in the way?

And what about safety? Again, without treatment, the patient dies. What treatment could be less safe than that? Perhaps they will die faster — but this is the sort of thing that can be ruled out fairly easily in animal tests, and anyway the safety hurdle is not what holds up most drug applications; it’s the efficacy requirement. Indeed, the Genzyme treatment has already passed the FDA’s initial safety hurdle, since recruiting for clinical trials is already underway.

If the FDA were operating under the pre-1962 rules, Brian White and many of the 5,000 other people with Pompe disease would be receiving treatment, rather than fighting to be among the few included in clinical trials.

But they can’t do that; they’ve been forced to become lobbyists:

Back in September, White and four other Pompe patients he met through Internet support groups and on Web sites containing detailed information about the disease, spoke with FDA officials to convey the severity of the ailment from a patient perspective. He also appeared in a recently completed video of Pompe patient testimonials.

White said he hopes the FDA will convene a patient advisory committee during the approval process and that members of Congress will use whatever influence they have with the agency to win approval.

As time goes on, White said his advocacy work has become more important, primarily because of his concern for others with Pompe in worse shape than him.

“Just that hope of a better chance is something we’d all jump on,” he said.

Just the hope of a better chance — a hope that, under current regulations, depends on the whims of members of Congress to use “whatever influence they have” with an overloaded agency. And all they are being asked to do is remove a barrier to treatment that they themselves — both Congress and that agency — have erected.

I’m sure most of the FDA’s employees are hard-working scientists, doctors, and bureaucrats who do the best they can with what they’ve got. But these are human beings we’re talking about. In addition to processing millions of pages of material for every new drug application, they have their own lives to live — they have to get their oil changed, mow their lawn, drive their kids’ carpools, take out the garbage, and do all sorts of other things that will not be the impacted in the least if some patient they never heard of dies because some piece of paper is sitting on their desks.

The problem is the fact that Congress and some bureaucrats decided a long time ago that people ought to die if those papers are still on those desks.

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