Cancer Drug for MS Brings Up More Thoughts on Off Label Prescribing

The other day, to the consternation of some, I discussed my opposition to "off label" prescribing, meaning when a drug is approved for use to treat one malady, it is prescribed for a different one even though the medication was not specifically tested for that circumstance. To me, off label prescribing is a form of human experimentation that should either not be allowed or which should be thoroughly disclosed to the patient at the time of prescribing--with potential civil liability if things go wrong.

That is not to say that when a drug is found to have potential off label uses, it should just be ignored. The proper approach, it seems to me, is to alert the medical community and FDA, and then test it properly for the new use. And that is exactly what has been done with a cancer drug that may be efficacious for treating MS. From the story:

The blockbuster cancer drug Rituxan may help treat multiple sclerosis, according to the results of a small clinical trial that opens up a broad new approach to understanding and possibly treating the disabling disease.

The study encouraged Rituxan's co-developers Genentech Inc. and Biogen-Idec, which hope larger trials will establish the drug as an approved treatment for the disabling neurological disorder. But the trial's more sweeping impact may come from the light it sheds on the mechanisms of the disease.

Such off label trials would move much faster than the original testing process because so much would already be known about the drug's working and risks. This is the proper way to go.

Botox Story Illustrates Danger of Off Label Prescribing

One of my pet peeves--yes, I know, I keep peeves like a crazy old man who keeps hundreds of cats--is off label prescribing. Off label prescribing allows a drug that has been approved for efficacy and safety for one condition, to be used instead to treat a different malady for which it has never been adequately tested. I think it should be illegal as a form of human experimentation, or at the very least, patients should be told as part of informed consent that the treatment is not specifically FDA approved to treat their condition.

Admittedly, some good has come from this. But also some evil, such as this story (perhaps) illustrates of deaths associated with Botox, the drug used in cosmetic procedures to erase wrinkles:

In a public alert issued Friday, the Food and Drug Administration said Botox, along with a similar drug called Myobloc, has been linked to life-threatening symptoms such as strained breathing and severe difficulty in swallowing, which can lead to a form of pneumonia. The FDA is advising doctors to monitor patients for such reactions while it decides whether to strengthen warnings on the drugs' labels.

Many of the most serious reactions--deaths and hospitalizations--occurred among children treated for cerebral palsy-associated limb spasticity, the agency said. The drugs are not FDA-approved for that use in children or adults.

FDA-approved drugs often have off-label uses, where physicians take medications approved for one disease to treat another. This practice often benefits patients and drug manufacturers, but can increase risks...

Botox and Myobloc are each forms of a toxin produced by bacteria that can paralyze muscles and lead to botulism, a fatal food poisoning. But in small amounts, the injected toxins can calm muscle spasms. A third product, Botox Cosmetic, is FDA-approved to improve the appearance of wrinkles between the eyebrows.

I have always been dubious of purely cosmetic procedures figuring that even an infinitesimal risk of harm isn't worth the false look of youth. In any event, the propriety of off label prescribing needs some pondering.