Off-label drugs

Alex has written much about the importance of off-label uses for drugs, which are not generally restricted by the FDA. I came across the following in the 17 March 2004 Wall Street Journal, Marketplace section:

A high-price biotech drug, developed in the 1980s to treat a rare form of hemophilia, is fast becoming a blockbuster, with physicians around the world using it to stanch severe bleeding from car accidents, gunshot wounds and postsurgery hemorrhaging.

But here’s the rub: The drug, a human bloodclotting protein called NovoSeven that costs $5,000 a dose, hasn’t been approved by the Food and Drug Administration for such uses…Some doctors are hailing NovoSeven as a lifesaver, with word spreading about near-miraculous cures of dying patients. It’s the new wonder drug,” says Thomas Scalea, director of the shock trauma center at the University of Maryland Medical Center. He says the center has used the drug 80 times in three years, saving about 35 lives.

Not all doctors agree about the merits of the drug, and insurance companies will not reimburse drug usage for this purpose.

Question: Should the FDA have regulatory power over the off-label use of this drug to stop traumatic bleeding? If it had had such power, would any of these lives have been saved at all? In his paper, Alex asks the requisite follow-up question. If it is bad idea to give the FDA regulatory power over off-label uses why give it so much power over initial uses?

As things stand now, the FDA will not allow “human trials” of the drug in the laboratory, although real life trials occur in hospitals on a regular basis. Furthermore the company’s sales force cannot promote the drug for non-hemophilia uses and must wait for doctors to ask, noting that it employs 25 full-time people simply to handle the flood of inquiries.

Further comment by Alex is welcome. This is his bailiwick not mine, I just thought the example was especially striking. Here is some basic information about the drug, here is the company’s web site.


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