The FDA has recently pulled pemoline (sold in its non-generic days as Cylert) from the market, citing risks of liver toxicity. It's used for narcolepsy, ADHD, fatigue in multiple sclerosis patients, and other indications. In theory, there are several other drugs that are useful for all of these.
In practice, though, we're talking about some poorly understood CNS indications here, and the patient response to drugs of this sort is so heterogeneous that no one understands what's going on. There are people who respond to pemoline that don't respond nearly as well to anything else. And they're an unhappy bunch, because they seem to be willing to take on the liver risks in order to have a drug that works.
One such customer is Teresa Nielsen-Hayden, well-known for the Making Light blog, and this issue has been the hot topic over there, as you'd imagine. You'll notice that the linked blog post takes a rather hostile attitude toward Ralph Nader and his Public Citizen group, because they're taking credit for petitioning the FDA for the drug's removal. The Nielsen-Haydens (no fans of the Bush administration) have been furious at Nader and his people since at least the 2000 election, so this new accomplishment has understandably pushed about all their accessible buttons.
I think they've got a point. I think that if the risks of a given drug are known, that informed patients should have a right to choose that drug's benefits with its risks in mind. Pemoline has had a black-box warning on it for years now, so it's not like its risks have been hidden. Now, it's true that if such a drug remains on the market, some people are going to take it who shouldn't, black box or no black box. But I have to wonder if such people are going to find some other way to get into trouble, no matter how much concerned bureaucracies try to save them.
The situation with Accutane (isotretinoin) and pregnancy is a similar one: there's no way you can miss the fact that it should by a pregnant woman. But every year, some do, despite strenuous efforts to prevent such cases. Mind you, Nader's people have been agitating for years to get that one pulled from the market as well.
Wouldn't a similar registration and/or liver-monitoring regime work for the people who have to have pemoline? Perhaps coupled with some sort of indemnification for its manufacturers? How many more potentially useful medications could we have available under such conditions?
If any of my industry readers have suggestions on where to obtain pemoline, I'd be glad to hear them, although Teresa Nielsen-Hayden and many others would be even happier.