Supremes tell Abigail no: why this is a good thing

Earlier this week, the US Supreme Court declined to hear the appeal of the Abigail Alliance--news from the LA Times here. The advocacy organization sought to change the Food and Drug Administration's policy of prohibiting the use of experimental and unapproved drugs to treat patients. While there are procedures on the books for compassionate use, which allows access for some patients to some drugs under some circumstances, the Abigail Alliance and its supporters believe that the current approach is too narrow, too onerous, and too time-consuming to help all those who could benefit. They sought to make access easier for terminally ill patients who are unable or ineligible to participate in clinical trials.

The Abigail Alliance makes a stirring argument: none of us would want to feel that we were unable to get access to the "silver bullet" that would make our loved one (or ourselves) well again or extend life because of some bureaucratic requirement. And certainly the story of Abigail Burroughs--by all accounts a lovely young woman, who died of a rare form of cancer when she was just 21--is terribly sad.

But does it mean FDA is wrong to deny access to unproven drugs? I don't think so. The FDA's mission is to ensure that the medicines and devices we use to improve human health are safe and effective. This is a vital function and one that must be preserved.

We--by which I mean the general public--have a tendency to assume that drugs are good, that they work, and that they generally don't make people sicker. This is not a great assumption: check out the news earlier this week about Zetia and Vytorin (cholesterol drugs that could actually increase atherosclerosis), or the Vioxx settlement (a pain-reliever that caused heart attacks and strokes in some patients), or reports that suggest that several common drugs for schizophrenia may increase diabetes risk, or fen-phen (an off-label use for weight loss caused permanent heart damage in some patients). These are just a few examples. Drugs can be dangerous. They can make people sicker, and in some cases, they can give people worse problems than they started out with.

"Aha!" the advocates say, "But what about if somebody is already dying? They're as sick as they're going to get. Surely they should be granted access!" Still, in general, I think not. Patients who are terminally ill are the very last people we should be treating with unproven agents. Clinical trials aren't all beer and skittles: If you doubt that drugs can be dangerous, check out reports of the TeGenero trials last summer, in which 6 previously healthy young men ended up in the ICU with permanent immune system damage. These were Phase I safety trials, not Phase II--the category the Alliance wants to make available--but my point is, should we really subject patients to such an ordeal in the pursuit of what is likely an understandable, but unrealizable, dream of a cure?

A key point to understand is that research is different from treatment. Research is directed toward the development of new solutions, and it may or may not benefit those individuals who volunteer to participate. It sounds harsh, but helping volunteers isn't the point of research. Treatment, on the other hand, is all about managing symptoms and effecting cures. It's just plain wrong to represent one as the other.

The legal questions are more subtle: argument in the original case focused on whether terminally ill patients have a Constitutional right to drugs that have passed Phase I clinical testing. Our very own Linda Glenn did a nice overview here.