Our drug approval system is broken


Repeat after me: our drug approval system is broken. Or, if it's working right, then I don't want to see it when it really is broken. A recent example of this malfunction, which appears in today's newsletter, is the tale of wealthy political donor Fred Baron, who at death's door, managed to get a drug for which he wasn't medically qualified.

Baron, who's dying of late-stage multiple myeloma, was desperate to get his hands on Tysabri, a drug that is FDA-approved only for patients with Crohn's disease or multiple sclerosis. With only days to live, he somehow managed to wrest an approval from the FDA, even though the drug's manufacturer had not given permission (as the FDA supposedly requires). Ultimately, Baron got his dose.

This little tale highlights the craziness that exists on the fringe of the drug approval process pretty clearly. For one thing, it reminds us that while the FDA approval process was designed to protect consumers, when patients are dying there's still not a lot of room for compassion. And as Baron's case makes clear, there's clearly some political gamesmanship involved as to which dying loved one gets a drug for off-label use or access to an experimental compound.

Don't get me wrong, I understand that agencies like the FDA need to try and be consistent where hot-button issues like compassionate use of experimental drugs are concerned. And I can understand, in theory, why the agency wouldn't want people offering themselves like so many guinea pigs to try any experimental drug they can find. But the truth is, there's probably more of a middle ground than we're seeing today.

As I see it, if people desperately need an experimental drug, they should be able to get it, regardless of what part of the approval process it is in. If the choices are between certain death and possible death, shouldn't we allow them to take possible death? And what about when people who are in desperate need of drugs get rejected as test subjects? Do we really want to have to be rich and have famous people and the Mayo Clinic advocating for us to be able to get drugs that we would certainly die without?

Until there's some reform in the system, I think we can safely expect to see more and more stories like the ones we've been seeing recently: people who are dying and suffering but having to go through protracted legal battles in order to get a chance to try a drug that might save them. After all, if you're dying, and think a drug might offer you a chance at longer life, you're going to fight like hell. Wouldn't you? - Anne

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