Letter to the NEJM

Everyone complains that drugs are kept arbitrarily expensive by the patent regime, and that may be true, as far as it goes. The drug companies make an excellent case that the FDA regs impose massive R&D costs on them (the $1-billion drug pipeline), and they need the extensive patent protection to guarantee recoupment of those investments. If the regs were dropped, say to the level of Europe, the costs would fall as well, and the needed patent protection would fall. Detente anyone? It’s not like people are dropping dead in Europe from bad drugs (and, no, Vioxx should not have been removed from the market either).

As with justice, treatment delayed is often treatment denied. Waiting for the regulator’s approval often means treatment delayed. But only responsible, well informed people can assess the balamce of risk between treatment delayed and treatment whose effects are not fully known.

The NEJM article calls for the FDA to gather more information. That looks worth paying tax dollars for. But it also implies restricting informed choice because to make such choices might be risky. No case is esablished for such restriction.

The ancient inicial principle of Western medicine is “First, do no harm.”; but as for centuries past doctors have to weigh up whether doing nothing or doing something is least likley to do avoidable harm. The classical resolution of that dilemma is that the doctor in charge of the particular case is responsible for deciding. Our modern case for restricting that discretion is based on the realisation that it is hard to be sure that the information reaching doctors is sound and impartial. We therefore entrust regulators with the decision on whether the risk of unsound “knowledge” in the medical system outweighs the risk of absence of knowledge.

Modern economics has produced a young mountain of evidence that decisions taken with some information tend to be superior to those taken with less information. We therfore tend to support strongly the classical reliance on decision by the informed person closest to the particular case; and we are right to press regulators on the costs of absence of knowledge in the medical system, and on the benefits of free dissemination of knowledge of uncertain quality. However, we do not yet assist the regulators with any assessments of the costs (in patients avoidably harmed) of eliminating “knowledge” from the system whyich later turns out to be unsound. For the regulators, those costs are important.

Mike Huben,

it seems to me that the question is not whether there are off-label uses that do more harm than good, but whether allowing off-label uses overall does more harm than good.

Mike Huben said:

“Of course they didn’t publish the letter. It is ridiculously one-sided and doesn’t even hint at the moral hazards that such deregulation would permit.

Drugs that actually DO something also have side effects. If an off-label treatment is ineffective, then patients are being harmed by those side effects. Without large trials, it is very difficult to know whether such an off-label treatment is effective. Doctors just can’t know from their practice if a drug is effective or not, and routinely don’t even care, judging from off-label prescriptions of antibiotics for viral diseases.

If the problem is that an off-label use is thought to be effective, then the solution is to prove it. Not to guess. Not to just wing it.”

I find this post to be offensive, one sided, and inherently ideological. To argue that all human beings shall produce the same “positive” response to the drug therapy before its approved borders on INSANE!

Try telling someone with pancreatic cancer that they may not use a potential therapy because it has yet to be approved. Why are you demanding regimentation, standardization, and top down approaches to problems that are so dire? What exactly does winging it mean to someone who is soon to die from cancer? Does that not seem unethical to you Mr. Huben?

Are we really so ideologically silly to think that the government must approve all drugs in a world where millions of people die from non-treatable diseases. It seems reckless.

Mike Huben misses the point, as usual (hint: it’s on the top of his head). The point is not that nobody should test drugs. The point is that the federal government (which was granted permission to regulate drugs through section XXXYYY WAIT TO POST THIS UNTIL YOU FIND IT IN THE CONSTITUTION of the Constitution) ensures drug safety at the cost of keeping beneficial drugs off the market. That’s like ensuring automobile safety (for all those people driving on post roads) by keeping everybody off the road (which you’ll notice the DOT doesn’t do).