Three people, Jim Glass, Dan Klein, and db, made important comments on my FDA post yesterday.

First, Jim Glass:

How the patent system and the FDA discourage study of cancer prevention (quoting…)

“R & D on cancer prevention and treatment of early-stage cancer is very socially valuable,” the authors told me in an email, “yet our work shows that society provides private firms — perhaps inadvertently — with surprisingly few incentives to conduct this kind of research.”

To secure F.D.A. approval, after patenting a drug, drug companies race the clock to show that their product is safe and effective. The more quickly they can complete those studies, the longer they have until the patent runs out, which is the period of time during which profit margins are highest. Developing drugs to treat late-stage disease is usually much faster than developing drugs to treat early-stage disease or prevention, because late-stage disease is aggressive and progresses rapidly. This allows companies to see results in clinical trials more quickly, even if those results are only small improvements in survival.

This very lesson is taught in some medicinal chemistry textbooks. For instance, one notes that “some compounds are never developed [into drugs] because the patent protected production time available to recoup the cost of development is too short.”…

Between 1973 and 2011 … there were over 17,000 trials of patients with the lowest chance of survival (those with recurrent cancers) but only 500 for cancer prevention, which confer the longest survival gains….

I was unfamiliar with the article that Jim Glass links to–and I shouldn’t have been.

Now Dan Klein:

Nice post. One comment: The focus of the NYT article is on shaving months off the decision on a new drug application. But the real issue is the extremely protracted many-years-long process of testing that precedes even submitting the application, a process ruled over by the FDA.

It’s great that Pazdur shaves a month or two off the final step, but the real issue is the regimented system of requirements preceding the application.

Debating the speed with which the FDA decides new drug applications is a bit like debating how quickly a state Bar examination is graded.

Good point. I was aware of this when I read the NYT piece, noting that the time has been cut by one month. Of course my focus was on the motivation of Dr. Pazdur and then, given that, on whether there is a better way.

But Dan’s point is important. Simply by going back to pre-1962 law and not requiring a test for efficacy in particular uses would shave not months, but years, off the process. And Dan’s analogy with grading the state bar exam is spot on.

Now db, making the point that there is precedent for my proposal in federal regulation of another potentially dangerous product:

This would be similar (but on a much larger scale than) the regulations allowing for homebuilt aircraft. The FAA regulations allow for an aircraft to be built for personal use and educational/recreational uses by a non-certified builder. There is a thriving industry in building components and equipment for such aircraft. The whole thing is predicated on the idea of placing the risk on the builder/pilot/passengers. Indeed, the regulations require that all such aircraft be marked “EXPERIMENTAL” and have a placard visible to passengers that reads to the effect of “THIS AIRCRAFT HAS NOT BEEN CERTIFICATED BY THE FAA AND DOES NOT MEET REGULATORY STANDARDS FOR CERTIFICATED AIRCRAFT”

Often, builders suffix the required language with “…IT EXCEEDS THEM.”

Professional aircraft mechanics can work on such aircraft without endangering their certificates or taking on any more liability than they would working on a certificated aircraft. This would provide an analog to licensed doctors experimenting with unapproved drugs.

It may be wise to set the liability standards a bit higher for medical experimentation, but there is a precedent in federal regulations for allowing this type of action in an otherwise extremely overregulated industry.