Food and Drug Administration

While I’m talking about changes to cherished institutions, let’s talk about the FDA.  There needs to be some control regarding drug distribution and claims about drugs, but I’m far from convinced that the FDA’s current approach is anything near the optimal approach.  Mark A.R. Kleiman (a liberal I respect but often disagree with) is apparently thinking along the same lines.  In a thought provoking post  he suggests a number of changes to the structure of the FDA. 

I do of course suggest that you read it all, but here is an interesting passage:

In the wake of the Vioxx/Celebrex flap, there's been lots of finger-pointing, but it seems to me not enough consideration of what principles ought to guide policy when it comes to approving new drugs or ordering existing drugs off the market.

Given patient-to-patient variability, the idea that a drug is "safe and efficacious" (or not) generically can't really be right. What if a drug is helpful to some patients and harmful to others and the difference between the two groups is observable, either in advance or by monitoring the patient during a trial period? There are almost certainly patients who, given the alternatives, would be better off staying on Celebrex rather than switching to the older generation of NSAIDs. But there's no guarantee that those are the patients who will actually be continued on the drug if it isn't yanked from the market.


Some know much more than others about pharmacology generally, or about a specific drug or class of drugs. While it makes sense to have a regulator in the middle to protect the less-knowledgeable from the persuasive powers of the drug companies, it make no sense to overrule the decisions of doctors and patients who may be as knowledgeable about a drug as the FDA regulators themselves, and are certainly more knowledgeable about the details of a particular case.

The SEC came up with a fix for the analogous problem in capital markets: the "sophisticated investor" rule. In the securities case, "sophistication" means no more than deep pockets and experience in high-risk investing. But in the case of drugs, why not craft categories of "sophisticated" doctors and patients who could, by passing examinations, win for themselves the right to prescribe, or to use, drugs not yet approved for marketing to the general public?

It would be reasonable to require, as a condition of participation in the program, agreement to collect and submit appropriate information about the use of the drug and its outcomes to the drug-review process. That would somewhat compensate for what might otherwise be the reduced pressure to participate in clinical trials.

Of course, mistakes would continue to be made. But is there any reason to think that, given the option of using non-approved drugs, truly knowledgeable physicians and patients would in fact make choices that led to worse outcomes rather than better ones? None that I can think of.

This is a key insight.  Americans have a funny idea about risk.  The same people who worry about getting cancer from high tension lines drive every day in their car.  Getting electricity and using personal transportation are both very useful facets of modern life.  For some reason people are willing to focus on the super-tiny risk (if it exists at all, studies seem to show that it is below the statistical significance threshold) of living near high tension wires, while they are willing to bear the much larger risk of driving to the mall.  (Please don't draw parallels between this and terrorist risks.  There are arguments to be made about intentional violent acts and societal trust that I just don't want to get sidetracked with in this post.)  Back to medicine.  Many people seem to have developed the strange idea that medicine is or ought to be a risk free endeavour.  We've come a long way, but we are nowhere near a deep enough understanding of individuals' bodies to be able to realisitically think of advanced medicine in that way.  It would be helpful to engage in a societal discussion of useful risk taking with respect to medicine.  It might be good to allow informed patients and informed doctors to take more risks if tailored to the specific patient.  Many risks might be very manageable if they were well monitored during treatment.  It is commonly said that aspirin wouldn't make it past the FDA nowadays.  But aspirin turns out to be an extremely useful drug, with real but manageable side-effects.  It isn't reccommended for some people, but for many it works very well. 

There used to be an old tort principle called assumption of risk.  It used to relieve manufacturers and other potential defendants of liability if the damaged party engaged in certain activity despite a risk that they could see.  A policy somewhat informed by a similar notion might be very useful in the drug arena.  We could get a lot more drugs out their which could help quite a few people, if we didn't have unrealistic expectations about drug safety and if we were better able to manage the fact that some drugs are safe and effective for some people even if they aren't safe and effective for everyone. 

A further comment about this post appears in Lines in the Sand:

After basic testing for general safety, the FDA could issue a provisional approval that pharmacists would have to stay up on to remain certified.  That would allow the FDA apply even more rigorous testing for final approval without delaying availability, and could provide a larger statistical base for that testing.

I agree with Kleiman that the FDA is a great place to collect statistics and do testing, but giving them absolute authority for approval has questionable value.  This approach moves the prescription decision closer to the the patient's unique situation without resorting to drug by drug certification.

I agree that could be a useful way of looking at it. 

Posted by Sebastian Holsclaw at January 6, 2005 3:22 AM