Forbes has an article on some recent work of Paul Rubin, an economist at Emory. He's looking at the situation in approvals of new antibiotic drugs, which isn't an encouraging sight.
He's of the opinion that too much government effort has gone into cutting overuse of the existing drugs (to try to slow down the development of resistant bacteria) and not enough has been done to make new drugs easier to bring to market. The use of antibiotics in general is being discouraged, and at the same time the FDA has made the regulatory environment for new submissions tougher. A quote:
". . .the FDA policy of requiring additional testing for antibiotics is a fairly bizarre policy and makes no sense. . .A much more cost-effective alternative would be to approve the drug in the normal manner (or even provide an accelerated approval) and spend additional resources on...post-approval surveillance."
He's probably right about this, but (and here's the usual problem) it makes sense only if you ignore the tort lawyers. If your new antibiotic goes out and causes trouble in some subset of the patient population, it's no use telling the attorneys that, hey, the FDA approved it. They're not going to get money out of the FDA; they're going to get it out of you. Nope, it was your willful, stupid, perverse, dare I say evil negligence that led to this completely avoidable tragedy, and. . .aargh, you can write the rest as well as I can.
That's the thing: the FDA requires that we show safety and efficacy. We can prove the presence of efficacy, but safety is merely the absence of harm. No one can prove a trial lawyer's definition of safety. A clinical trial can tell us that in the population that participated in the trial, X adverse events took place. Whether X is a greater or lesser number than we'd expect in the general population is a question that can be answered statistically, but whether our drug caused those X events isn't a question that can usually be answered at all.
In such cases, our best chance is to see if the affected patients had something in common, or if the problem increased in proportion to the dose. Often enough, neither is the case - does that make the compound safe, or not? Even if there weren't any signs during the trials, what will happen when our drugs hit the orders-of-magnitude larger population of paying customers? We don't know. We can rule out what our clinical trials were powerful enough to see, but we will never see the one-in-fifty-thousand kinds of trouble. Not until the lawsuits start flying.
There's yet another problem with Rubin's argument, scientific rather than regulatory, which I'll address tomorrow. . .