When I was at the FDA, we made a conscious effort to stop referring to “compassionate use” because it made it sound like “noblesse oblige.” We began to call it what it should be – expanded access.
In a house editorial, the New York Times misses the mark not only on this point (where they refer to “humanitarian use” – a nonsensical misnomer), but also as it relates to “comparative effectiveness.”
The Gray Lady opines, “This case raises the question of whether the F.D.A. should demand more rigorous trials before a device is granted a humanitarian exemption. It clearly shows the value of conducting rigorous controlled studies with enough patients to provide meaningful results. This is just the kind of comparative effectiveness research that the national health care reforms seek to promote.”
Well, yes and no.
Sure, the FDA should always strive to better understand where expanded access programs may lead. (And, it’s important to note, many patient groups – such as the Abigail Alliance – believe the FDA is already too slow and stingy with such protocols.) But even the best folks at the FDA are only so prescient. I think it wise to give the folks at White Oak the benefit of the doubt. Adoption of the Precautionary Principle (where nothing is done until everything is known) only leads to nothing being done and the death of innovation.
And as far as the “comparative effectiveness” statement is concerned, this situation has nothing to do with it whatsoever. A larger scale trial of these stents uncovered the safety problem -- precisely what such trials are designed to do. It has nothing to do with "comparative effectiveness." When it comes to the FDA -- it's about safety and efficacy.
But when you’ve got a hammer, every problem looks like a nail.
