I was stunned when I saw this headline in the New York Times last week: “Court sides against FDA in ‘off-label’ drug promotion case.” In case you missed it, here is the lede:
The maker of a prescription fish-oil pill won an early victory Friday against the Food and Drug Administration over its right to publicize unapproved uses of its drug.
The gist of the story is that the pharmaceutical company successfully claimed that restricting its ability to promote off-label use with (in the words of the court) “truthful and non-misleading information” violated its First Amendment right to free speech.
Let me be clear here. I am all for maintaining the longstanding prerogative that physicians and other licensed prescribers have to prescribe approved medications for unapproved indications. That’s not what this is about.
Continue reading This is a Very Bad Idea
A recent FDA advisory panel recommended the approval of 2 new agents in a novel class of cholesterol lowering drugs known as PCSK-9 inhibitors. What makes this remarkable is that these drugs illustrate all the promise and pitfalls of modern pharmaceutical development.
First, a little science. The target of the new drugs – a protein named proprotein convertase subtilisin/kexin type 9 (PCSK-9) – was discovered in 2001. Two years later, investigators reported that “gain-of-function” mutations in the gene that codes for PCSK-9 were associated with familial hypercholesterolemia and high rates of atherosclerotic vascular disease. Mutations of the gene that led to reductions in the function of PCSK-9 were associated with low LDL-cholesterol levels, and a lower incidence of vascular disease. That made the compelling case that PCSK-9 had a counter-regulatory function in LDL-cholesterol metabolism, so that interfering with its function would lead to lower cholesterol levels.
Continue reading The New Paradigm