Great BioCentury lead article by Steve Usdin, Debating Limited Use.
FDA is asking the public to consider whether it should supplement the current all-or-nothing approval structure with a system that would couple approval of selected new drugs with measures designed to discourage off-label use.
The agency says it could approve drugs for conditions such as antibiotic-resistant infections and life-threatening obesity based on small, fast trials if it were confident use would be limited to well-defined populations.
There is little controversy about approving drugs based on relatively small studies that demonstrate high levels of efficacy in tightly targeted populations. But FDA’s suggestion that it could work with physicians and payers to limit use of a marketed drug in the absence of documented safety concerns is controversial.
To say the least.
Patient and medical groups support the concept of a limited-use pathway, and the President’s Council of Advisors on Science and Technology (PCAST) has endorsed the idea. PCAST’s recommendation to create a Special Medical Use (SMU) pathway was drafted in close consultation with senior FDA officials and reflects the agency’s thinking.
But, whether or not you believe the FDA has the legal authority for a “new” pathway, Janet Woodcock nails the problem dead on:
“We can indicate things for small populations, but often we are very concerned there might be a temptation to use them much more broadly.”
There are many important issues, let’s discuss two.
First – does the FDA have the authority, the resources, and the desire to direct the practice of medicine? Can the agency really do anything other than suggest, via labeling, how physicians should prescribe approved medicines? No doubt they can deter pharmaceutical companies from detailing or even discussing off-label use via prior agreement (and Caronia notwithstanding). Whether or not doctors are “tempted,” however, is another matter entirely. Is the FDA going to “ban” certain scientific publications or conference presentations? Is the FDA going to add a new tenet to the Hippocratic oath, “First do no reading?”
It’s a slippery slope indeed.
Woodcock added that FDA is not seeking to ban off-label prescribing of drugs approved through a new limited use pathway. The agency is interested in exploring whether payers might play a role in reducing inappropriate use, she said.
More power to the payers? Is that something we want?
Next is the issue of whether such expedited approval pathways are in the best interests of the sponsor. While there’s no doubt getting new treatments for serious and life-threatening diseases to market more swiftly is a good thing, is the threat of having a product license revoked after a “test” period in the sponsor’s best interest? Isn’t it a penny-wise, pound-foolish proposition? It depends. What is certain is that such decisions should not be driven by financial considerations of biotech venture capitalists or the panjandrums of Wall Street.
Naïve? Perhaps -- but none-the-less disturbing.
Is FDA PCAST-ing it’s net too wide?
Whenever there is authority, there is a natural inclination to disobedience.
-- Thomas Haliburton