President Donald Trump has nominated Scott Gottlieb, MD, to be the next FDA Commissioner. To his credit, Gottlieb is not certifiably crazy like several of the other candidates who were reported to be under consideration for the job. But, as I have noted in the past, he is is a deeply conservative ideologue who is determined to reduce the government's role in healthcare. To illustrate, here is a lightly edited version of a CardioBrief :
WSJ Op-Ed On Sham Surgery Gets It Wrong
In Scott Gottlieb, a former FDA official under President George W. Bush, argues that the FDA should stop requiring medical device companies to use sham procedures when they test certain new products. To support his argument he uses the example of renal denervation, a once highly promising new technology for lowering blood pressure. Unfortunately, Gottlieb extracts exactly the wrong lesson from this story, because the renal denervation story is a perfect example of why sham procedures can be both necessary and more ethical than any alternative.
At first glance it's hard to disagree with Gottlieb. The idea that patients would receive an invasive surgical procedure that could do no good appears abhorrent. "Research that introduces harm or risk with no opportunity for benefit would seem to conflict with the principles governing research on humans," writes Gottlieb.
But the same exact words could and should be used to describe medical devices with no proven benefit. In such cases, however, instead of the relatively small number of patients potentially exposed to harm in clinical studies, the number of patients exposed to harm in the real world may be larger by many orders of magnitude.
Let's examine this issue using the same example used by Gottlieb: renal denervation. In case you haven't been following this story, for the last few years renal denervation -- the zapping of the renal artery with electric energy to lower blood pressure -- has been widely considered to be one of the most exciting and promising new medical technologies around. Based on preliminary -- and uncontrolled -- studies renal denervation was thought to consistently lower blood pressure by an astounding 25-30 mm Hg and was hailed by many hypertension experts as a potential breakthrough cure for people who had persistently and dangerously high blood pressure even after taking several blood pressure pills.
Then reality intruded. that its pivotal trial of renal denervation had not met its primary endpoint. It became clear that renal denervation was neither a cure nor a breakthrough. Although the details of the trial have not been revealed, the underlying cause of the trial's failure is fairly well understood. Last summer a few skeptical British researchers carefully examined the underlying science and concluded -- contrary to what industry and the experts had been saying -- that . And the reason was simple: because all the early studies had been either uncontrolled or poorly controlled, the companies and the researchers had greatly overestimated the true effect of renal denervation. The Medtronic trial, by contrast, was much more rigorous, in part because it included the notorious sham procedures decried by Gottlieb.
As Gottlieb notes, renal denervation "is already available in Europe, where regulators approved it based on traditional studies." Gottlieb fails to notice how this undercuts his point. After all, many thousands of people in Europe have received a device from which they may have received no or little benefit and may have experienced harm. But this doesn't seem to concern Gottlieb. Here, writing about the effect of well-controlled trials, is what does concern Gottlieb:
"All of this raises development costs -- and it encourages firms to skip the U.S. market and commercialize new products overseas. This can suppress innovation. When a sham trial doesn't produce positive results, the company may have exhausted its resources and have no capital left to refine a good idea into a beneficial product."
But, as the renal denervation story demonstrates, the trial didn't "suppress innovation." Instead it showed that the so-called "innovation" was, well, a sham.
Although he waves the flag for ethics, it's clear to me that what really motivates Gottlieb to stand up and salute is business profits. He's far more interested in the health of businesses than he is in the health of patients. If he were actually interested in the welfare of patients he would pay attention to the medical details of his examples. But instead his real concern seems to be the health of the bottom line.
This isn't the first time Gottlieb has confused ethics and economics. In 2012 he wrote about another new medical technology, transcatheter aortic valve replacement (TAVR), and argued that the FDA and CMS had prevented the American public from benefiting from the procedure. , Gottlieb completely ignored the complex issues of the story, spinning an untrue but compelling tale in which the forces of government were preventing desperately sick people from getting a new life-saving therapy.
It seems clear that Gottlieb has very little interest in a serious discussion of clinical trials or the best way to test and introduce new medical technology. What he really wants is to provide a basis for campaign slogans that undermine the FDA and government involvement in medicine. It doesn't matter that sham surgery is a complex issue or that TAVR had as much potential danger as benefit. It's easy to score polemical points by scaring people, telling them that they will be subject to harmful procedures or that their government is preventing them from getting life-saving treatments. It's much harder, and less politically advantageous, to consider the full complexity of these issues.
Commented cardiologist Sanjay Kaul, MD:
"'Those who cannot remember the past are condemned to repeat it.' There are many examples where the 'scam' of some device-based therapies has been exposed by the 'sham' procedure. How can anyone forget the classic examples of laser TMR (transmyocardial revascularization) or pacemaker for hypertrophic cardiomyopathy, both touted as breakthrough interventions. It would not surprise me one bit if RF ablation for atrial fibrillation ends up with a similar fate!"