CardioExchange Archive

From the first early stages of its development, the prospect of transcatheter aortic valve replacement (TAVR) provoked two broad and competing fears:

1. Regulatory safeguards would kill a promising new technology, denying its life-saving benefits to many thousands of desperately sick people.

2. The stampede to stake a claim in a promising, highly lucrative new territory would lead to the exploitation and mistreatment of many thousands of desperately sick people.

Remarkably, neither scenario occurred. Instead, at a very early stage, medical societies, regulators, and industry worked together to ensure the smooth introduction of TAVR in the U.S. The final decision earlier this week by the Centers for Medicare & Medicaid Services (CMS) to provide reimbursement for TAVR was the latest step in a long, ongoing process that, for once, didn’t appear broken, and, in fact, represented an unusual consensus among physicians, regulators, insurers, and other involved parties.

However, in an apparent attempt to inject politics where it’s neither needed nor wanted, Scott Gottlieb, a conservative activist who is a former FDA deputy commissioner and CMS adviser, concludes that the CMS ruling means “that for costly procedures, Washington will be making more of these choices for us.” In a posting on the American Enterprise Institute’s The Enterprise Blog, Gottlieb writes that the decision “is a vivid example of how our healthcare is going to get reimbursed now that Washington calls more of the shots.”

Gottlieb doesn’t make a clear statement that explains his hostility to the CMS decision. Instead, he cites several facts that he thinks makes his case for him. He’s wrong.

For instance, Gottlieb writes:

CMS is also restricting the number of doctors that can perform the new procedure.

Actually, CMS has done nothing of the sort. It has insisted that doctors who perform the procedure have adequate training and that the hospitals where the procedures are performed have sufficient experience and adequate facilities. Perhaps Gottlieb would be happy to send an elderly relative for TAVR  to a local community hospital with little experience in the procedure. It was precisely to avoid this scenario that the American College of Cardiology and the Society of Thoracic Surgeons supported CMS in this coverage decision. I fail to see how anyone would benefit by widespread proliferation of TAVR by novice operators at inexperienced centers.

Gottlieb again:

CMS is also going to tightly control the ability for the device to be used in less sick patients. It will require any patient who wants the procedure to be assessed by two separate cardiac surgeons to certify they were not suitable for open-heart surgery.

I suppose it’s possible to view this as government intrusion in the medical decision-making process between a doctor and a patient. I tend to think this represents CMS providing some reasonable safeguards to protect elderly patients from being victims of a system that in the past has sometimes ruthlessly exploited this same population. Again, however, it should be noted that the CMS decision is based on the evidence, as coverage is only given for a population in which benefit has been demonstrated.

Gottlieb takes a strong stand against clinical trials:

CMS only agreed to cover the device for its FDA approved use and only if the manufacturer agrees to continue studying the device in its FDA approved indication. The FDA approval, already based on unusually large and long-term trials that enrolled thousands of patients, was not enough to secure clear Medicare coverage.

Why is it a bad thing to insist that the manufacturer continue to study the device? To be sure, the TAVR trials were enough to gain FDA approval, but we are still very far from knowing the ideal role that TAVR can and should play in our healthcare system. Time and again problems with devices and drugs have popped up years after approval. Requiring continuing studies with this extremely complex device is a no-brainer. If the device is as good as many people seem to feel, these followup studies will provide convincing evidence for expanded use of the device. If not, not. In either case we will be better off than we would be without additional studies.

Gottlieb also criticizes CMS for refusing to cover patients who are eligible for open-heart surgery unless they are being treated within the context of a clinical trial. What exactly is the basis for Gottlieb’s objection here? At this point, we should remember, TAVR does not even have an FDA-approved indication for patients who are surgical candidates. The FDA will soon review an expanded indication for these patients. It seems likely CMS will expand its own coverage when and if the new indication is approved.

But Gottlieb misses another key point here. He fails to recognize that if the procedure were to become more widely available it would then become nearly impossible to establish its true worth through a clinical trial, as the interests of industry, hospitals, and physicians would converge and destroy all chance of enrolling patients in such a trial. This has happened on many occasions in the past with new devices and procedures. By insisting on trials, CMS is helping to insure that we won’t get fooled again.

Gottlieb believes TAVR should be widely available:

The same device has been approved in Europe for almost 5 years and has gained widespread use by patients who want to forgo very invasive open-heart surgeries—for good reason. The minimally invasive approach poses a lot less hardship on patients… This ruling will force people to get open-heart surgeries that might have been avoidable.

This certainly sounds good. After all, who wouldn’t want to avoid open-heart surgery? The problem is, as the literature makes clear, the choice of TAVR or surgery in patients eligible for surgery is extremely complex. The FDA, as I mentioned, is about to take up this issue, and it seems likely that the device will gain approval for this indication, but the decision to choose TAVR over surgery is by no means easy. In a discussion about the trial comparing TAVR to surgery, the editorialist in the New England Journal of Medicine wrote:

for those who are candidates for either transcatheter or surgical replacement, the findings present a dilemma in balancing the risks of increased neurologic complications against the benefits of avoiding sternotomy and cardiopulmonary bypass.

This assessment isn’t as simple as Gottlieb’s, but it has the virtue of being the product of a careful review of the actual data. But, of course, idealogues like Gottlieb never let a few facts get in the way of a compelling story.