The story has been out for a while about transcatheter aortic valve replacement (TAVR), so maybe it's time to project into the future. Before we head there, however, we should revisit the past.
The pivotal event in this story is the drug-eluting stent, a device that gave birth to what FDA's Bram Zuckerman, MD, described as the DES trauma. The DES story is twofold; rapid physician adoption and late-stent thrombosis (LST).
FDA saw the rate of adoption as an unruly and harmful stampede because of LST, but thrombosis was not the prime driver of the development of DES. Restenosis was. As of 2008, the rate of thrombosis in DES at 24 months roughly matched the rate in bare-metal stents, although first-generation DES units held it off until after a year whereas thrombosis in BMS units almost always took place within 12 months. Still, the episode sparked a lot of anxiety at FDA about other devices.
This leads us to the Sapien aortic valve made by Edwards Lifesciences, which snared an FDA approval in 2011 roughly four years after its approval in Europe and which the Centers for Medicare & Medicaid Services is still deciding how to cover. The central feature here is that both agencies are considering a very strict deployment which not only stipulates the involvement of at least half a dozen physicians, but also limits the use of the device to 100 or so centers in the U.S. By the way, this is the perfect way to virtually outlaw off-label use.
In the meantime, Edwards is working the U.S. pivotal trial for the slimmer Sapien XT, which already bears a CE mark. Ironically, Edwards no longer sells the original in Europe, but the most conspicuous aspect of the Sapien's FDA approval is how dependent it was on three medical specialty societies whose involvement gave FDA the framework for the restrictions the agency placed on the device. Regardless of whether one sees it as a quid pro quo, the conditions FDA imposed on the device's use guaranteed doctors that lots and lots of them would be able to grab some of the jingle that will accompany the device's roll-out in the U.S. market.
Whether that works for CMS's attempts to rein in costs remains to be seen, but the trade-off appears to be that CMS will pay more doctors per valve in exchange for fewer valves. I'm pretty sure this agency knows what it's in for and is confident it won't be in the red when all is said and done.
There may be several messages here, but what this says to device makers with a first-of-a-kind interventional cardiology device – perhaps a first-of-a-kind implantable device of any sort – is “get in bed with the medical specialty societies because you will not get to market otherwise.” Physicians already have control over the CPT codes used by CMS to determine Medicare coverage, and they now have much more sway over FDA's regulatory approval beyond their traditional role as advisory committee members.
It's tough to avoid the conclusion that doctors are relieving both agencies of a lot of difficult political work and are taking home a nice chunk of change for their troubles. In any event, doctors are more deeply entrenched than ever as another set of gatekeepers and now constitute a third regulatory entity device makers must engage “early and often,” as they say at FDA, in order to get to market. And that's what TAVR says about FDA and CMS.