Sluggish CMS Reimbursement Lags Cardiac Device Approvals: Should Value Endpoints be Added to Trials?

The Take Home.  Sluggish CMS Reimbursement Lags Cardiac Device Approvals: Should Value Endpoints be Added to Trials?For cardiac device clinical trialists, the protracted gap between FDA approval and a Centers for Medicare & Medicaid Services (CMS) reimbursement decision is more than frustrating—it creates a bottleneck of available devices that cannot be accessed by many patients who would benefit the most. But if trials were designed from the outset to prove not only safety and efficacy but also increased value, this gap could be shortened, according to authors of a special report.

For background, after years of anticipating that the FDA would approve the Watchman device (Boston Scientific) for left atrial appendage closure—which finally happened in early 2015—investigators had to wait another 11 months for CMS to release its final reimbursement decision. Additionally, CMS did not rule on what it would cover for the first TAVR valve until 6 months after the FDA approved it for use in patients with severe aortic stenosis.

“These delays arise in part from the incomplete overlap between safety/effectiveness and reasonable/necessary standards,” write Donald Cutlip, MD, and Daniel Kramer, MD (Beth Israel Deaconess Medical Center, Boston, MA), in a special report published last week in Circulation: Cardiovascular Interventions.

To remedy the broken process, they propose that future cardiac device trialists work with CMS and FDA in the design phase to add both meaningful patient-reported outcome measures (PROMs) and cost-effectiveness metrics that will prove value beyond the novelty of new technology. “You can’t just have a theoretical advantage and expect that you can charge a premium for that because you haven't demonstrated value,” Cutlip told TCTMD.

PROMs can include a myriad of verified quality of life questionnaires, functional status, and perceived symptom burden, Cutlip and Kramer suggest. The obvious limitations to these include their subjective nature and difficulty to assess, they add, so while PROMs will be unlikely to replace hard clinical endpoints like mortality and major morbidity, “it can be argued that they are often at least equally important from the patient perspective.”

Rendering PROMs Useful

Acknowledging that emphasizing PROMs and cost-effectiveness measures in large device trials will be “more work” for investigators, Cutlip said that most of the recent trials have included some components of them. “So it’s not new in terms of making the measures. What would be new would be to actually use them as an approval type endpoint,” he said. “They’ve always been regarded kind of as interesting but not really usable as an indication for approval or reimbursement for that matter.”

If each trial had an obvious quantifiable hard clinical endpoint, “we would all prefer that,” Cutlip noted. “But the problem we run into with the device trials is that we’re getting to a point where it’s very hard to measure differences.” That’s why a noninferiority design has become standard in the field, he said.

But from patient or payer perspectives, why would they want to shell out substantially more money for a new device that is at best noninferior to the current standard? This is where PROMs come in, Cutlip said. “Using validated systems with core laboratories and careful implementation of the process, [we can] end up with information that’s valuable for showing that secondary difference.”

Looking forward to mitral valve trials currently in the works, Cutlip said this concept will be very important, especially given the fact that “we really aren’t even expecting these devices to show a difference in terms of major clinical outcomes.” Ideally, he’d like to see a trial compare mitral valves to standard medical therapy and show noninferiority of the new device in terms of mortality and heart failure hospitalization, but also “a very clear advantage” for a cardiomyopathy questionnaire or other PROM. “That’s probably enough for CMS or other payers to acknowledge that there’s a value here that’s probably worth paying for,” Cutlip added.

Commenting on the report, William Weintraub, MD (Christiana Care Hospital, Newark, DE), told TCTMD in an email that researchers should “have a faster, more responsive process” for obtaining both FDA and CMS reactions. But he noted that there is an advantage to separating FDA approvals from CMS reimbursement decisions because “they are fundamentally different. The time lag is the unfortunate part.”

As for PROMs, they should be included in trial designs “when relevant,” Weintraub said, adding that they should “at least be considered at the design stage.” His skepticism increases, however, when thinking about including cost-effectiveness measures as secondary endpoints.

Cost-effectiveness metrics “can establish that a new therapy provides value,” but it could be hard to show more precise technical improvements, Weintraub said. “When new technology improves patient outcomes by prolonging life or preventing clinical events, but where there are higher upfront costs, then cost-effectiveness can be useful in showing value. However, cost-effectiveness generally involves a number of assumptions about variables which cannot be measured (eg, life expectancy). Cost-effectiveness is useful in this situation of revealing underlying assumptions.”

‘In a Perfect World’

In terms of how interested CMS might be in getting involved in clinical trial design with the aim of expediting reimbursement decisions, Cutlip said that he is sure they are “looking for ways to be more efficient in their process,” but he has yet to speak to anyone there about his ideas.

“I don’t think there’s any reason they want to delay these approvals,” he said, adding that he hopes to work with a sponsor to demonstrate that prespecifying certain endpoints from the beginning of a trial will ensure a more timely response from CMS. “In a perfect world going forward, we would have our design meetings like we do now with FDA, . . . and hopefully also be able to have [a] discussion with CMS about which endpoints we need to show value,” Cutlip observed.

Then if those endpoints are met by the end of the trial, the criteria for reimbursement would be established pending review, he continued. So long as safety and effectiveness endpoints are also met, “[you would] be able to have marketing approval and reimbursement much closer together than we do now.” It’s unlikely that these decisions will ever be announced at the same time, however, Cutlip noted, “just because of resources. . . . But it should be much sooner than what we see now.”

  • Cutlip DE, Kramer DB. Value-based hypothesis testing for cardiac device clinical trials: a pathway for accelerated reimbursement decisions. Circ Cardiovasc Interv. 2016;9:e003627.

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  • Cutlip reports receiving institutional research funding from Medtronic, Boston Scientific, and CeloNova.
  • Kramer reports receiving funding from the National Institutes of Health-National Institute on Aging.
  • Weintraub reports no relevant conflicts of interest.

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