A national Medicare coverage policy is now set for transcatheter aortic valve replacement, stabilizing the reimbursement environment for the new procedure, but also establishing a level of payer restrictions that is rare for a technology so early in its life cycle.

The TAVR procedure, an alternative to open heart valve surgery, is being touted as practice-changing by cardiologists and a blockbuster business opportunity for device companies. CMS' reimbursement approach to the revolutionary technology may provide a window into how the agency will address future high-profile, early-stage technologies in an era of heightened concerns over costs and evidence.

The coverage decision is also one of the first to be completed under a 2010 memorandum of understanding between CMS and FDA allowing for enhanced data-sharing between the two agencies. (See Also see "FDA And CMS Agree To Share Data Routinely In Step Toward Parallel Reviews" - Medtech Insight, 28 June, 2010..)

CMS finalized its coverage-with-evidence-development determination for transcatheter aortic valve replacement (TAVR) May 1, tweaking a February proposal in response to input from physicians and device companies.

The final policy comes about six months after Edwards Lifesciences Corp. launched Sapien, the first FDA-approved TAVR device. The American College of Cardiology (ACC) and the Society of Thoracic Surgeons (STS) requested the coverage analysis – and CMS began it – prior to FDA's approval of the Sapien, a highly unusual move that reflects a focus in the clinical and payer communities on preventing both overly quick adoption of the procedure by unprepared physicians and use of the device in inappropriate patient populations. (See Also see "CMS Jumps Out Ahead Of FDA With Transcatheter Valve Coverage Analysis" - Medtech Insight, 3 October, 2011..)

Notable Changes From Proposal

Superiority standard axed: Transcatheter valves can be covered in a clinical study assessing an off-label use, even if the study is designed to prove only non-inferiority, the final rule says, but CMS includes some caveats and words of caution.
Registry follow-up: Patients enrolled in the Medicare-linked national registry will need to be followed for at least one year, rather than the five years of follow-up called for in the proposal.
Non-coverage: Instead of listing specific non-covered co-morbidities, CMS gives more leeway to clinicians to determine which patients are ineligible for TAVR, even as part of a clinical trial.
Facility/operator requirements: The prior-experience volume requirements were tweaked based on a consensus document from clinical societies. The “heart team” concept is further emphasized.

The final decision incorporates the same basic coverage conditions mapped out in the earlier proposal. It includes the requirement that Medicare beneficiaries treated for FDA-approved indications with TAVR be enrolled in a national registry, which has already been established by ACC and STS. And, as proposed, those treated for off-label indications will need to be enrolled in a more controlled trial. It also lays out a set of facility and operator standards, including procedure-volume experience thresholds, that need to be met to qualify for TAVR Medicare coverage.

CMS also made some adjustments from its proposal in response to outside criticism. Most notably, it backed off on its proposal that off-label TAVR procedures be covered only in trials designed to prove statistical superiority. It also shifted its policy to give practitioners more flexibility to decide whether other conditions suffered by a patient should preclude TAVR.

On many coverage provisions, however, the agency stuck to its guns, with support from the primary clinical specialty societies.

Most industry observers agree that the policy is likely to have a positive impact on Edward’s sales push for Sapien. The company has reported that some hospitals and clinicians have held back from initiating TAVR programs in response to unclear signals from local Medicare contractors waiting on final word from CMS.

The new Medicare coverage policy, combined with recent, relatively favorable coding and payment decisions for TAVR, may increase confidence among prospective users. (See Also see "Sapien Valve Reimbursement Begins Taking Shape, But Key Questions Remain" - Medtech Insight, 8 August, 2011..)

“With the Medicare reimbursement issue now reaching a resolution, we believe certain sites will now ramp up utilization/procedure volumes,” concludes Amit Bhalla, an analyst with Citigroup, in a May 1 research note.

CMS Displays Some Flexibility

But the longer-term impacts of the coverage policy on procedure adoption remain to be seen. Part of it will depend on CMS’ willingness to adjust some of its standards as clinicians gain experience.

There are signs that CMS is approaching the matter with flexibility, while taking seriously its role in steering adoption toward only evidence-supported physicians and patients. Of note, as in the proposal, the policy simply references “FDA approved indication” to designated uses of TAVR that are covered with patient registry enrollment. This allows newly approved indications to automatically qualify for the less-restrictive coverage option without need for a new coverage analysis process.

St. Jude Medical’s investigational Portico transcatheter aortic heart valve

Photo: St. Jude Medical

This differs, for instance, from CMS’ approach to carotid stenting, another procedure impacted by national coverage restrictions very early in the device's market life. In that instance, the agency established a covered indication that was more restrictive than FDA labeling, and has since been reluctant to expand its policy. (See Also see "Carotid Stenting: CREST Makes A Splash, But Market Will Depend On CMS" - Medtech Insight, 8 March, 2010..)

Edwards’ Sapien device is currently approved for patients with severe aortic valve disease deemed to be inoperable, but in June an FDA panel will consider Edwards' request for further approval of the product as an alternative to surgery in patients at high risk for the open procedure. Along with other companies pursuing the TAVR market, Edwards envisions a continuous evolution in expanded labeling for transcatheter valves as the technology gains adoption and more data is collected.

CMS also showed wiggle room in removing its proposed requirement for superiority trials for off-label TAVR indications. Several device makers and clinical groups had protested that the mandate would have stalled clinical development of the technology, and some suggested it was an unprecedented attempt by CMS to dictate protocol details for trials. (See Also see "Superiority Standard? Groups Bristle At CMS’ Transcatheter Valve Trial Proposal" - Medtech Insight, 12 March, 2012..)

Although Sapien was approved for inoperable patients based on superiority compared to best medical management in Edwards’ PARTNER/cohort B study, that was likely the last superiority TAVR trial design in the foreseeable future. Edwards’ pending high-risk surgical PMA is tied to a finding of non-inferiority to open valve surgery in its PARTNER/cohort A study. Both Edwards’ and Medtronic PLC’s (CoreValve) FDA trials for next-generation products also involve non-inferiority designs. And no doubt other firms, including St. Jude Medical Inc. (Portico) and Boston Scientific Corp. (Lotus), which are further back in the development process, are also planning non-inferiority strategies.

CMS accepted the argument, but made clear that it was not wholeheartedly embracing non-inferiority designs. It included some caveats in its supporting decision memo that could inform individual coverage decisions by Medicare contractors for TAVR trials.

The agency points out non-inferiority protocols often leave too much open to misinterpretation, depending on the non-inferiority “margin” chosen by investigators.

“Where feasible, superiority study designs should be used to investigate non-approved, off-indication and off-label uses,” CMS stated. If a non-inferiority trial is performed, sponsors should take care to disclose on the national ClinicalTrials.gov public database specific statistical information about the trial, CMS said.

Another change in the final rule is a reduction in the minimum required follow-up period for patients in the national registry from five years to one year.

Also highlighted by industry observers as a positive adjustment was CMS’ removal of specific patient co-morbidities, such as “mixed” aortic valve disease or hypertrophic cardiomyopathy, that would automatically make a patient non-covered for TAVR. Instead, the final policy states that the procedure “is not covered for patients in whom existing co-morbidities would preclude the expected benefit from correction of the aortic stenosis.”

The new wording “will allow physicians to use their judgment when treating a patient ... thus providing physicians with greater flexibility,” writes Larry Biegelsen, with Wells Fargo, in a May 2 report.

Experience Requirements Tightened

Another major debate during the coverage process centered on what facility and operator accreditation standards should be required for reimbursement.

CMS adopted the cautious approach advocated by ACC and STS, both of whom sought to minimize safety issues while the procedure is in its early commercial phase.

The final decision makes several tweaks to the proposed experience requirements based on a consensus document issued in March by the societies, in several cases increasing the necessary number of procedures needed to qualify for TAVR coverage.

Dozens of individual clinicians had written to CMS complaining that some of the proposed operator requirements were too strict and would overly limit access to the procedure. In particular, interventional cardiologists criticized the mandate that interventionalists would have to perform at least 50 structural heart procedures before qualifying for TAVR. ACC, in its written comments to CMS on the coverage proposal, actually adjusted some of its recommendations compared to the consensus document, based on the individual doctors' remarks. (See Also see "Cardiologists Push Back On Proposed Transcatheter Valve Credentialing Proposals" - Medtech Insight, 12 March, 2012..)

But CMS relied on the formal consensus document as its guide. “While individual practitioners have expressed opinions contrary to the specialty societies, we find the consensus document generally more persuasive and representative of the specialty physician community,” the agency wrote.

“We expect that professional societies, device manufacturers and thought leaders in this field will regularly evaluate their own requirements and standards set forth in the consensus document and keep CMS abreast of any changes in light of newly gained experience.”

For hospitals that will be newly adopting TAVR, the final decision increased the experience requirement to 100 prior structural heart cases for interventionalists, but also provided an alternative threshold of 30 left-sided structural procedures per year, of which 60% should be balloon aortic valvuloplasty. It also clarified that interventionalists at hospitals that have already adopted TAVR (such as PARTNER trial clinical sites) would be judged primarily on their TAVR-specific experience.

The final policy also puts increased emphasis, as a fundamental requirement for coverage, on the so-called “heart team” approach, where surgeons, interventional cardiologists and other specialties have a formalized collaborative arrangement in place for evaluating and treating patients with severe aortic valve disease.

It also newly lists infrastructure requirements for hospital TAVR programs, including that certain advanced imaging systems be incorporated into a hospital's cardiac catheterization lab and that there be sufficient space for the complex procedure.

Model For The Future?

The TAVR coverage process has attracted significant attention and feedback from the device industry, in part because of strong interest in developing TAVR technology among the major firms, but also because it serves as a high-profile opportunity for CMS to test new policy approaches that could be applied to other technologies and clinical arenas.

One new strategy employed in the decision was the enhanced collaboration with FDA, as laid out in the 2010 memorandum of understanding between the agencies. But CMS is not disclosing what specific non-public information or advice it received from its sister agency during the TAVR coverage process.

Another program being targeted for reforms in the midst of the TAVR process is CMS’ coverage-with-evidence-development initiative. The agency has said it plans to increase use of CED, in which coverage is tied to registry and trial requirements and, as part of that, to adjust its policies and procedures for the program. (See Also see "CMS Coverage-With-Evidence-Development Policy Could Face Revamp" - Medtech Insight, 14 November, 2011..)

Device companies used the TAVR coverage process to reiterate their push for a more collaborative but also more restrained CED program. Under industry proposals, CMS would work with stakeholders, including manufacturers, after a national CED policy is finalized to work out the details of qualifying evidence collection. In particular, industry has pushed for the minimum necessary data collection and clear-cut end dates. (See Also see "Device Makers Want Say In Medicare 'Coverage-With-Evidence-Development' Policy" - Medtech Insight, 30 January, 2012..)

But CMS declined to signal any broader changes to its CED approach in the TAVR decision, pointing to its recent effort to collect public comments about potential CED reforms and its planned May 16 Medicare Evidence Development and Coverage Advisory Committee meeting on the topic.

“The NCD process is an open and transparent process with specific statutory timeframes that allows the public to comment on the specific decision, including the CED elements of the decision,” CMS said in the final decision memo. “However, general comments regarding CED are outside the scope of this decision.”

CMS Policy Sets Boundaries On Transcatheter Valve Procedure

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