The Medicare Payment Advisory Committee has begun exploring whether CMS needs more help in fostering its coverage-with-evidence-development program.

Coverage with evidence development (CED) provides reimbursement for technologies or procedures that the Medicare agency feels are not yet fully proven in exchange for patients' participation in a registry or randomized clinical trial in which more data can be collected.

According to MedPAC analyst Nancy Ray, CMS "has come under particular pressure to find ways to reconcile the tension between developing evidence-based policies and being responsive to the pressure from product developers, providers and patients to pay for new services and new indications of existing services."

The program has been used sporadically over the past decade and a half, but CMS officials have indicated they may want to use it more often going forward (¹ Also see "CMS In Brief" - Medtech Insight, 19 October, 2009.).

Speaking during a MedPAC meeting Nov. 5 in Washington, D.C., Ray said that CMS has moved slowly with its CED rollout, calling for coverage with evidence development in about 10 instances over the past 14 years.

Two key challenges to better utilizing CED are the need to clarify CMS' statutory authority to use the tool, and to find sustainable sources of funding to support the clinical studies.

Questions About Agency's Authority To Pursue CED

At the Nov. 5 meeting, MedPAC Executive Director Mark Miller asked the committee if CED was something CMS should be pursuing at all, and if so, whether the agency's authority needs to be clearer in statute.

"Some stakeholders have commented that Medicare's use of coverage with evidence development is outside the program's statutory authority," Ray said.

She added that CMS has seen "considerable pushback" when it tried to use CED, particularly in a case where a health care service had already started to diffuse through the market. Ray said the pushback can come from device makers, patients and providers.

MedPAC chair Glenn Hackbarth, J.D., suggested that the agency's reluctance to use CED more often may be as much a question of politics as legalities.

Ray noted that CMS has modified its legal rationale for using CED. For example, in 2006 guidance, CMS articulated a different statutory path for using CED in a clinical trial than in a registry, she said (² Also see "CMS Offers Two Paths To “Coverage With Evidence” In Revised Guidance" - Medtech Insight, 17 July, 2006.).

Miller said CMS may be in a "middle zone" between having no legal authority to conduct CED and an absolutely clear legal authority to do so.

"You can construct a rationale," Miller said, but "how well it would withstand a challenge I think is where people feel threatened."

Hackbarth answered that the lack of clear legal authority "in turn influences how firm a stand you want to take with somebody that is resistant."

MedPAC member Tom Dean, M.D., urged the committee to back any statutory changes needed to support the agency's CED efforts "because the history is, there are just tons of situations where things have become standards of care without ever having the justification or the evidence base to support them."

"And once they get to be a standard of care, it's extremely difficult to change the patterns," Dean added.

Funding For Studies Is Still Ad Hoc

MedPAC members debated whether CMS should have a discrete set of funds devoted to the CED program. Lack of funding can prevent or slow patient access to new technologies if no sources come forward to finance the CED studies.

Currently, funding for the studies is project-specific, with some funded by the private sector, some by government research agencies and some through a mix of public and private support.

In addition, CMS must have the resources to design study protocols and analyze the clinical data resulting from the studies.

"At least one study has not yet begun to move forward because no funding source has come forward," Ray said.

She added that some stakeholders argue CMS should cover the costs of CED studies because the agency stands to benefit from more appropriate utilization based on the evidence generated.

MedPAC member Ron Castellanos, M.D., suggested that device manufacturers should fund CED studies on their technologies as a "cost of doing business."

Ray also raised the issue of potential study bias if CED studies are funded by industry.

"Some parties are concerned that the private sector may not always provide an impartial source of funding," she said. Others say "private-sector funding may be appropriate as long as Medicare develops clear standards regarding the design and methods of the study," Ray added.

CED Has Led To Refinements In Device Coverage

Ray said that CED has been used in national coverage determinations ranging from surgical procedures and devices to imaging and laboratory tests.

For example, in 1995 Medicare linked coverage of lung volume reduction surgery to treat emphysema to participation in a trial sponsored by the National Institutes of Health, although CMS had not yet officially created its CED policy.

When a 2003 study showed that some participants in the National Emphysema Treatment Trial were more likely to die with the surgery than with rehabilitation alone, Medicare revised its coverage policy to pay for only patients who matched the characteristics of trial participants who achieved survival benefit or improved quality of life.

"Since then, use of this surgery has remained low," Ray noted.

A second CED case study that emerged at the meeting was the use of implantable cardioverter defibrillators (ICDs) for primary prevention of sudden cardiac death (³ Also see "CMS Expands ICD Coverage In Long-Awaited Decision; Stakeholders Rejoice" - Medtech Insight, 31 January, 2005.).

"One of the issues with ICDs is that they had the potential to be used by a lot of beneficiaries," Ray explained.

When a prospective registry created in 2005 left questions unanswered on the benefits of ICDs in certain patient subgroups, a 2007 ICD working group called for a longitudinal study of 3,500 patients.

There were initial challenges in funding the additional longitudinal analysis, but it has financial support this year from both the private sector and NIH, Ray said.

"This case study highlights the capability of registries at collecting enormous amounts of data through research," Ray said, adding that CED study designs may need to be modified over time.

And while public and private financial support for CED trials and registries is possible, "sustained funding [challenges] could cause delays in some aspects of study implementation," she added.

In some cases, CED ultimately leads to better coverage. For example, CMS recently removed CED requirements for positron emission tomography scans in diagnosing or staging many types of tumors.

The topic of gathering evidence as part of the Medicare coverage process also surfaced at a reimbursement conference held Nov. 10 in Washington, D.C., by the Medical Device Manufacturers Association.

Steve Phurrough, a medical officer in the Center for Outcomes and Effectiveness Research at the Agency for Healthcare Research and Quality, and former director of the coverage and analysis group at CMS, suggested that CMS may want to collect evidence on new technologies in exchange for granting earlier coverage.

In the future, Phurrough predicts, certain innovations may enter the market with somewhat lax standards on both the regulatory approval and Medicare payment side "in return for a continuing collection of information that would allow that coverage to be withdrawn if subsequent information showed that the preliminary data was not seen in follow-up data; and you get that in large observational studies."

- Monica Hogan ( ⁴ m.hogan@elsevier.com )

What Ails Coverage With Evidence Development? MedPAC Discusses Issues

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