Medicare CED Could Enhance Efficiency Of FDA Post-Approval Studies – CMS Draft Guidance

Medicare’s coverage with evidence development policy could help enhance the efficiency of research conducted under FDA post-approval requirements, CMS says in a draft guidance on CED released Nov. 29.

Medicare’s coverage with evidence development (CED) policy could help enhance the efficiency of research conducted under FDA post-approval requirements, CMS says in a draft guidance on CED released Nov. 29.

CED is a Medicare coverage mechanism that allows for Part B reimbursement of a medical item or service on the condition that patients receiving treatment are enrolled in a research study that evaluates the treatment’s outcomes, effectiveness and appropriateness. It does not apply to Part D.

CMS’ draft guidance revises a 2006 guidance on CED and is an important step in the agency’s re-evaluation of its approach to coverage with evidence development. Public comments on the draft are due Jan. 28, 2013, and a final guidance is expected shortly thereafter.

The draft guidance addresses instances where CED could be coordinated with FDA approvals, an issue that was not discussed in the 2006 document. “CMS has found historically that various products may be approved or cleared through FDA though significant Medicare-related evidentiary questions are unresolved,” the draft notes. Therefore, the “alignment” of CED with an FDA post-approval study requirement “presents an opportunity for greater research efficiency.”

However, the document indicates such coordination would not represent a major new purpose for CED. “This is simply an example of CED application rather than a new CED paradigm,” it says. In comments to CMS in January, policy groups offered an ambitious proposal in which CED would be used to enable expanded types of accelerated drug approvals at FDA (Also see "Medicare CED Could Enable Expanded Use Of Expedited Drug Approvals, Policy Groups Suggest" - Pink Sheet, 6 Feb, 2012.).

Louis Jacques, director of the coverage and analysis group within CMS, emphasized the research advantages of coordinating CED with FDA post-approval research during a conference in December 2011. He noted, “If you have an FDA postmarket requirement, you’re certainly going to enroll and complete that requirement much quicker if Medicare is in fact incentivizing enrollment. And if Medicare is also paying for your particular technology … it also seems like there’s some advantage there.”

Disappointing Provisions For Drug Manufacturers

Despite requests from drug company stakeholders, the draft guidance does not prohibit local Medicare claims administrators from implementing CED decisions. Nor does it require that CED will only be applied within the context of a national coverage determination, though it acknowledges CED could be part of a national coverage decision.

In earlier comments to CMS, Eli Lilly & Co. and Genentech Inc. had urged the agency to require that CED decisions can only be made on a national basis and not by local contractors (Also see "Medicare CED Decisions By Local Contractors Would Be Unworkable, Drug Firms Argue" - Pink Sheet, 6 Feb, 2012.). The companies argued that allowing CED under local coverage decisions introduces additional complexity, duplication and cost for stakeholders involved and would be unreasonably burdensome for sponsors.

In the draft guidance, CMS notes that Medicare policy “directs that providers, practitioners and suppliers submit claims to local administrative contractors that process and review those claims. This includes claims arising pursuant to beneficiary participation in clinical research.” Furthermore, CMS points out that its rules for national coverage determinations, which might involve a CED decision, specifically give Medicare claims contractors the authority to determine which routine clinical costs should be paid for in the context of relevant clinical trials.

The draft guidance does not specifically preclude the application of CED decisions to drugs or biologics, which may be another disappointment for drug firms.

Industry stakeholders had maintained CMS should avoid using CED for biopharma products at all, including for off-label uses, based on the concern that the coverage policy would unnecessarily restrict access. CMS has used CED just once for drugs, in a 2005 decision covering off-label uses for four colorectal cancer treatments. But clinical studies on those drugs have not supported any new coverage decisions for them.

The draft does exclude a reference to off-label drug uses that appeared in the 2006 guidance, which said a CED decision could be prompted when “the available clinical research may have failed to address adequately the risks and benefits to Medicare beneficiaries for off-label or other unanticipated uses of a drug, biologic, service or device.” However, the draft does not explain why the specific language was dropped, only that it has “modified the earlier list of the evidentiary findings that might result” in a CED decision.

The draft emphasizes that CMS will use CED to generate research data that will inform coverage decisions. “An approved CED study must be designed and conducted prospectively to produce evidence to inform future Medicare coverage,” the document states. It specifies that the agency will apply the mechanism to:

• Generate additional evidence on the relevance of outcomes to Medicare beneficiaries;
• Generate additional evidence using research subjects that are representative of a particular Medicare population;
• Re-evaluate evolving evidence that may call into question a past conclusion about a treatment’s impact on health outcomes in Medicare; and
• Generate evidence that is more representative of the typical community-based setting in which a Medicare beneficiary receives care.

In line with that emphasis on research generation, the draft no longer includes “coverage with appropriateness determination” as a type of CED. In the 2006 guidance, coverage with appropriateness determination (CAD) was defined as data generation including patient registries. CAD and coverage with study participation (CSP) were listed as two types of CED data collection. Now, CMS explains, “we believe that coverage with study participation is more consistent with our current conceptualization of CED.”

AHRQ And CED

The draft emphasizes the important role the Agency for Healthcare Research and Quality is expected to play in advancing the use of CED.

“We believe that AHRQ’s role will continue to develop as both agencies gain more experience in CED, and that the two agencies may address the implementation challenges together,” the draft says. CMS points out that AHRQ’s “authority and resources complement” CED in that AHRQ can convene stakeholders to design clinical studies, has the capacity to establish public/private partnerships to finance CED, and can invoke confidentiality protections regarding certain uses of data.

AHRQ’s capabilities can help address existing challenges to the successful completion of CED studies, CMS points out. Challenges have included the lack of clinical study experience or infrastructure in some stakeholder communities, limited financial resources to undertake studies, no “clearly authoritative professional society” to represent the multiple physician specialties managing certain conditions and “a culture that historically may not have embraced evidence-based patient care.”