CMS Splits Coverage With Evidence Development Into Two Categories

Medicare is offering added flexibility to pay for research-related expenses that arise in the clinical trials conducted for a Part B national coverage decision, under revisions to Medicare's coverage with evidence development policy, released July 12.

The ¹ revised document invokes new legal authority to permit the agency to cover clinical research costs in specific circumstances.

The Centers for Medicare & Medicaid Services can make national coverage decisions (NCD) when costs of an item or service are reimburseable, overriding the judgment of local Medicare payment contractors.

Within the NCD option, CMS has established a coverage with evidence development (CED) policy, allowing payment for drugs, medical devices or medical procedures - but conditioning payment on collection of data.

The CED policy allows Medicare to offer payments and at the same time collect the added information needed to decide whether the product or service is "reasonable and necessary," Medicare's formal critieria for coverage.

Though CED offers added opportunity for Medicare payment, stakeholders have taken note that CMS has only used it to cover prescription drugs in one circumstance, the off-label use of four anticancer drugs being studied in clinical trials by the National Cancer Institute (² (Also see "A Closer Look: Colorectal Cancer National Coverage Decision After One Year" - Pink Sheet, 16 Jan, 2006.), p. 19).

The revised document updates a draft policy set forth in April 2005 (³ (Also see "CMS Proposes Broad Criteria For Linking Reimbursement To Data Collection" - Pink Sheet, 11 Apr, 2005.), p. 24). CMS seeks public comment on the revisions, though it did not : "⁴ CMS Coverage With Evidence Development Update"set a comment deadline (see chart).

CMS Creates "Coverage With Study Participation" Concept

A key change affecting research costs is the addition of the concept of "coverage with study participation." CSP allows for coverage of medical items or services in a research setting where the agency has determined there is limited existing evidence to support a coverage decision and more evidence is needed to "clarify the impact of these items and services on the health of Medicare beneficiaries."

Under CSP, an item or service could be covered only when the research includes "added safety, patient protections, monitoring and clinical expertise" as is defined in the legal authority directing the health services research activities of the Agency for Healthcare Research & Quality, according to the guidance.

Some public comments on the April 2005 draft said CMS did not cite proper legal authority permitting coverage for products or services still in research as part of an NCD (⁵ (Also see "Part B “Evidence Development” Plan Needs Defined Scope – Manufacturers" - Pink Sheet, 13 Jun, 2005.), p. 21).

CMS had earlier justified its approach to linking coverage decision to collection of additional data by citing Sec. 1862(a)(1)(A) of the Social Security Act, which states that Medicare may not provide payment for items and services unless they are determined to be "reasonable and necessary" for the treatment of an illness or injury. The new guidance cites a different authority, 1862(a)(1)(E), that would allow additional coverage in the research setting.

The guidance lists a number of situations in which CMS could use CED for items and services provided to Medicare patients enrolled in a clinical trial:

Available evidence may be a product of "rigorous evaluations," but may not have evaluated outcomes relevant to Medicare beneficiaries;

Available research may have failed to adequately address the risks and benefits to Medicare enrollees for off-label or other "unanticipated uses of a drug, biologic, service or device;"

Available clinical research may not have included specific patient subgroups or patients with disease characteristics that are highly prevalent in the Medicare population;

A product or service is already marketed but there is little published research available to evaluate a new application; or,

Evidence about the health benefits of an item or service to support a "reasonable and necessary" determination is available for only a subgroup of Medicare patients and/or for providers with certain experience or other qualifications.

Although CSP research may include a broader range of studies than randomized clinical trials, such as observational studies, all research must conform to standards underlying the agency's national coverage decision Clinical Research Policy.

Changes To Clinical Trial Policy Under Consideration

The Clinical Research Policy, previously named the Clinical Trials Policy, was established in 2000. In a separate ⁶ notice accompanying the CED guidance, CMS outlines several issues it is reconsidering and for which it seeks public comment.

These issues include clarifying payment criteria for clinical costs in research studies other than clinical trials, devising a strategy to ensure that Medicare-covered clinical studies are enrolled in the National Institutes of Health clinical trials registry website, and clarifying how an item or service that is not covered nationally may be covered in the context of clinical research to "elucidate" its impact in Medicare beneficiaries.

The "overarching" goals of the review are to allow Medicare beneficiaries to participate in research and to encourage studies that add to the knowledge base about the "efficient, appropriate, effective and cost-effective use of products and technologies in the Medicare population."

The revision is also aimed at allowing Medicare beneficiaries to receive care that may offer a health benefit but for which evidence on effectiveness is insufficient to merit full, unrestricted coverage. Meeting these goals may require regulatory changes in addition to revisions to the clinical trial policy, CMS notes. Comments on the issues raised are due Aug. 9, 2006.

Data Collection Described As "Coverage With Appropriateness Determination"

A second "precept" of the revised NCD/CED guide is "coverage with appropriateness determination" (CAD), which permits a coverage determination now, while also requiring additional data to ensure the item or service is being used appropriately.

"In the application of CAD," the guidance explains, "CMS may decide that there is adequate evidence to determine that an item or service is reasonable and necessary...but that additional clinical data is needed that is not routinely available on claim forms to ensure that the item or service is being provided to appropriate patients in the manner described in the NCD."

Although the CAD concept is not new, CMS describes its approach in a way that attempts to answer concerns about protecting patient privacy. "Our agreements with entities establishing the registries will include protections to ensure that Medicare health information is protected," the agency states.

CMS also notes that it "does not intend to routinely develop, oversee or maintain these databases or registries" but that it will only accept data from registries that have "qualified scientific oversight, tested and validated data collection methods, adequate patient safety and monitoring, quality assurance and data protection, and appropriate human subject protections."

The data collected under a CED may be used for a number of purposes outside the coverage determination, the guidance points out. Among the other possible uses are to stimulate industry development of products for patients with specific characteristics, use by health plans for quality improvement programs, including cost analyses and use by investigators for post-coverage analyses, including head-to-head comparisons and case control studies.

[Editor's note: This article appears courtesy of F-D-C Reports' Medicare Drug Focus. For a free trial, visit ⁷ www.MedicareDrugFocus.com or call 800-332-2181.]