Eligible Drug Costs Need Defining In Medicare Clinical Trial Policy, GSK Says: Drug Firms Recommend Revisions

Phase I studies and public access to data generated by CMS-approved research under national coverage determinations.

Medicare coverage of the cost of marketed drugs used in clinical trials may be appropriate in some readings of the current CMS national coverage determination clinical trial policy, GlaxoSmithKline suggests in recent comments to the Centers for Medicare & Medicaid Services.

In response to a request for input on revisions to the clinical trial policy, GSK highlights the need for added clarity around the definition of what items and services can be covered. CMS announced July 10 that it is planning to develop a new clinical research policy as part of a reconsideration of its approach to national coverage determinations.

The policy currently allows coverage for the "routine costs of a clinical trial, including all items and services that are otherwise generally available to Medicare beneficiaries." CMS also outlines what is included and excluded in the definition of routine costs (see box above). For example, Medicare will not cover the investigational item or service itself, items and services provided solely to satisfy data collection and analysis needs, and items and services customarily provided by the research sponsors without charge.

However, there may be cases when the investigational item is eligible for coverage and CMS should identify those circumstances, according to GSK. For example, "a marketed drug is used in a technically 'investigational' sense under conditions indistinguishable from conventional care," the company points out.

In other words, "the trials' use of the drug would qualify as an item that would be 'typically provided absent a clinical trial' - which the current clinical trial policy uses to define covered items." Specifically, a trial may include the use of a marketed product within its approved labeling in order to collect further safety or efficacy data or to compare it to treatment alternatives, GSK says.

Similarly, "a technically unlabeled use of a marketed drug being studied in a trial may be well-established as 'conventional care,' recognized in widely-consulted compendia, and routinely covered under existing Medicare standards for reimbursement in the treatment context." Excluding such a use from eligibility "would effectively reduce Medicare coverage and create a disincentive to trial participation."

To clarify the policy's exclusion of items or services "customarily provided by research sponsors without charge to any enrollee in the trial," GSK had two suggestions. First, the firm recommended the policy state that "Medicare should not be billed by a trial investigator for an item or service if in fact the trial sponsor is paying the investigator for it (or providing it)."

Second, GSK continued, "Medicare should not be billed for an item or service in a clinical trial unless all third-party payors that have coverage obligations to patients enrolled in the trial at the same site...will also be billed for it."

A number of drug companies raised issues with the study design criteria that must be met to qualify for Medicare coverage under the policy. GSK and Pfizer sought clarification of the requirement that a trial must have "therapeutic intent" to be eligible for coverage and not be designed only to test toxicity or disease pathophysiology.

GSK proposes that "many early-stage clinical trials...have a component of potential therapeutic benefit. CMS should clarify that a trial which includes measurement of an endpoint to measure direct or indirect (e.g., a surrogate marker) potential therapeutic benefit should be considered a trial with 'therapeutic intent.'"

Pfizer concurs that CMS should specify the types of studies, "including registries, observational studies and practical/pragmatic clinical trials that will qualify for Medicare coverage. We believe that the qualifying criteria should include all types of research that are intended to evaluate a therapeutic intervention." The firm also urges "the inclusion of Phase I clinical trials, if designed to test a hypothesis, and any other hypothesis-testing confirmatory clinical research studies."

Pharmaceutical manufacturer groups are also asking CMS to continue allowing Medicare coverage for drug trials conducted outside the investigational new drug process.

Pfizer advocates "timely and equal access" to data generated from CMS-approved clinical research. The company requests further detail on the agency's plans for releasing public data generated from covered trials, particularly those conducted as part of a national coverage determination. The agency should also clarify what kind of access medical product manufacturers will have to the data, Pfizer says.

Historically, CMS has not allowed direct access to such data if a medical-product manufacturer funds the study, the company notes. However, Pfizer suggests that CMS might develop a process that is similar to the National Institutes of Health data sharing policy, in which manufacturers are allowed to access data derived from clinical research on a study-by-study basis.

A number of drug firms challenged the applicability of Medicare secondary payer rules in the national coverage determination clinical research policy.

Companies expressed concern with the prospect that Medicare coverage may be denied if a research sponsor has committed to covering the costs of injuries resulting from the trials. Such a commitment could be construed as a "liability insurance policy" or self-insured plan that would excuse Medicare from primary responsibility under the secondary payer rules.

However, such an assumption is "unfounded," J&J maintains. Instead, the agency should "make clear that sponsors limit their payments for research-related injuries to costs not covered by the subject's health insurance."

Pfizer adds that "we do not believe that Congress intended the [Medicare secondary payer] provisions to preclude Medicare from being a primary payer when a study sponsor promises to pay for uncovered clinical research services." If the agency determines a sponsor may be considered an insurer under these circumstances, "we suggest that CMS create a mechanism for determining which standard of care services Medicare covers," Pfizer continues.

- Cathy Kelly ([email protected])