CMS To Issue Clinical Evidence Policy Paper In August

The Centers for Medicare & Medicaid Services will issue a position paper in August outlining ideas for improving the quality of clinical evidence, CMS Chief Medical Officer Sean Tunis, MD, said.

"We hope to have in the next two to three weeks a public position paper related to ongoing and new CMS initiatives related to improving the quality of clinical evidence," he said during a July 20 Drug & Device Dialogue audio conference sponsored by F-D-C Reports (the publisher of "The Pink Sheet") and Polidais LLC.

"We are trying hard through our evidence-based coverage process to create an incentive to do well-designed, practical clinical trials, because the criteria for coverage and reimbursement obviously influence the design and funding of clinical research studies," Tunis explained.

The benefits and pitfalls of Tunis' vision for "practical" clinical research were debated during the audio conference.

In addition to Tunis, conference participants included former FDA Center for Devices & Radiological Health Director David Feigal, MD; Johnson & Johnson VP-Health Policy & Government Affairs Kathy Buto; and Medtronic VP-Chief Quality & Regulatory Officer Susan Alpert.

[Editor's note: To sign up for a ¹ replay of the July 20 audio conference, visit www.fdcreports.com/dialogue or call 800-332-2181.]

One topic covered by the CMS position paper will be work the agency is doing on "coverage under protocol," whereby a drug or technology is reimbursed within a clinical study while it is being evaluated.

"What we're pursuing, at least for the time being, is limiting reimbursement to activities that are actually generating data," Tunis said.

CMS would also prefer clinical evidence based on functional outcomes, he said. "Since patients don't experience intermediate or surrogate outcomes, it's less useful for decision making...because then you would have to rely on the linkage between a surrogate or intermediate outcome and a final outcome."

Tunis noted that comparative effectiveness research data is also important. CMS is working with the Agency for Healthcare Research & Quality to develop a comparative study agenda under the Medicare Modernization Act of 2003 (² (Also see "Rx Comparative Effectiveness Research Funding In Doubt" - Pink Sheet, 19 Jul, 2004.), p. 22).

A Drug & Device Dialogue conference on cost-effectiveness was held in April (³ (Also see "FDA Mission Should Not Include Rx Cost-Effectiveness, Rep. Allen Says" - Pink Sheet, 3 May, 2004.), p. 35).

Tunis cited several CMS activities to contribute to practical clinical trials. He pointed to the research incentives created by the agency's recent change to its policy that obesity is not a disease.

The determination creates "for the first time the incentive to do meaningful studies of the long-term impacts of obesity treatments," Tunis said.

This could also drive investment in obesity-related therapies "because now there is the possibility of actually getting them paid for," he added.

CMS is "working on designing practical clinical trials on the use of PET scanning for suspected dementia with the National Institute on Aging," and a trial on daily dialysis in conjunction with the National Institute of Diabetes & Digestive & Kidney Diseases, Tunis said.

The agency is working on similar collaborations for practical research with the National Cancer Institute and the National Institute of Mental Health.

Buto noted that the pharmaceutical industry would be interested in partnering with CMS on practical clinical trials.

"I certainly know that our company, among others, would be very interested in collaboration on some of these practical clinical trials," the J&J exec said. "But we would want to really sit down and be able to be a partner in helping identify those good candidates from among the things we know about."

Buto also urged CMS to engage in an "open and transparent process" about the use of information resulting from practical clinical trials, expressing concern that results could be used to limit access.