The Food and Drug Administration may have approved Eli Lilly and Company’s Amyvid, but insurers remain unconvinced that Aβ imaging will do patients much good. On September 27, the Centers for Medicare and Medicaid Services (CMS) formally issued its final decision on coverage of the technology. As expected from the draft decision released in July, CMS denied coverage to Medicare beneficiaries for any clinical use of the technology, but left the door open for future approval pending more data on the benefits of amyloid imaging. In a process known as “coverage with evidence development” (CED), the agency will pay for a single scan for people participating in clinical trials designed to gather evidence about health outcomes. In its final decision, CMS broadened the parameters for a CED trial, in particular noting that it would accept short-term outcomes such as a change in medication or avoidance of unnecessary tests as sufficient evidence of a health benefit. A path toward such a trial has begun to take shape.

In its decision, which has had broad impact (see ARF related news story; ARF related news story), CMS cited the lack of peer-reviewed evidence that amyloid imaging has any effect on patient health outcomes. To date, only one published study (see Grundman, et al., 2013) has looked at how scans affect clinical diagnoses, and none have examined changes in disease management, noted an article by Steven Pearson, president of the Institute for Clinical and Economic Review in the September 30 JAMA Internal Medicine. Based at the Massachusetts General Hospital Institute for Technology Assessment in Boston, ICER evaluates healthcare interventions in different medical fields. It receives unrestricted funds from several insurance companies. In his article, Pearson summarized findings from ICER’s previous white paper that examined health outcome data for AD biomarkers in general; that paper was part of the evidence considered by CMS (see ARF related news story; ARF related news story).

In the same issue of JAMA Internal Medicine, an editorial by Robert Steinbrook at Yale University in New Haven, Connecticut, supported the CMS decision. “This approach serves the public interest. It would be irresponsible for the CMS to cover Aβ PET imaging without adequate evidence about its role in AD or other neurodegenerative diseases,” Steinbrook wrote. He has served on committees for CMS, but was not involved in evaluating amyloid imaging.

Stakeholders in the field voiced unanimous disappointment in the decision to deny clinical coverage, maintaining that amyloid scans should be available to patients now. George Vradenburg, who chairs the advocacy group USAgainstAlzheimer’s, wrote in a statement, “CMS has stifled an important tool for establishing an early diagnosis in millions of patients.”

At the same time, many saw the relaxed CED requirements as an opportunity, and vowed to gather the necessary evidence quickly. Importantly, the CMS decision specifies that CED trials could include “potentially hundreds of thousands of patients” in clinical settings, opening the door for reimbursement for many scans.

The Alzheimer’s Association intends to coordinate a community-wide effort to design and implement a CED trial. Leaders from industry, imaging associations, advocacy groups, clinics, and academia voiced support for this plan and expressed willingness to work together to realize it. The group includes academic researchers such as Keith Johnson at MGH and Reisa Sperling at Brigham and Women’s Hospital in Boston, Massachusetts; clinicians such as Norman Foster at the University of Utah in Salt Lake City, and Stephen Salloway of Butler Hospital in Providence, Rhode Island; PET imaging experts, including Peter Herscovitch at the National Institutes of Health and Satoshi Minoshima at the University of Washington, Seattle; and many others who participated in a brainstorming conference call organized by the Association.

The researchers agree the proposed trial should look at short-term patient outcomes, be simple to implement, and allow broad access to scans for as many Medicare patients as possible across the country. The Association plans to appoint a work group to design the effort, and its representative Maria Carrillo invited suggestions to present a single, unified plan for CMS approval. Leaders stressed that this community-wide effort will benefit all amyloid tracers in development, not just Amyvid. Lilly appears on board with this collaboration. “We will engage with the key stakeholders in the Alzheimer’s disease and imaging communities, so that together we can identify the most appropriate path forward,” Eric Dozier, who directs Lilly’s Alzheimer’s business division, told Alzforum.

For inspiration, the AD field looks to The National Oncologic PET Registry (NOPR). Under this seven-year program, clinicians in the cancer field gathered health outcome data that succeeded in gaining full coverage for oncologic PET ligands. During the conference call, researchers who had worked on NOPR offered to lend their expertise to the AD community. In addition, NOPR has the infrastructure in place to conduct large national trials. If it can be adapted for AD trials, this might speed up the process, researchers said.—Madolyn Bowman Rogers


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News Citations

  1. FDA Approves Amyvid for Clinical Use
  2. Me, Too: Florbetaben, Flutemetamol Look Good in Trial

Paper Citations

  1. . Potential impact of amyloid imaging on diagnosis and intended management in patients with progressive cognitive decline. Alzheimer Dis Assoc Disord. 2013 Jan;27(1):4-15. PubMed.

Other Citations

  1. ARF related news story

External Citations

  1. final decision
  2. draft decision
  3. Institute for Clinical and Economic Review
  4. The National Oncologic PET Registry

Further Reading