CMS is proposing to limit coverage of beta amyloid positron emission tomography to help diagnosis dementia and neurodegenerative disease only to patients enrolled in qualifying clinical trials. The decision is a setback for Eli Lilly & Co. and its PET imaging agent Amyvid.

Last July, Lilly requested that CMS reconsider its national non-coverage policy for beta amyloid PET scans. CMS initiated a national coverage analysis on the topic in October. (See Also see "CMS Urged To Cover Beta Amyloid PET Scans For Alzheimer's, But Cautiously" - Medtech Insight, 12 November, 2012..) The process included a review by the Medicare Evidence Development and Coverage Advisory Committee, which was not convinced that there is adequate evidence to support coverage of beta amyloid PET. (See Also see "Medicare Panel Finds Little Support For Amyloid PET Imaging Changes" - Medtech Insight, 4 February, 2013..)

Amyvid (florbetapir F 18 injection) is the only imaging agent approved by FDA for use in beta amyloid PET. Sales of the product have been slow due to the lack of Medicare reimbursement, a situation Lilly was hoping to address through its coverage request. A beta amyloid PET scan with Amyvid is indicated for use in adults with thinking or memory problems who are being assessed for possible Alzheimer’s disease.

Evidence is insufficient to conclude that beta amyloid PET improves health outcomes, CMS says, but it may be a promising tool to exclude Alzheimer’s disease in certain instances and to “enrich” clinical trials for Alzheimer’s treatments or prevention strategies.

In its proposed decision memo released July 3, CMS “proposes that the evidence is insufficient to conclude that the use of positron emission tomography (PET) amyloid-beta (Aβ) imaging improves health outcomes for Medicare beneficiaries with dementia or neurodegenerative disease, and thus PET Aβ imaging is not reasonable and necessary” for coverage under the program.

However, CMS is proposing to cover the procedure under a very limited coverage-with-evidence-development policy.

The proposed memo explains that “there is sufficient evidence that the use of PET Aβ imaging could be promising in two scenarios: (1) to exclude Alzheimer’s disease (AD) in narrowly defined and clinically difficult differential diagnoses, such as AD versus frontotemporal dementia (FTD); and (2) to enrich clinical trials seeking better treatments or prevention strategies for AD, by allowing for selection of patients on the basis of biological as well as clinical and epidemiological factors.”

CMS therefore proposes to cover one beta amyloid PET scan per patient for those who are enrolled in CMS-approved clinical studies.

“Clinical study objectives must be to (1) develop better treatments or prevention strategies for AD, or, as a strategy to identify subpopulations at risk for developing AD, or (2) resolve clinically difficult differential diagnoses (e.g., frontotemporal dementia versus AD) where the use of PET Aβ imaging appears to improve health outcomes,” the memo states.

It says the studies must be approved by CMS; involve subjects from appropriate populations; be comparative, prospective and longitudinal; use randomization; and also use postmortem diagnosis as the endpoint where appropriate. And, the radiopharmaceuticals used in the studies must be approved by FDA.

In addition, CMS says the studies must address at least one of three questions:

“Do the results of PET Aβ imaging lead to improved health outcomes? Meaningful health outcomes of interest include: avoidance of futile treatment or tests; improving, or slowing the decline of, quality of life; and survival.”
“Are there specific subpopulations, patient characteristics or differential diagnoses that are predictive of improved health outcomes in patients whose management is guided by the PET Aβ imaging?”
“Does using PET Aβ imaging in guiding patient management to enrich clinical trials seeking better treatments or prevention strategies for AD, by selecting patients on the basis of biological as well as clinical and epidemiological factors, lead to improved health outcomes?”

Industry Reaction

Lilly had been preparing for a negative outcome from CMS, but the company still expressed disappointment with the agency’s proposal. (See [A#14130516006].)

“CMS appears to be challenging the value of an adjunctive tool that can assist physicians in making a more informed diagnosis for patients with cognitive impairment. Restricting coverage could hinder a timely and accurate diagnosis," Daniel Skovronsky, president and CEO of the Lilly subsidiary Avid Radiopharmaceuticals Inc., said in a July 8 statement. “In addition, it may stifle future innovation aimed at improving diagnosis,” he said.

The Medical Imaging and Technology Alliance also criticized the proposal. “We are encouraged by CMS’ acknowledgement that beta amyloid PET can exclude the possibility of Alzheimer’s disease. However, we are concerned that CMS considers the existing evidence inadequate for coverage for all Medicare beneficiaries,” said Gail Rodriguez, executive director of MITA, in a July 9 statement.

Rodriguez argues that evidence used to support appropriate-use criteria developed by a taskforce from the Alzheimer’s Association and the Society of Nuclear Medicine and Molecular Imaging for beta amyloid PET is sufficient to support broader Medicare coverage.

“We are concerned about the unintended consequence of this decision impacting both referring physicians and Medicare beneficiary access to this diagnostic technology,” she noted.

The Alzheimer’s Association, a patient advocacy group, also expressed disappointment with the proposal, noting that it would seek to educate CMS “about the benefits of coverage for brain amyloid imaging” during the public comment period.

Comments on the proposed decision are due Aug. 2. CMS plans to issue a final determination by Oct. 1.

[Editor’s note: This story was contributed by “The Pink Sheet” Daily, providing daily, in-depth analysis of the key developments shaping the biopharma industry. www.thepinksheetdaily.com.]

Trial-Only Medicare Coverage Proposed For PET Alzheimer’s Scans

More from Policy & Regulation

More from Medtech Insight