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Is Safe and Effective Still the Standard?

By Jim Knaub

In July, the Centers for Medicare & Medicaid Services (CMS) announced its proposal to reimburse beta-amyloid PET imaging only under coverage with evidence development programs that enroll patients in clinical trials addressing the value of these scans for diagnosing Alzheimer’s or other neurological disorders. Florbetapir F18 injection (marketed by Eli Lilly under the trade name Amyvid) was approved earlier this year for imaging amyloid plaque density in patients being evaluated for Alzheimer’s disease.

While the comment period on the July proposal is closed, the final rule has not yet been published. If it reflects the proposal, an FDA-approved agent would not be covered under Medicare and Medicaid programs outside of clinical trials.

It’s also possible that reimbursement status for florbetatir F18 could change from July’s proposal, like it did this past June when the CMS announced it would cover up to three FDG-PET scans for treatment planning—an increase from its earlier proposal to cover just one such scan (and exclude prostate cancer as a covered indication). The CMS also eliminated the requirement for FDG-PET scan recipients to participate in the National Oncology PET Registry to receive reimbursement in most cancer cases, The PET registry rule was to coverage with evidence development but not as restrictive because it only required submitting to the registry, not clinical trial participation. Presumably, as data supporting PET’s role grew, the CMS eased the restrictions for its reimbursable use.

Still, the July proposal shows that the path from FDA approval to routine reimbursement and broad clinical acceptance seems to have become an increasingly winding road. While florbetapir F18 is approved to image plaques, a positive scan for those plaques does not establish an Alzheimer’s diagnosis. Those plaques also have not been proven to predict the development of dementia or other neurologic conditions or monitor the response to therapy.

While many researchers believe the imaging agent is useful for developing a diagnosis for Alzheimer’s and other neurological disorders—and could help in the development of a treatment—that value really hasn’t been proven yet. Broader coverage could speed imaging research to provide that evidence, but to what extent should Medicare and other insurers pay for an approved imaging agent before its value at affecting changing outcomes is proven? That’s a fair question to which I don’t know the answer. The coverage with evidence develoment requirement in the CMS’ florebetatir proposal tries to address that question. Insurers, researchers, manufacturers, clinicians, and patients have differing views on whether that requirement helps or hinders development in diagnosing and treating Alzheimer’s.

It’s also my opinion that in better economic times with less focus on health care’s growing cost, the CMS might see things differently. But we live in a time when cost pressures are intensifying. Consideration of new therapies increasingly will be looked at through the economic prism and the project number of potential Alzheimer’s evaluations is very large. Reimbursement for health care innovation is augmenting the safe and effective designation with a modified version: safe, effective, and cost-effective.

— Jim Knaub is editor of Radiology Today.