Stat News
March 13, 2023
By Ed Silverman

In an unexpected move, the Veterans Health Administration decided that it will widely cover a new Alzheimer’s treatment, even as Medicare has decided to wait for additional data about the medicine before taking the same step.

The VA will provide coverage to veterans who meet agency criteria for using the medicine — called Leqembi — as well as stipulations on the product labeling, according to a statement from Eisai, which is partnering with Biogen to market the treatment. About 80% to 90% of veterans who clear these hurdles may be eligible for the treatment, but the exact number is uncertain, according to an Eisai spokesperson.

The decision comes two months after the Food and Drug Administration approved the medicine based on clinical trials  showing Leqembi moderately slows cognitive decline in people with mild cognitive impairment or early-stage disease. There are, however, some risks of swelling and bleeding in the brain that require monitoring.

Notably, the FDA endorsed the drug under a designation called accelerated approval. The agency typically grants such an approval when a drug that is designed to treat a serious disease has few alternatives, but also has an uncertain benefit. Significantly, accelerated approval requires companies to conduct another clinical trial of their medicines before full approval can be considered.

The FDA decision followed a searing controversy over an earlier Alzheimer’s treatment from Biogen, which produced data that some experts considered insufficient for approval. Meanwhile, Biogen and FDA officials maintained unusually close contacts to push the drug forward. And the FDA also told its advisory committee it would not consider a surrogate endpoint for approval, but did so anyway.

The episode tarnished the FDA and raised questions about its approval process, prompting a congressional investigation that yielded a scathing rebuke. As a result, the FDA took a more cautious approach toward Leqembi, especially since the drug has safety risks that caused a prominent physicians group to urge the FDA to hold an expert panel meeting before granting full approval.

Meanwhile, the Centers for Medicare and Medicaid Services restricted coverage of Leqembi by using the same criteria — known as a national coverage determination — that were applied last year to Aduhelm, the Alzheimer’s treatment developed by Biogen. An NCD is an extraordinary step taken to determine whether a medicine is “reasonable and necessary.”

CMS applied the criteria to all monoclonal antibody drugs that target amyloid, a protein believed responsible for Alzheimer’s. And the agency has held fast to this position. Last month, CMS rejected requests from the American Academy of Neurologists and the Alzheimer’s Association, a leading patient advocacy group, to widen coverage for Leqembi.

Instead, CMS officials are waiting for full FDA approval.

For this reason, there seemed to be little expectation the Veterans Administration would cover Leqembi. The agency published a guide on its formulary saying coverage will extend to any veteran who meets specified criteria, including an MRI scan within the previous year, amyloid PET imaging consistent with Alzheimer’s and a staging test indicating mild Alzheimer’s dementia. There is also a long list of criteria that would exclude veterans.

The agency can negotiate prices for drugs, but the price it will pay for Leqembi was not listed and the Eisai spokesperson did not offer a cost. Leqembi has an annual wholesale price of $26,500, although the Institute for Clinical and Economic Review recently said the treatment should cost between $8,900 and $21,500 per year to be considered cost effective.

A spokesperson for the VA said the decision was made by VA National Formulary Committee. “Based on the clinical evidence that was available at the time of the FDA’s accelerated approval, the committee believes that there are potential patients who could benefit from this therapy,” he said, He added that the “VA will continue to monitor the clinical evidence and safety data for this agent and adjust the criteria for use as appropriate,” and is able to “conduct real-time medication-use evaluations that will allow for continued safe use of the medication.”

A large swath of patients may qualify for treatment. Approximately 12% to 18% of people age 60 or older are living with mild cognitive impairment, and 10% to 15% develop dementia each year, according to the Alzheimer’s Association. The VA spokesperson said there were estimated to be nearly 168,000 enrolled VA patients with Alzheimer’s dementia in 2022.

Not surprisingly, patient advocacy groups were pleased that the agency is providing coverage.

“This is an important step toward access to medically necessary and beneficial treatments for people living with mild cognitive impairment and Alzheimer’s, a fatal disease,” said Joanne Pike, who heads the Alzheimer’s Association, in a statement. “Each day matters to someone living with early stage Alzheimer’s when it comes to slowing the progression of this disease and for our nation’s veterans living with Alzheimer’s, today is a good day.” However, the group disagrees with some criteria set by the agency without being specific.

“We are thrilled to see that the VA respects the FDA’s approval process, and we hope CMS follows their lead,” Susan Peschin, who heads the Alliance for Aging Research, wrote us. “The decision to treat is between doctors and people living with mild cognitive impairment and early dementia due to Alzheimer’s—CMS has no business using its coverage authority to take that option away.”

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