Decision looms that could determine fate of Alzheimer's drug

Federal officials are wrestling with a decision that could go a long way toward determining the future of the controversial new Alzheimer’s drug, Aduhelm, and whether significant numbers of patients use it.اضافة اعلان

In January, Medicare, the federal health insurance program for people 65 and older, plans to issue a preliminary decision on whether it will cover the expensive medication. The Food and Drug Administration’s approval of Aduhelm in June has drawn fierce criticism because clinical trials showed the drug had significant safety risks and unclear benefit to patients.

Roughly 80% of potential Aduhelm patients are old enough to receive Medicare, making the program’s coverage decision crucial. Private insurers often follow Medicare’s lead.

Medicare almost always pays for Food and Drug Administration-approved drugs, at least for the medical conditions designated on their label, health policy experts said. But with Aduhelm, Medicare officials have undertaken a monthslong review that could result in no coverage, full coverage or limited coverage.

“It’s truly unprecedented,” said James Chambers, a researcher at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center in Boston.

Chiquita Brooks-LaSure, administrator of the Centers for Medicare & Medicaid Services, declined to discuss the deliberations. “It’s a process our coverage team goes through,” she said. “We will let the scientists continue their work.”

Aduhelm, made by Biogen, is a monoclonal antibody also known by its scientific name, aducanumab. Patients receive it in monthly infusions and also require regular MRI scans to monitor for potentially dangerous side effects, including brain swelling and bleeding.

Last week, after weak sales, Biogen slashed Aduhelm’s price to $28,200 a year from $56,000.

In a statement, Michel Vounatsos, Biogen’s CEO, said, “It is a critical time for the Alzheimer’s disease community as the Centers for Medicare & Medicaid Services (CMS) is considering the possibility of coverage of not only Aduhelm, but also this entire new class of Alzheimer’s disease therapies. We hope our actions today will facilitate patient access to these innovative Alzheimer’s treatments.”

The new price is still much higher than many analysts have said is warranted.

The total cost to Medicare would depend on the terms it sets and how many patients decide to use Aduhelm. About 1.5 million Americans may be eligible because they have mild Alzheimer’s-related dementia.

“While it of course depends on Aduhelm’s utilization, it seems that even at $28,000 a year, Aduhelm would still be one of Medicare’s biggest drug expenses,” Chambers said.

Before Biogen’s price cut, Medicare’s actuarial division, acting without knowing what the coverage decision would be, imposed one of the biggest-ever increases in Medicare Part B premiums for 2022, partly driven by the possibility of Aduhelm coverage.

Medicare’s review comes as Aduhelm faces criticism and hurdles around the world.

Reviewers at the European Union’s drug regulator, the European Medicines Agency, recommended against approving the drug, a decision that Biogen is requesting be reexamined. Leading Canadian Alzheimer’s research organizations earlier said that approving Aduhelm in Canada “cannot be justified.”

Large U.S. medical systems, including Cleveland Clinic, Mount Sinai Health System, Mass General Brigham and the Department of Veterans Affairs have declined to offer Aduhelm.

The American Academy of Neurology said recently that if Aduhelm was covered by Medicare, which typically pays 80% of a drug’s price, “many beneficiaries would pay thousands of dollars of out-of-pocket costs for a drug with substantial risks and without proven clinical benefit.”

During the evaluation process, Medicare officials have met with stakeholders, including supporters of coverage, like Biogen and the Alzheimer’s Association, a leading patient group.

“We have been engaging with CMS towards the goal of providing access in a way that is sustainable for Medicare beneficiaries and the health care system,” Biogen said in a statement.

Medicare officials have also met with those that oppose coverage or want it sharply narrowed, like the American Geriatrics Society and the Blue Cross Blue Shield Association, which, during a public comment period this past summer, wrote, “We feel strongly that aducanumab does not meet the statutory coverage standard of ‘reasonable and necessary’ as the current evidence highlights numerous safety risks and uncertain clinical benefits.”

In approving Aduhelm, the FDA acknowledged that there was insufficient evidence that it would help patients but greenlighted it under a program called “accelerated approval,” which allows authorization of unproven drugs for serious diseases with few treatment options if the drug affects a biological mechanism in a way considered “reasonably likely to predict clinical benefit.”

As its justification, the agency cited Aduhelm’s reduction of a protein, amyloid, that forms plaques in the brains of people with Alzheimer’s. But many Alzheimer’s specialists say that extensive research has shown little evidence that reducing amyloid slows cognitive decline.

In evaluating Aduhelm, Medicare officials are supposed to decide if it is a “reasonable and necessary” treatment, a phrase that usually “means adequate evidence of improved health outcomes,” said Dr. Sean Tunis, a former official with CMS who is now a senior fellow at the Tufts center.

“If you go strictly by what the language is, this doesn’t meet Medicare’s ‘reasonable and necessary’ criteria because the FDA themselves says there’s no direct evidence of improved cognition,” said Tunis, who also works as a consultant, including advising Biogen, before Aduhelm was approved, about Medicare’s various options for coverage. He added that the FDA’s requirement that Biogen conduct another trial (which will take years while Aduhelm is available to patients) “implies that the cognitive benefits have not yet been shown.”

If Medicare coverage for Aduhelm is denied, Tunis said, “to my knowledge, it will be the first time ever that CMS has declined to pay for a drug that was approved by the FDA for its on-label indication.”

The high-stakes coverage decision, expected to be finalized by mid-April after a public comment period, has drawn impassioned and detailed opinions, reflected in comments posted on the CMS website.

“Please, please, please, do not cover this medicine,” implored Dr. James Castle, an Illinois neurologist who treats Alzheimer’s patients. “Send a strong and clear message to the pharmaceutical industry that they need to show proof of both efficacy and safety before releasing their medicines on the market.”

Posts supporting coverage include comments from people who have consulted for Biogen, like Dr. Jeffrey Cummings, a research professor in the department of brain health at the University of Nevada, Las Vegas. He wrote that the drug “prolongs the mental life of our declining patients,” adding, “Medicare should unequivocally support coverage for aducanumab, and they should do it as soon as possible.”

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