The Washington PostDemocracy Dies in Darkness

Alzheimer’s groups vow to fight proposed Medicare restrictions on controversial drug

Advocates will join the drug’s manufacturer in arguing the program should broaden coverage for Aduhelm

Phil Gutis sits in front of his toy collection at his home in New Hope, Pa., in May. Gutis was diagnosed with early stage Alzheimer’s disease in 2016. (Rachel Wisniewski for The Washington Post)
Placeholder while article actions load

Alzheimer’s advocacy groups on Wednesday vowed to fight a preliminary Medicare decision to restrict coverage of a controversial new drug to patients in clinical trials, a move that would drastically limit the number of people who could get the treatment. But winning broader coverage could be an uphill fight, experts said.

The Centers for Medicare and Medicaid Services (CMS) on Tuesday proposed covering a $28,200-per-year Alzheimer’s drug called Aduhelm only for Medicare patients enrolled in randomized controlled trials cleared by the agency.

The proposal, if finalized, would have major implications for patients, pharmaceutical companies, insurers, the Food and Drug Administration, Medicare and Medicaid. The FDA approved the drug in June for anyone with mild cognitive impairment or dementia due to early-stage Alzheimer’s disease.

The CMS draft ruling reflects continued doubts about the effectiveness of Aduhelm and concerns about its safety — including among many doctors and medical centers that have declined to offer it. But advocacy groups and Biogen, the biotech giant that makes the drug, already are planning a push for more generous coverage for what they believe is the first treatment that can affect disease progression, not just symptoms.

“The Alzheimer’s Association will use all avenues of communication to ensure those affected, the broader public and the administration truly understand the ramifications of this unprecedented and terrible draft decision that usurps FDA’s role in drug approvals,” Harry Johns, chief executive officer of the Alzheimer’s Association, said in a statement to The Washington Post.

For Massachusetts-based Biogen, which already was hatching cost-cutting plans amid abysmal sales of Aduhelm, the Medicare draft decision spells danger. The company plans to encourage patients and doctors to file protests about the CMS proposal during a 30-day public comment period, according to a company email obtained by Endpoints News, a trade publication. CMS officials, who opened the comment period Tuesday, said they plan to announce a final decision by April 11.

CMS sometimes broadens coverage in final decisions, but Sean R. Tunis, a former top CMS official, said that might be less likely in this case. That’s because so many medical centers and experts oppose full coverage and support a CMS-overseen clinical trial to generate more evidence.

“We haven’t really seen that alignment before” in the case of an FDA-approved drug, he said. In 2019, CMS proposed similar restrictions for a novel cancer treatment called CAR T-cell therapy, but an outcry from medical centers and cancer doctors who wanted to use the drug pressured the agency to drop the curbs.

“With CAR T, there was a strong feeling that it was ready for clinical use,” and it was not worth the additional effort and cost to conduct trials for CMS, Tunis said.

The FDA in June granted Aduhelm accelerated approval based on a “surrogate marker” — a sharp reduction in amyloid plaque in the brain that the agency said would probably result in clinical benefits to patients, such as a slowing of cognitive decline. The approval was not based on evidence showing a benefit. The approval, along with reports about staff interactions with Biogen, set off a furor and led to an ongoing investigation by the inspector general of the Department of Health and Human Services.

CMS officials said Tuesday they wanted to determine whether the drug produces clear benefits — something they said was especially important because the treatment can cause serious side effects, including brain swelling and bleeding, and dizziness and falls.

On a call with reporters, CMS officials acknowledged the agency doesn’t yet have estimates on the number of Medicare patients who would probably enroll in the clinical trials. That would depend on how the trials are set up, according to Tamara Syrek Jensen, director of CMS’s coverage and analysis group. But outside experts said it would probably be hundreds or a few thousand, at most.

CMS officials also stressed the proposal didn’t take cost into account when making decisions on how — and whether — Medicare will cover a drug. Earlier this week, HHS Secretary Xavier Becerra directed CMS to consider lowering this year’s 14.5 percent hike for premiums for Part B, which covers outpatient care, given that drugmaker Biogen nearly halved the drug’s initial price tag, to $28,200.

It’s extremely unusual for the FDA, which assesses products’ safety and effectiveness, and CMS, which scrutinizes whether treatments are reasonable and necessary, to go in such radically different directions, especially on a drug.

The discrepancy had some advocates fuming. HHS, the parent agency of FDA and CMS, “is clearly at war with itself, with one agency approving this class of drugs and another slamming the door shut on treatment,” said George Vradenburg, chair and co-founder of UsAgainstAlzheimer’s. “It’s outrageous. Does CMS no longer trust the FDA’s work?”

Others applauded CMS’s insistence on using its own independent standards.

If the proposed decision is finalized, CMS would issue criteria for clinical trials, which would be randomized controlled trials, with some patients getting placebos. In trials that qualify, Medicare participants would be eligible to receive coverage of the drug, related services and other routine costs, which may include brain scans to check for amyloid in the brain.

Aduhelm is a monoclonal antibody — a kind of laboratory-made protein that binds to a substance, in this case beta amyloid, which is associated with Alzheimer’s.

Drug companies, including Roche, Eli Lilly and Eisai, which is partnering with Biogen, are developing other monoclonal antibodies for Alzheimer’s that target amyloid plaques. The CMS draft policy was billed as applying to the entire class of amyloid-busting antibodies, but experts Wednesday said more clarity was needed on exactly what that meant.

Some wondered whether such restrictive coverage would be applied to other monoclonal antibodies, if they have stronger efficacy data.

“A key area for further discussion and clarity is what are the evidence requirements for coverage of other drugs in this class,” said Mark McClellan, who headed CMS and FDA during the George W. Bush administration. Clinical trials, he said, are time-consuming and expensive to conduct. Since major trials are already underway and some questions can be addressed in studies after approval, it’s important for study designers to know whether they are meeting FDA and CMS requirements, he said.

Rachel Sachs, a law professor at Washington University in St. Louis, said the proposed decision will affect other players. The ruling could impose extra costs on Medicaid, the state-federal program for the poor and elderly, because Medicaid, unlike Medicare, is required to cover all FDA-approved drugs.

State Medicaid programs’ “financial exposure goes up if they are on the hook for the full cost” of Alzheimer’s patients enrolled in both Medicare and Medicaid, Sachs said. “So it’s a big financial issue for states.”

Rachel Roubein contributed to this report.