Alzheimer’s patients 'devastated' at Medicare limits on drug critics say needs more testing

Michele Hall was convinced she was on the right path in December when she got her first dose of Aduhelm.

The new Alzheimer’s drug gave the 54-year-old Florida woman hope it could slow her early-stage memory and thinking problems. But about a week before she was scheduled to get a second dose last month, the Centers for Medicare and Medicaid Services delivered startling news: It planned to limit coverage of the first new Alzheimer's drug in nearly two decades to people enrolled in clinical trials.

Even if she could convince a health care provider to administer Biogen's $28,200-per-year drug, she might need to pay for the medication herself.

“We were extremely disappointed and devastated by this decision,” said husband Doug Hall. “Right now is the time where it can be really helpful to Michele and lots of people like her.”

Biogen and other drug companies seeking Food and Drug Administration approval for a class of Alzheimer's drugs known as monoclonal antibodies are furiously contesting Medicare’s preliminary decision to pay for the drugs only in clinical trials. The drug companies and their allies urged families and supporters to pressure Medicare to tear up its plan and offer full coverage.

Others say Medicare's proposal to further evaluate Aduhelm in clinical trials is a prudent move. The FDA's accelerated approval last year was controversial because it was based on two studies that yielded mixed results. The FDA’s outside expert advisers recommended against approval, and when the agency approved the drug anyway, three advisers quit in protest.

Biogen faced withering criticism when it charged $56,000 per year for the drug. The Cambridge, Massachusetts, company slashed the price in half, but critics say it's still too expensive for a drug not proven to slow memory and thinking problems.

Clinical trials show Aduhelm is effective at removing beta-amyloid, a sticky protein that forms clumps and plaques in the brains of Alzheimer’s patients. But two large trials reported contradictory results on whether the drug slowed clinical decline from Alzheimer’s, a disease that afflicts more than 6 million Americans.

What's more, the trials showed patients treated with Aduhelm had side effects such as brain swelling, minor bleeding and headaches while taking the medication.

Advocates, critics press Medicare's coverage limits

Major health systems such as Cleveland Clinic and Mount Sinai don't recommend the drug, and private insurers also have balked at paying for the medication. The U.S. Department of Veterans Affairs broadly recommends not using the drug except for "highly selected patients" at VA centers "that have the necessary diagnostic and management expertise."

Because most Alzheimer's patients are on Medicare, the federal program's proposal to limit Aduhelm to clinical trials will affect coverage for those who are most likely to get the drug.

The Centers for Medicare and Medicaid Services Chief Medical Officer Lee Fleisher cited "potential for harm and important questions" that must be addressed through additional clinical trials.

The agency solicited public feedback and was flooded with responses from supporters and detractors during a 30-day public comment period that closed on Feb. 10. The centers received 19,914 comments from doctors, patients, advocates, detractors and others about the proposal to limit coverage.

Many comments appeared to be gathered in an organized fashion. More than 4,500 commenters used the same phrase, "eviscerated the agency's standards," when describing the FDA's accelerated approval of the drug.

More than 3,000 comments came from groups or individuals representing Down syndrome patients criticizing the Centers for Medicare and Medicaid Services' coverage limitation. About 30% of Down syndrome patients in their 50s have Alzheimer's. Aduhelm is not among Alzheimer's drugs that have been tested on Down syndrome patients.

Medicare's preliminary decision already has influenced other drugmakers. Eli Lilly makes an Alzheimer's drug called donanemab, a monoclonal antibody that also would be assigned to post-approval clinical trials under Medicare's proposal. Originally expected to seek FDA approval by March, Eli Lilly this month said it would delay its completed application until later this year.

A Genentech official told the Centers for Medicare and Medicaid Services that its policy would potentially jeopardize patients' access to future drugs without considering the evidence. Owned by pharmaceutical giant Roche, Genentech has studied two Alzheimer's drugs, gantenerumab and crenezumab, in late-stage clinical trials.

Dr. Peter Whitehouse, a neurologist at Case Western Reserve University who led a group of Alzheimer's experts who called on the FDA to withdraw its approval of Aduhelm, said he expects the Centers for Medicare and Medicaid Services might soften its coverage decision. Other amyloid-targeting monoclonal antibodies work differently than Aduhelm and centers must be "careful about being too restrictive and too broad."

Still, he said the controversy has raised important questions about how the FDA approved Aduhelm.

"In this case, CMS stepped up and did the FDA's job," Whitehouse said.

Patients and some doctors are uneasy about Medicare's proposal, said Dr. Stephen Salloway, a professor of neurology and psychiatry at Brown University who directs a memory and aging program at Butler Hospital in Providence, Rhode Island.

“This decision is not only going to limit access severely, but it’s also going to stifle innovation,” said Salloway, who was involved in early clinical trials of Aduhelm and has treated more than two dozen patients with the drug since it gained FDA approval. “Companies are unsure. Patients are unsure. Can they get the drug?”

First step toward Alzheimer's treatment?

Hall, an attorney, formerly served as general counsel for a sheriff's office with more than 1,000 employees. Before she retired in 2019, she began to notice minor lapses such as missed appointments or using incorrect words when talking with judges.

She initially thought the lapses might have been stress related. Her outlook changed following a routine doctor's appointment. The administrative staff handed her paperwork to fill out. The forms didn't make any sense.

That started a yearlong journey of tests and medical appointments before she learned she had mild cognitive impairment, a diagnosis confirmed in late 2020 when a spinal tap revealed tau and beta-amyloid proteins, telltale markers of the disease.

Hall took other medications to manage symptoms, but none has slowed the effects of the disease. That's why she became hopeful when she took her first dose of Aduhelm, administered via IV, on Dec. 28 at a medical clinic in St. Petersburg, Florida.

The Halls signed a financial responsibility form guaranteeing payment before she took the first dose. The clinic submitted a claim to Medicare for payment.

Before she was scheduled to get her second dose four weeks later, the clinic informed the Halls about Medicare's proposal to limit the drug to clinical trials. They might qualify for Biogen's interim dose program offering up to three free or discounted doses. The details took weeks to work out, delaying her second dose until Feb. 11. She is scheduled to return for a third dose in March.

The Halls have no idea whether she will continue to get access to the drug when Medicare finalizes its decision in April. They're willing to pay for the drug if necessary, but they are not certain that will be an option.

If Medicare's proposal to pay for the drug through clinical trials is finalized, that could mean her only option is to enroll in a randomized clinical trial where she could get the drug or a placebo. In other words, her odds of getting the drug are reduced to a coin flip. And if she gets a placebo over the 18 months it takes to complete the clinical trial, the Halls worry her condition might worsen to the point that no drug would be able to slow the disease.

She realizes the drug might not be the final answer. She knows the contradictory results of the clinical trials, but she views the drug as a first step. If it doesn't work, maybe the next drug will.

"This drug might not be the savior," Doug Hall said. "Our hope was to be on this for some period of time and move to the next one, then the next one that can really do more damage against this disease."

Ken Alltucker is on Twitter as @kalltucker or can be emailed at alltuck@usatoday.com.

This article originally appeared on USA TODAY: Medicare wants more tests for Alzheimer's drug; patients ‘devastated’