Medicare Officially Limits Coverage of Aduhelm to Patients in Clinical Trials

Ever since Medicare proposed to sharply limit coverage of the controversial Alzheimer’s drug Aduhelm, the agency has been deluged with impassioned pleas.

Groups representing patients insisted the federal insurance program pay for the drug. Many Alzheimer’s experts and doctors cautioned against broadly covering a treatment that has uncertain benefit and serious safety risks.

On Thursday, Medicare officials announced their final decision. Though the Food and Drug Administration has approved Aduhelm for some 1.5 million people, Medicare will cover it only for people who receive it as participants in a clinical trial.

Chiquita Brooks-LaSure, the administrator of the Centers for Medicare and Medicaid Services, or C.M.S., said the decision was intended to protect patients while gathering data to indicate whether Aduhelm, an expensive monoclonal antibody given as a monthly infusion, could actually help them by slowing the pace of their cognitive decline.

“It’s our obligation at C.M.S. to really make sure it’s reasonable and necessary,” Ms. Brooks-LaSure said in an interview Thursday. “The vast majority” of the approximately 10,000 comments the agency received on its website, she said, were in favor of “really limiting coverage of Aduhelm to a really controlled space where we could continue to evaluate its appropriateness for the Medicare population.”

Aduhelm’s manufacturer, Biogen, said the decision “effectively denies all Medicare beneficiaries access to Aduhelm,” adding that “Biogen is carefully considering its options and will provide updates as the company further evaluates the business impact of this decision.”

A major issue for Medicare had been how to deal with other similar drugs for Alzheimer’s, several of which are likely to be considered for F.D.A. approval soon. In a proposal in January, Medicare had said it would cover them in the same way as Aduhelm because it typically made coverage decisions for an entire class of drugs.

But after both experts and advocacy groups raised concerns, Medicare officials said Thursday that they would not automatically apply the same restrictions to each new drug. If, unlike with Aduhelm, the F.D.A. finds that there is clear evidence that a drug can help patients, Medicare would cover it for all eligible patients and would only impose a requirement that the patients’ experience be tracked.

Dr. Lee Fleisher, the chief medical officer at the Medicare agency., said the two-track way of dealing with the fast-developing field of Alzheimer’s therapies, a program called Coverage with Evidence Development, “is meant to be nimble and really respond to any new drugs in this class that are in the pipeline, and do demonstrate clinical benefit.”

The decision is extremely unusual for Medicare, which almost always automatically pays for drugs that the F.D.A. has approved, at least for the medical conditions designated on labels.

But Aduhelm’s path has been very unusual, too. The F.D.A. itself acknowledged that it was unclear if the drug was beneficial when it approved Aduhelm last June, authorizing it for people with mild Alzheimer’s-related cognitive decline.

The clinical trial evidence reviewed by the F.D.A. showed that patients in one trial of Aduhelm appeared to experience slight slowing of cognitive decline, while patients in a nearly identical trial didn’t appear to benefit at all. About 40 percent of patients on the dosage later approved experienced brain swelling or brain bleeding, often mild, but sometimes serious. Both a council of senior F.D.A. officials and the agency’s independent advisory committee had said there wasn’t enough evidence for approval.

Instead of giving the drug full approval, the F.D.A. greenlighted it under a program called “accelerated approval,” which allows authorization of drugs that have uncertain benefit if they are for serious diseases with few treatments and if the drug affects a biological mechanism in a way considered reasonably likely to help patients.

The agency’s justification was that Aduhelm targets a protein, amyloid, that forms plaques in the brains of Alzheimer’s patients. But many Alzheimer’s experts say that years of data have not shown that reducing amyloid can slow cognitive decline.

Questions about the approval, and whether the F.D.A. worked too closely with Biogen, have prompted investigations by congressional committees, the Health and Human Services department’s inspector general, the Federal Trade Commission and the Securities and Exchange Commission. Major medical centers, including the Cleveland Clinic, have declined to offer Aduhelm.

As a result of concerns raised by Alzheimer’s experts and some groups, Medicare officials announced several other changes to their earlier proposal. Instead of requiring randomized controlled trials approved by C.M.S., Medicare will cover participants in any trial approved by the F.D.A. or the National Institutes of Health. It will allow those trials to be conducted in a broader array of locations, not just hospital settings, and to include people with other neurological conditions like Down syndrome, many of whom develop Alzheimer’s but had been excluded from the earlier proposed plan.

The trials will still need to comply with a Medicare requirement to recruit a racially and ethnically diverse group of participants, contrasting with the previous trials of Aduhelm, in which most participants were white.

In the trials, “the manufacturers will have to come to us with how are they going to include all patients that represent the Medicare population, and how are they going to ensure that all of these patients are getting appropriate medical treatment and monitoring of their treatment while they’re in each of these studies,” Tamara Syrek Jensen, the director of coverage and analysis for the Medicare agency’s Center for Clinical Standards and Quality, said in an interview.

The F.D.A. has also required Biogen to conduct another clinical trial to determine if the drug provided any evidence of benefit, but it said that in the years it will take for that trial to be completed, Aduhelm would be available to patients. Under Thursday’s decision, Medicare would cover the costs for participants in Biogen’s trial.

In a statement after the Medicare announcement, the F.D.A. said, “At the end of the day, both agencies have a shared goal of ensuring that safe and effective medical products are available for Americans.”

Medicare’s coverage evaluation team makes decisions without considering the cost of a drug, but the Aduhelm decision could ease some concerns about how covering the drug might affect the pocketbooks of the country’s millions of Medicare beneficiaries.

Last year, 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 coverage for Aduhelm, which at the time was priced by its manufacturer at $56,000 a year.

Since then, Biogen, facing weak sales of the drug after many hospitals and doctors would not prescribe it, lowered the price to $28,800 a year, still much higher than many analysts have said is warranted.

Xavier Becerra, secretary of health and human services, had said that he would consider lowering premiums after the final coverage decision for Aduhelm was made, adding that “We’re going to make sure that seniors don’t pay more than they have to.”

In the interview Thursday, Ms. Brooks-LaSure, the C.M.S. administrator, said, “The secretary told us to look at it, and we are going to engage in the process of reviewing the Part B premium.”

Advocacy groups, several of which receive some funding from Biogen and other pharmaceutical companies, had campaigned vigorously for broad Medicare coverage. These groups said patients should be able to decide with their doctors whether to try an F.D.A.-approved drug and claimed it was discriminatory to only reimburse participation in clinical trials that may not be easily accessible to many patients.

“We just can’t let it stand as it is,” Harry Johns, the chief executive of the Alzheimer’s Association, told the organization’s staff, according to a recording of the meeting obtained by The New York Times.

After the Medicare decision was announced on Thursday, Mr. Johns said the association was still evaluating it, but he added, “At initial review we are very disappointed with the immediate impact it will have on Americans living with Alzheimer’s and their families today. While we note some of the recommendations provided by people living with Alzheimer’s and the Alzheimer’s Association have been incorporated into the C.M.S. decision, denying access to FDA-approved Alzheimer’s treatments is wrong.”

Medicare officials said the decision was an attempt to provide what they consider important limits on Aduhelm’s coverage, while not necessarily consigning future anti-amyloid monoclonal antibody drugs to similar restrictions.

If another drug in that class were to win full, or traditional, F.D.A. approval, which usually requires two convincing clinical trials, that would signal that there is persuasive evidence that the drug can help patients and that its benefits outweigh its risks, Medicare officials indicated.

“If a drug were approved under traditional approval tomorrow, we are ready,” Ms. Jensen said, adding that such a medication would be available “in a real-world setting” and patients would be enrolled in a registry or another program that would allow Medicare to monitor whether they are benefiting from the medication.

“There is such a need to really understand what is happening that we want to make sure that we are providing all of that additional or appropriate clinical care,” Ms. Brooks-LaSure, Medicare’s administrator, said. “So, we’re going to make sure that…