Medicare Officially Limits Coverage of Aduhelm to Patients in Clinical Trials

For the past few months, Medicare officials have been inundated with impassioned pleas about how to handle coverage of the controversial new Alzheimer’s drug Aduhelm.

Advocacy groups for patients have said the federal insurance program for people 65 and over must pay for a drug approved by the Food and Drug Administration. Many Alzheimer’s doctors and experts cautioned against broadly covering a treatment that scientific evidence shows has uncertain benefit and serious safety risks. Individual patients and families weighed in on both sides with emotional statements.

On Thursday, Medicare officials announced their final decision. The program will cover Aduhelm only if people receive it as participants in a clinical trial, likely a small percentage of the estimated 1.5 million people in the United States who have mild Alzheimer’s-related cognitive decline, the condition Aduhelm was approved to treat.

Chiquita Brooks-LaSure, the administrator of the Centers for Medicare and Medicaid Services 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.”

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, C.M.S. 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 C.M.S., 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. It greenlighted the drug 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 clinical trial evidence reviewed by the F.D.A. showed that patients in one trial 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.

Questions about the approval, and whether the F.D.A. worked too closely with Biogen, Aduhelm’s manufacturer, 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 C.M.S.’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.

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.

In an interview before the Medicare announcement, Mr. Johns indicated that the association wouldn’t be satisfied if Medicare’s restrictions applied only to Aduhelm, saying, “We absolutely believe there is sufficient evidence to provide coverage for the first approved treatment.”