Medicare Officially Limits Coverage of Aduhelm to Patients in Clinical Trials

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

Patient advocacy groups have said the federal insurance program for people 65 and older must pay for a drug approved by the Food and Drug Administration. Many doctors and Alzheimer’s experts have warned against widespread coverage of a treatment that scientific evidence shows has uncertain benefits 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 only cover Aduhelm 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 suffer from mild cognitive decline related to the disease. Alzheimer’s, the disease Aduhelm has been approved to treat.

Chiquita Brooks-LaSure, 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 rate of their cognitive decline.

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

A major issue for Medicare was how to treat other similar drugs for Alzheimer’s disease, several of which are soon to be reviewed for FDA approval. In a proposal in January, CMS said it would cover them in the same way as Aduhelm, as it typically makes coverage decisions for an entire class of drugs.

But after experts and advocacy groups raised concerns, Medicare officials said Thursday they would not automatically apply the same restrictions to every new drug. If, unlike Aduhelm, the FDA finds that there is clear evidence that a drug can help patients, Medicare would cover it for all eligible patients and impose only a patient experience tracking requirement.

CMS Chief Medical Officer Dr Lee Fleisher said the two-track approach to addressing the rapidly developing field of Alzheimer’s disease therapies, a program called Coverage with Evidence Development, “is meant to be nimble and really respond to any new drugs of this class that are in development and that demonstrate clinical benefit.

The decision is extremely unusual for Medicare, which almost always automatically pays for FDA-approved drugs, at least for medical conditions named on the labels.

But Aduhelm’s journey has also been highly unusual. The FDA itself acknowledged that it was unclear whether the drug was beneficial when it approved Aduhelm last June. He greenlighted the drug under a program called “fast track approval,” which allows drugs with uncertain benefits to be approved if they are for serious conditions with few treatments and if the drug affects a biological mechanism in a way considered reasonably likely to help patients.

Evidence from clinical trials reviewed by the FDA showed that patients in one trial appeared to experience a slight slowing of cognitive decline, while patients in a nearly identical trial did not appear to benefit at all. About 40% of patients receiving the later approved dose experienced brain swelling or bleeding, often mild but sometimes severe. A board of senior FDA officials and the agency’s independent advisory board had both said there was insufficient evidence for approval.

Questions about the approval and whether the FDA was working too closely with Biogen, the maker of Aduhelm, prompted investigations by congressional committees, the inspector general of the Department of Health and Human Services, the Federal Trade Commission and the Securities and Exchange Commission. Major medical centers, including the Cleveland Clinic, refused to offer Aduhelm.

Following concerns raised by Alzheimer’s disease experts and some groups, Medicare officials announced several other changes to their earlier proposal. Instead of requiring CMS-approved randomized controlled trials, Medicare will cover participants in any trial approved by the FDA or the National Institutes of Health. This will allow these trials to be conducted in a wider range of settings, not just in hospital settings, and to include people with other neurological conditions like Down syndrome, many of whom develop Alzheimer’s disease but had been excluded from the previously proposed plan.

The trials will still have to adhere to a Medicare requirement to recruit a racially and ethnically diverse group of participants, unlike Aduhelm’s previous trials, in which most participants were white.

In the trials, “manufacturers will have to tell us how they are going to include all patients who represent the Medicare population, and how are they going to ensure that all of these patients receive appropriate medical treatment and follow-up on their treatment while they are in each of these studies,” said Tamara Syrek Jensen, director of coverage and analysis for the Center for Clinical Standards and Quality at CMS, in an interview.

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

Medicare’s Coverage Assessment Team makes decisions without considering a drug’s cost, but the Aduhelm decision could allay some concerns about how the drug’s coverage could affect the wallets of millions of drug recipients. Medicare in the country.

Last year, Medicare’s actuarial division, acting without knowing what the coverage decision would be, imposed one of the largest-ever increases in Medicare Part B premiums for 2022, in part due to the possibility of coverage. for Aduhelm, which at the time was priced by its manufacturer at $56,000 per year.

Since then, Biogen, faced with weak sales of the drug after many hospitals and doctors won’t prescribe it, has lowered the price to $28,800 a year, still much higher than many analysts have said warranted.

Xavier Becerra, Health and Human Services Secretary, had said he would consider lowering premiums after the final coverage decision for Aduhelm, adding that “We will ensure that older people do not pay more than necessary. ”

In Thursday’s interview, Ms Brooks-LaSure, the CMS administrator, said: “The secretary told us to look into it, and we’re going to go through the Part B premium review process. “

Advocacy groups, many of which receive funding from Biogen and other pharmaceutical companies, have campaigned vigorously for broad Medicare coverage. These groups said patients should be able to decide with their doctor whether or not to try an FDA-approved drug and claimed it was discriminatory to only reimburse participation in clinical trials that might not be readily available to them. many patients.

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

In an interview ahead of Medicare’s announcement, Johns said the association would not be happy if Medicare restrictions applied only to Aduhelm, saying: “We absolutely believe there are enough evidence to cover the first approved treatment.”