If the preliminary decision is finalized this spring, it would restrict coverage to patients in randomized clinical trials.
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Medicare officials said Tuesday that the federal health insurance program should restrict its coverage of the controversial new Alzheimer’s drug Aduhelm to patients who are participating in approved clinical trials, a move that would vastly limit the number of patients who could use the expensive drug.
In a preliminary decision, the Centers for Medicare and Medicaid Services said that after a thorough review of the scientific evidence and the opinions of many different stakeholders, it had concluded that there remain significant doubts about whether the potential benefits of Aduhelm for patients outweigh the safety risks.
As a result, the agency said that it would provide coverage only for patients who receive the drug in randomized controlled trials — considered the gold standard of scientific evaluation because they compare patients receiving a drug with a control group of patients, usually those receiving a placebo.
Dr. Lee Fleisher, the agency’s chief medical officer and director of its Center for Clinical Standards, said Tuesday that the evidence showed that “while there may be the potential for promise with this treatment, there’s also the potential for serious harm to patients. This harm may include headaches, dizziness, falls, and brain bleeds.”
Dr. Fleisher added, “Our foremost goal is to protect beneficiaries from potential harm from an intervention without known benefits in the Medicare population. As a practicing physician, I cannot overemphasize the need to understand the risks and benefits of a given treatment in order to better inform patients and their families as they make decisions about their care.”
In a statement, Biogen, the company that makes Aduhelm, said that the agency’s position “denies the daily burden of people living with Alzheimer’s disease” and that the clinical trial requirement “will exclude almost all patients who may benefit.”
The decision is highly unusual, “the first time that C.M.S. limited Medicare beneficiaries’ access to an F.D.A.-approved drug in this way,” said James Chambers, a researcher at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. The program almost always pays for drugs that the Food and Drug Administration has approved, at least for the medical conditions designated on their label, he and other health policy experts said.
About 1.5 million Americans have the type of condition the F.D.A. approved the drug to treat: mild Alzheimer’s-related cognitive decline. But if the decision becomes final this spring, it could limit the use of the Aduhelm to “perhaps a few thousand patients that enroll in randomized trials over the next three-to-five years,” said Dr. Sean Tunis, a former Medicare official, who is now a senior fellow at the Tufts center.
The restriction, Dr. Tunis said, “effectively means that the vast majority of patients that meet the F.D.A. approval criteria” will not get the drug. “So it’s almost the same as a non-coverage decision,” he said.
The agency’s final decision, expected by April 11 after a public comment period that runs from now until mid-February, would also apply to similar drugs for Alzheimer’s that are currently in trials and expected to be considered for F.D.A. approval.
Aduhelm was approved by the F.D.A. in June, a decision vigorously criticized by many doctors and Alzheimer’s experts because clinical trial results showed it had significant safety risks and unclear benefit to patients.
Congress is investigating whether the F.D.A. worked too closely with Biogen and why Aduhelm was approved despite strong objections from some senior F.D.A. officials and the agency’s independent advisory committee.
So far, only a small number of patients — a fraction of the number Biogen and industry analysts had initially expected — have used Aduhelm, a monoclonal antibody administered as a monthly infusion that is also known by its scientific name, aducanumab.
Many large American health care systems, including the Cleveland Clinic and Mount Sinai, have declined to offer the drug, citing questions about its benefits and risks. Last month, a group of Alzheimer’s experts and health advocates called on the F.D.A. to pull Aduhelm off the market and said they were supporting an effort to file a formal petition with the agency to withdraw it.
The F.D.A. itself acknowledged in its approval of Aduhelm that there was insufficient evidence it would help patients. Instead, it greenlighted the medication — the first new treatment for Alzheimer’s in 18 years — under a program called “accelerated approval,” which allows authorization of unproven drugs for serious diseases with few treatment options if the drug affects a biological mechanism in a way considered “reasonably likely to predict clinical benefit.”
It based the drug’s qualification for accelerated approval on Aduhelm’s reduction of a protein, called amyloid, that clumps into plaques in the brains of people with Alzheimer’s. But many Alzheimer’s specialists say that extensive research has shown little evidence that reducing amyloid slows cognitive decline.
The F.D.A. also required Biogen to conduct another clinical trial to determine if the drug provided any evidence of benefit. In the years it will take for that trial to be completed, Aduhelm will be available to patients.
Medicare’s coverage decision is considered pivotal to the future of Aduhelm, which is priced at $28,200 a year per patient. Roughly 80 percent of the patients eligible for the drug are old enough to receive coverage under Medicare.
Biogen initially priced the drug at $56,000 a year, but slashed the price in half last month after months of weak sales. Before Biogen’s price cut, 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 Aduhelm coverage.
After Biogen’s price cut, advocacy groups for Alzheimer’s patients urged C.M.S. to lower the premium increase. On Monday, the secretary for health and human services, Xavier Becerra, said that he was instructing C.M.S. to re-examine the premium hike “given the dramatic price change of the Alzheimer’s drug, Aduhelm.”
Medicare officials are supposed to determine coverage based on whether a drug is a “reasonable and necessary.,” and in a briefing with reporters, officials said price wasn’t a factor in their decision.
Dr. Fleisher, acknowledging the unusual nature of the decision, said that “C.M.S. is using its authority provided by Congress to determine if the drug is considered reasonable and necessary, meaning that the benefits of improvement of cognition outweigh the harms in the Medicare population.”
Several advocacy groups for patients, including the Alzheimer’s Association, had pushed for approval of the drug and for Medicare coverage, and they expressed strong disappointment in the agency’s preliminary decision.
Harry Johns, chief executive officer of the Alzheimer’s Association, said in a statement that the decision amounted to “shocking discrimination against everyone with Alzheimer’s disease,” adding that “with this approach, access to treatment would now only be available to a privileged few, those with access to research institutions, exacerbating and creating further health inequities.”
Medicare’s proposed criteria for coverage also says the randomized trials must be approved by C.M.S. or the National Institutes of Health. Officials listed a series of criteria that clinical trials would need to follow in order to be considered approved, including that the trials be conducted in a “hospital-based outpatient setting” and that patients have evidence of amyloid in their brains. It said the agency would cover the cost of the main method for diagnosing the presence of amyloid, a PET scan, and that approved trials would also include any that are supported by the National Institutes of Health.
Tamara Syrek Jensen, director of coverage and analysis for C.M.S.’s Center for Clinical Standards and Quality, said that the agency considered it a requirement that the clinical trials include a diverse patient population, in contrast to the mostly white patients who participated in previous trials of Aduhelm. People who are Black or Latino are a greater risk for Alzheimer’s disease.
“Given the disappointing lack of inclusion of underserved populations in past trials, we are requiring a representative population,” Ms. Jensen said, adding “We think this is this is important for this disease.”
The trial that Biogen is required by the F.D.A. to conduct can also apply to the C.M.S. for approval, she said, and if it meets the agency’s criteria, Medicare would cover the costs for participants in that trial.