Federal officials are grappling with a decision that could go a long way in determining the future of the controversial new Alzheimer’s drug, Aduhelm, and whether a significant number of patients are using it.
In January, Medicare, the federal health insurance program for people 65 and older, plans to issue a preliminary ruling on whether to cover expensive drugs. The Food and Drug Administration’s approval of Aduhelm in June drew fierce criticism because clinical trials showed the drug had significant safety risks and uncertain benefits for patients.
About 80 percent of potential Aduhelm patients are old enough to receive Medicare, making the program coverage decision crucial. Private insurers often follow Medicare’s lead.
Health policy experts said Medicare almost always pays for FDA-approved drugs, at least for the medical conditions listed on their labels. But with Aduhelm, Medicare officials conducted a months-long review that could result in coverage, full coverage, or limited coverage.
“It’s truly unprecedented,” said James Chambers, a researcher at the Center for Value and Risk Assessment in Health at Tufts Medical Center.
Chiquita Brooks-LaSure, director of the Centers for Medicare and Medicaid Services, or CMS, declined to discuss the negotiations. “This is a process our coverage team goes through,” he said. “We’ll let the scientists continue their work.”
Aduhelm, made by Biogen, is a monoclonal antibody, also known by its scientific name, aducanumab. Patients take it in monthly infusions and also need regular MRI scans to monitor for potentially dangerous side effects, including: brain swelling and bleeding.
Last week, after weak sales, Biogen lowers the price of Aduhelm, from $56,000 to $28,200 per year.
In DeclarationMichel Vounatsos, Biogen’s CEO, said: “This is a critical time for the Alzheimer’s disease community as the Centers for Medicare and Medicaid Services (CMS) evaluate the possibility of covering not only Aduhelm but also this entire new class. Alzheimer’s disease treatments. We hope our actions today will facilitate patients’ access to these innovative Alzheimer’s treatments.”
new price still much higher than many analysts say.
The total cost of Medicare will depend on the terms it sets and how many patients decide to use Aduhelm. About 1.5 million Americans may be eligible, as they have mild Alzheimer’s-related dementia.
Dr. “While it depends, of course, on the use of Aduhelm, Aduhelm will apparently continue to be one of Medicare’s largest drug expenditures, even at $28,000 a year.”
Prior to Biogen’s price reduction, Medicare’s actuarial division, acting without knowing what the coverage decision would be, One of the biggest-ever increases in Medicare Part B premiums For 2022, partly due to the possibility of Aduhelm coverage.
Medicare’s scrutiny comes as Aduhelm faces criticism and obstacles around the world.
Judges at the European Medicines Agency, the European Union’s drug regulator, advice against approval of the drug, a decision Biogen wants it reexamined. Canada’s leading Alzheimer’s research organizations have previously endorsed Aduhelm in Canada “can’t be right”
This American Academy of Neurology Recently, Aduhelm said, if Medicare were covered, which typically pays 80 percent of the price of a drug, “many beneficiaries would pay thousands of dollars out-of-pocket for a drug with significant risks and no proven clinical benefit.”
During the evaluation process, Medicare officials, met with stakeholders, including supporters of coverage such as Biogen and the Alzheimer’s Association, a leading patient group.
“We are working with CMS towards the goal of providing sustainable access for Medicare beneficiaries and the healthcare system,” Biogen said in a statement. Said.
Medicare officials have also spoken to those who oppose coverage or want coverage to be sharply narrowed, such as the American Geriatrics Society and Blue Cross Blue Shield Association. general comment During this summer, he wrote: “We feel strongly that aducanumab does not meet the ‘reasonable and necessary’ standard of legal coverage as the available evidence highlights multiple safety risks and uncertain clinical benefits.”
In approving Aduhelm, the FDA acknowledged that there was insufficient evidence that it would help patients, but gave the green light under a program called “accelerated approval” that allows unproven drugs to be authorized with few treatment options for serious diseases if the drug affects a biological mechanism. a way that is considered “reasonably likely to predict clinical benefit.”
The agency cited Aduhelm’s reduction of amyloid, a protein that forms plaques in the brains of people with Alzheimer’s. But many Alzheimer’s experts say extensive research shows little evidence that reducing amyloid slows cognitive decline.
When evaluating Aduhelm, Medicare officials need to decide whether it is a “reasonable and necessary” treatment, which often means “sufficient evidence of improved health outcomes,” said Dr. . Sean Tunis. Senior man at Tufts headquarters.
Working as a consultant, Dr. “If you stick to what the language is, that doesn’t meet Medicare’s ‘reasonable and necessary’ criteria because the FDA itself says there is no direct evidence of improved cognition,” Tunis said. Aduhelm advised Biogen about Medicare’s various coverage options before it was approved. He added that the FDA’s requirement for Biogen to run another trial (which will take years while Aduhelm is open to patients) “implies that cognitive benefits have yet to be demonstrated.”
If Medicare coverage for Aduhelm is denied, Dr. “To my knowledge, this will be the first time CMS has refused to pay a drug approved by the FDA for its indication on the label,” Tunis said.
Dr. Chambers said Medicare has at times denied coverage of medical devices approved by the FDA, which can have lower evidence standards for devices than drugs. But he said Medicare may be reluctant to do so for a drug. Since both CMS and FDA are part of the Department of Health and Human Services, “It’s definitely going to be very revealing for people at CMS to say, ‘Yeah, we just disagree with our sister agency,'” said Dr. Tunis. “
Medicare’s decision standards for the evaluation of several similar Alzheimer’s drugs in the pipeline.
If Medicare covers Aduhelm, the agency may limit it to certain types of patients, such as those with mild cognitive problems related to Alzheimer’s, similar to participants in clinical trials. (The FDA initially approved Aduhelm for all Alzheimer’s patients, but after objections from medical professionals, collapsed the tag patients in mild stages.) Medicare may also require administration by certain specialists and may require strict safety monitoring.
Or it could place Aduhelm in a framework called Evidence Enhancement Coverage, which allows payment only for patients who participate in clinical trials or whose data is collected in a registry. Dr. Chambers said that while this would allow to collect information about Aduhelm from patients, it would not prove whether the drug worked because it would not compare outcomes for people who did not take Aduhelm.
The high-stakes reporting decision, which is expected to be finalized by mid-April after a period of public comment, garnered enthusiastic and detailed opinions, which were reflected in the published comments on the subject. IYS website.
An Illinois neurologist who treats Alzheimer’s patients, Dr. “Please, please, please, don’t cover this drug,” begged James Castle. “Send a strong and clear message to the pharmaceutical industry that they must show proof of both efficacy and safety before putting their drugs on the market.”
Posts to support the coverage are by Dr. Includes comments from people who have consulted with Biogen, such as Jeffrey Cummings. He wrote that the drug “prolongs the mental lifespan of our declining patients,” and “Medicare should firmly support aducanumab coverage and do so as soon as possible.”
Dr. Cummings and several others who took charge from Biogen were among the 25 signers of another letter on the site. Supporting comments also came from Infusion Access Foundation officials and Eli Lilly, who is developing a similar anti-amyloid drug.
Aducanumab is not involved in their trials, but is working with other anti-amyloid drugs, says Dr. David Weisman wrote that Medicare requires safety monitoring and medical expertise. But since the FDA has approved Aduhelm, it wrote that whether to use it “should be left to individual patients, family members, and physicians.”
More people signed up for individual or group comments that oppose coverage or say it should be strictly limited.
Advice on what the data might be like, UsAgainstAlzheimer, an advocacy group that supports Aduhelm’s endorsement, is Dr. “It was somewhat surprising that public comment content actually said cautionary things or was not in favor of coverage,” Tunis said. It should be collected if Medicare requires it. “It’s actually more common to have comments that argue in favor of coverage,” he said.
Commentators cited many reasons for opposition or caution. Besides the insufficient evidence of benefits, they cited disparities that could be exacerbated by the fact that Medicare coverage does not include all treatment costs; Unknown about the effects of Aduhelm on non-white patients because most trial participants were white; and the risks that for-profit clinics can feed into the desperation of patients and families.
a letterSigned by 180 Alzheimer’s doctors, it raised numerous issues, including the harm people might experience on aducanumab in the real world outside of a clinical trial setting, given that those who participated in clinical trials of the drug were “younger and in earlier stages of cognitive decline.” , had less comorbidities and were much better followed than patients who would have been under the conditions of broad Medicare coverage.
One of the authors of that letter, the medical director of the Stanford Center for Memory Disorders, Dr. “I’m still quietly hoping January will roll around and they’ll say, ‘Look, we,'” Michael Greicius said. we will not cover it. The evidence is very weak.’”
Reed Abelson contributing reporting.