The future of the controversial new Alzheimer's drug, Aduhelm, is in the hands of federal officials who are wrestling with a decision.
Medicare, the federal health insurance program for people 65 and over, will make a decision in January on whether to cover the expensive medication. The FDA approval of Aduhelm in June has drawn fierce criticism because of clinical trials showing the drug had significant safety risks and unclear benefit to patients.
The program's coverage decision is crucial because 80% of potential Aduhelm patients are old enough to receive Medicare. Private insurers follow Medicare.
Health policy experts said that Medicare usually pays for F.D.A.-approved drugs for medical conditions on their label. Medicare officials have undertaken a monthslong review that could result in no coverage, full coverage or limited coverage.
James Chambers is a researcher at the Center for the Evaluation of Value and Risk in Health at the Tufts Medical Center.
The administrator of the Centers for Medicare and Medicaid Services, or C.M.S., declined to discuss the deliberations. She said that the coverage team goes through a process. The scientists will be allowed to continue their work.
Aduhelm is a monoclonal antibody made by Biogen. Patients receive it in monthly injections and need M.R.I scans to monitor for potentially dangerous side effects.
The price of Aduhelm was slashed last week by Biogen.
It is a critical time for the Alzheimer's disease community as the Centers for Medicare and Medicaid Services is considering the possibility of coverage of not only Adu. We hope that our actions today will make it easier for patients to access these innovative Alzheimer's treatments.
Many analysts have said that the new price is justified.
The total cost to Medicare depends on the terms it sets and how many patients use Aduhelm. 1.5 million Americans may be eligible because they have mild Alzheimer's-related dementia.
Even at $28,000 a year, Aduhelm would still be one of Medicare's biggest drug expenses.
One of the biggest increases in Medicare Part B premiums in history was imposed before Biogen slashed its prices, partly because of the possibility of Aduhelm coverage.
The price of Aduhelm was slashed by Biogen after weak sales. It is still more expensive than necessary according to many analysts.
Aduhelm is facing criticism and hurdles around the world.
The reviewers at the European Medicines Agency recommended against approving the drug, which Biogen is requesting be re-examined. The leading Canadian Alzheimer's research organizations said that approving Aduhelm in Canada cannot be justified.
Cleveland Clinic, Mount Sinai Health System, and the Department of Veterans Affairs are some of the large American medical systems that do not offer Aduhelm.
Many Medicare beneficiaries would pay thousands of dollars of out-of-pocket costs for a drug with substantial risks and without proven clinical benefit if Aduhelm were covered by Medicare, according to the American Academy of Neurology.
During the evaluation process, Medicare officials met with supporters of coverage, like the Alzheimer's Association.
Biogen said in a statement that they have been engaging with C.M.S. towards the goal of providing access in a way that is sustainable for Medicare beneficiaries and the health care system.
The American Geriatrics Society and the Blue Cross Blue Shield Association wrote to Medicare officials that they did not think aducanumab met the coverage standard.
The F.D.A. greenlighted Aduhelm under a program called accelerated approval, which allows the approval of drugs if there is insufficient evidence that they will help patients.
The agency cited Aduhelm's reduction of amyloid in the brains of people with Alzheimer's. There is little evidence that reducing amyloid slows cognitive decline.
According to Sean Tunis, a former official with C.M.S. who is now a senior fellow, Medicare officials should decide if Aduhelm is a reasonable and necessary treatment.
Aduhelm is administered in monthly injections to patients who must also have M.R.I. scans to monitor for potentially dangerous side effects.
The F.D.A. says there is no evidence of improved cognitive function, so if you go strictly by what the language is, this doesn't meet Medicare's reasonable and necessary criteria. He said that the cognitive benefits have not yet been shown, and that the F.D.A. requirement that Biogen conduct another trial is proof of that.
It will be the first time ever that C.M.S. will refuse to pay for a drug that was approved by the F.D.A. for its on-label indication.
Medicare sometimes denied coverage for medical devices approved by the F.D.A., which can have lower evidentiary standards than for medicines, according to Dr. Chambers. He said that Medicare might be hesitant to do so for a drug. It will be a lot to explain to the people at C.M.S. that they don't happen. That is awkward.
Standards for evaluating Alzheimer's drugs will be established by Medicare.
If Medicare covers Aduhelm, it could be limited to patients with mild Alzheimer's-related cognitive problems, similar to those in the clinical trials. The F.D.A. initially approved Aduhelm for all Alzheimer's patients, but after objections from medical experts, it narrowed the label to patients in mild stages. strict safety monitoring could be imposed by Medicare.
It could place Aduhelm under a framework called Coverage With Evidence Development, which only allows payment for patients in clinical trials or whose data is collected in a registry. It would not prove whether Aduhelm works because it would not compare outcomes for people not receiving Aduhelm.
The high-stakes coverage decision, expected to be finalized by mid-April, has drawn passionate and detailed opinions, reflected in comments posted on the C.M.S. website.
Dr. James Castle, an Illinois neurologist who treats Alzheimer's patients, asked that the medicine not be covered. Send a strong message to the pharmaceutical industry that they need to show proof of efficacy and safety before releasing their medicines on the market.
The F.D.A. acknowledged that there was insufficient evidence that Aduhelm would help patients, but that it allowed authorization under a program for unproven drugs for serious diseases with few treatment options.
The University of Nevada Las Vegas has a research professor who has consulted with Biogen. He wrote that the drug prolongs the mental life of our declining patients, and that Medicare should support coverage for it.
Several people who earned fees from Biogen were among 25 people who signed a letter on the site. Eli Lilly has developed a similar anti-amyloid drug.
One neurologist, Dr. David Weisman, wrote that Medicare should require safety monitoring and medical expertise because he worked with other anti-amyloid drugs. He wrote that the F.D.A. approved Aduhelm, so it should be left to individual patients, family members and physicians.
More people signed comments that opposed coverage or said it should be limited.
The public comment content was not in favor of coverage, and it was surprising how much of it was saying cautionary things. He said that it is more common to have comments in favor of coverage.
Commenters had many reasons for opposing or cautioning. They mentioned that there was inadequate evidence of benefit, that Medicare coverage wouldn't include all treatment costs, that there were unknowns about Aduhelm's effects on nonwhite patients, and that profit-seeking clinics could prey on the desperation of patients.
180 Alzheimer's doctors wrote a letter raising many issues, including the fact that participants in the drug's clinical trials were younger and in earlier stages of cognitive decline.
Dr. Michael Greicius, medical director of the Stanford Center for Memory Disorders, said that he was hoping that January would roll around and they would not cover this. The evidence is not good.
Reed Abelson was involved in reporting.
