The Centers for Medicare and Medicaid Services will formally consider whether to adopt nationwide rules for coverage of Biogen's new Alzheimer's disease drug Aduhelm, announcing Monday the opening of a process that could shape how millions of Americans are able to access the controversial treatment.
Patient advocacy groups and insurers have urged CMS to set a single Medicare policy on coverage of Aduhelm, which Biogen priced at an average cost of $56,000 per year. Experts fear the drug — the first new Alzheimer's treatment approved in nearly two decades and the first ever meant to treat the disease's underlying cause — could add tens of billions of dollars to Medicare spending without clear evidence it actually improves patients' lives.
CMS hasn't yet decided whether it will set a universal policy, known as a national coverage determination. The agency will convene two meetings this month to solicit input, as well as collect comments from the public over the next 30 days.
After gathering information, CMS will post a proposed policy, on which it will ask for further comment over another month-long period. Sometime thereafter, a final determination would be made, a process that CMS estimates will complete within nine months.
"We want to consider Medicare coverage of new treatments very carefully in light of the evidence available," said CMS Administrator Chiquita Brooks-LaSure in a Monday statement. "That's why our process will include opportunities to hear from many stakeholders, including patient advocacy groups, medical experts, states, issuers, industry professionals, and family members and caregivers of those living with this disease."
CMS has previously issued national coverage determinations for cancer cell therapy, the heart failure drug Natrecor and an immunotherapy called Provenge, as well as for Alzheimer's diagnostic tests.
Many of the estimated millions of Americans living with Alzheimer's are over 65 and would therefore likely be covered through Medicare for treatment with Aduhelm. Biogen estimates about half of patients treated would have a cap on their out-of-pocket expenses, while about 10% could pay roughly 20% of the drug's cost, or just over $11,000 a year.
Coverage is currently is set regionally by 12 insurers that contract with CMS. Biogen has not said how many patients have received Aduhelm since approval, although the first was treated about nine days after the FDA's clearance.
The Alzheimer's Association, which advocated for Aduhelm's approval, has pushed for a national coverage determination, arguing that leaving policy for a drug like Aduhelm fragmented could limit patient access. Insurers, through their lobby America's Health Insurance Plans, have also indicated their support for a single policy, though their concerns center on Aduhelm's uncertain clinical benefit and potential broad use.
CMS said its consideration of a national policy for Aduhelm would also apply to drugs like it that target a type of brain plaque associated with Alzheimer's. Eli Lilly recently indicated it would ask the Food and Drug Administration for an accelerated approval of its experimental drug donanemab, and Roche is reportedly considering a similar course with another drug.
In a document posted Monday, the agency said it's seeking comment on the degree of benefit patients would find meaningful. Two identical studies of Aduhelm run by Biogen showed contradictory results, with one finding treatment modestly slowed cognitive and functional decline while the other did not.
CMS is also asking for comment on what patient characteristics are likely to correlate with positive health outcomes from treatment, and the preferred setting for treatment and care. In testing, Biogen enrolled patients with mild dementia who were positive for the type of brain plaque Aduhelm targets. The FDA's original approval, granted June 7, did not limit use of the drug to only those patients, and did not recommend obtaining proof of the plaque's presence as a requirement of treatment.
The FDA, after heavy criticism, last week revised the drug's prescribing information, instructing doctors to initiate treatment only for patients with mild dementia. According to Biogen, about 1 million to 2 million people in the U.S. are in that category, roughly 15% to 33% of the estimated 6 million Americans living with Alzheimer's.
Depending on how many people are treated, experts estimate Aduhelm could swell Medicare Part B spending by anywhere from about $6 billion a year to as much as $29 billion, quickly dwarfing what the program pays for any other drug.
Amid uproar over the drug's potential impact on Medicare spending, Biogen said it would work with insurers to "address pricing" should its assumptions on patient population and adoption prove incorrect. The company maintains uptake will be gradual, citing the complexity of Alzheimer's diagnoses and the limited number of specialists.
The Institute for Clinical and Economic Review, a nonprofit group that assesses drug pricing and value, estimates Aduhelm would be cost effective at between $3,000 and $8,400 a year, assuming the drug is as beneficial as Biogen claims its studies show.