Medicare will restrict coverage of Biogen's controversial Alzheimer's treatment only to patients who take part in a rigorous clinical trial, the federal government announced Tuesday, a decision that would likely keep use of the drug low if finalized later this year.
After a six-month review, the Centers for Medicare & Medicaid Services said that the study results generated so far by the drug, which won Food and Drug Administration approval last June and is now sold as Aduhelm, were "insufficient to establish that the treatment is reasonable and necessary."
The decision, which is a draft and could be revised, will also cover other drugs that work in the same way to treat Alzheimer's disease. Following the FDA's approval of Aduhelm, Japan's Eisai and then Eli Lilly started submissions of their respective experimental Alzheimer's drugs for clearance based on early trial data.
CMS will collect comments on its proposal over the next 30 days and plans to finalize its decision by April 11.
By approving Aduhelm, the FDA endorsed an much studied, but unproven, scientific hypothesis that removing a sticky protein called amyloid from the brain could slow the loss of cognition and function in Alzheimer's. CMS officials acknowledged it is plausible drugs that do this might be beneficial, but argued the uncertainty needs to be resolved with additional research.
"We do know based on some of the evidence that there may be potential promise with this treatment," Lee Fleisher, CMS' chief medical officer, said in a call with reporters Tuesday. "That's why it is critical for us to pursue additional scientific evidence; that we at CMS make the right decision."
Patients wanting to receive Aduhelm will need to enroll in a "randomized controlled trial," a type of study that selects some patients to receive a placebo. As a result, some patients might be less willing to pursue treatment than they would if Medicare had proposed wider coverage.
Harry Johns, CEO of the Alzheimer's Association, sharply criticized the decision in a statement, calling it "shocking discrimination against everyone with Alzheimer’s disease" that limits treatment to only the "privileged few."
CMS, in formulating its proposal, did not consider Aduhelm's price, which Biogen recently cut in half to $28,500 per year for an average patient. The agency had separately set out plans to raise premiums in Medicare Part B to cover the costs of Aduhelm, a recommendation that Health and Human Services Secretary Xavier Becerra on Monday asked to be reassessed.
The draft determination offers some clarity about the status of the first new Alzheimer's drug approved in two decades, and the first that's meant to treat the disease's underlying cause. Insurers have largely refrained from establishing policies on Aduhelm until Medicare, the government program that covers many of the patients most likely to receive the drug, made its position known.
As a result, relatively few patients have been treated with Aduhelm and sales, which totaled just $300,000 through September, have been dramatically lower than anticipated.
"People can assume that, really, the launch is starting once you have reimbursement," Biogen CEO Michel Vounatsos said earlier this week, during a virtual presentation at J.P Morgan's annual healthcare conference.
The proposed decision by CMS, though not entirely unexpected, isn't a good outcome for Biogen, which had hoped coverage might match the prescribing criteria approved by the FDA.
In a statement, Biogen said the decision would "significantly limit patient access to an FDA-approved treatment" and noted that these types of coverage with evidence development frameworks "can take months to years to initiate."
Analysts at Cowen & Co. had expected the agency would settle on a determination like this as a sort of middleground stance, allowing for the collection of more evidence on Aduhelm while also offering "cover should future data from the class turn out worse than expected."
Yet CMS chose to specify coverage only when patients are enrolled in a randomized clinical trial, the most thorough and prescriptive kind. Another option could have been to cover treatment through studies that follow patient outcomes but don't compare a therapy against a placebo or another drug.
"We have chosen the most rigorous form of coverage with evidence development in covering this class of FDA-approved drugs," said Fleisher. Coverage would also apply to trials run by the National Institutes of Health, CMS said.
Aduhelm's effectiveness had been hotly debated among doctors even before the FDA cleared it for market. A panel of outside experts voted against approval and, when the agency later overruled their advice, several resigned.
Those concerns have persisted in the months since, in turn hampering Aduhelm's sales and adoption. A number of high-profile medical and academic centers, including the Cleveland Clinic and Mount Sinai, have refused to administer the drug.
"Medicare made the right call here," said Howard Fillit, chief science officer at the Alzheimer's Drug Discovery Foundation, describing the determination in an emailed statement as "consistent" with the FDA's accelerated approval.
Another worry is Aduhelm's safety, specifically a type of brain swelling related to amyloid-targeting drugs that occured in about 40% of participants given a high dose of the drug in Biogen's Phase 3 trials. Most cases were either mild or produced no symptoms, however.
In its determination, CMS said current evidence was insufficient to determine the drug's benefits outweigh known side effects.
Biogen shares fell about 7% in post-market trading Tuesday, while Lilly shares dropped 2%.
Note: This story has been updated with additional detail throughout and comment from Biogen and Alzheimer's disease groups.