Those trials could be approved by the FDA itself or by the National Institutes of Health, and would not have to be sanctioned by CMS, as originally proposed.
In a small victory for Alzheimer’s patients, CMS said if the FDA approves future drugs through a more traditional process, it would allow wider access to coverage for those medications. Those drugs also would target the amyloid plaques suspected of causing the devastating effects of Alzheimer’s.
The decision means Aduhelm will remain unavailable for now to some of the nation’s 6.2 million people with Alzheimer’s who had hoped to try it for mild cognitive impairment or dementia that results from the disease. The population with milder Alzheimer’s disease, the target group for the drug, is estimated at 1 million to 2 million people.
Aduhelm is the first Alzheimer’s treatment intended to slow cognitive decline.
Lee A. Fleisher, chief medical officer and director of CMS’s Center for Clinical Standards and Quality, said in a briefing for news media Thursday that “there is not currently enough evidence of clinical benefit to say [Aduhelm] is reasonable and necessary for people with Medicare.”
The decision reflects a wide spectrum of opinion on whether Aduhelm is effective enough to warrant greater distribution. A monoclonal antibody taken as a monthly infusion, the drug is the first new treatment for Alzheimer’s approved by the FDA since 2003. Its cost — $28,200 per patient, per year — also has raised concerns.
Alzheimer’s was the seventh-most-common cause of death in the United States in 2020, resulting in more than 134,000 fatalities.
The FDA’s approval and Medicare’s proposal in January to limit coverage have created twin furors over the medication. Alzheimer’s advocacy groups vowed to press for wider access to a drug that had offered the hope of slowing cognitive decline.
But some major health systems have refused to offer the medication, developed by the drugmaker Biogen, because some experts doubt its efficacy. The watchdog group Public Citizen — which frequently criticizes the FDA — called for three top FDA officials to resign.
Alzheimer’s advocacy groups fiercely fought CMS’s proposed coverage restriction when it was announced in January, angered that it would drastically limit the number of people who could receive the treatment.
Volunteer advocates with the Alzheimer’s Association have held an estimated 400 meetings on Capitol Hill. Groups such as the Global Alzheimer’s Platform Foundation said they have met with CMS and other administration officials to make their case. And UsAgainstAlzheimer’s launched a seven-figure ad campaign targeting the D.C. and Baltimore media markets.
“Every treatment for every other disease has been covered when there has been an FDA approval, and to set a precedent on the backs of people with Alzheimer’s is just flatly wrong,” Harry Johns, chief executive of the Alzheimer’s Association, said hours before the announcement.
In addition, questions about the drug’s unusual approval process have prompted investigations by the Department of Health and Human Services’ inspector general, among others.
Much of the uproar centered on the FDA’s use of accelerated approval to clear the drug after concluding there was not enough evidence of clinical effectiveness for a full approval. In two large clinical trials for early-stage patients, Biogen said there was a mild slowing of cognitive decline in the high-dose group of one study and no clinical benefit in the other.
But the FDA concluded that the drug’s ability to clear the brain of plaque meant there was a “reasonable likelihood” it would slow the disease. The agency approved the drug based on that “surrogate marker” and directed Biogen to conduct another trial to verify the clinical benefit, with results due in nine years.
CMS reviewed 250 documents and nearly 10,000 comments on its proposed coverage decision, vastly more than the 131 comments it received during a public input period ahead of the proposal last summer.
Biogen issued a statement Thursday evening expressing dismay at the CMS decision.
“This unprecedented CMS decision effectively denies all Medicare beneficiaries access to Aduhelm, the first and only FDA-approved therapy in a new class of Alzheimer’s drugs,” Biogen said. “These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas.”
Johns, of the Alzheimer’s Association, said his organization is “very disappointed with the immediate impact it will have on Americans living with Alzheimer’s and their families” despite incorporation of some of their recommendations.
“Denying access to FDA-approved Alzheimer’s treatments is wrong,” Johns said.
But Sean Tunis, a former CMS official and now a senior fellow in the Tufts Medical Center’s Center for the Evaluation of Value and Risk in Health, called the decision “very reasonable.” He praised the agency for backing off its initial plan to review the randomized trials and switching that authority to FDA and NIH.
The decision is still “quite restrictive” in allowing people to access the Biogen drug, Tunis said. But he added, “I think that’s quite justifiable given the controversy around the evidence that is currently available.”