On Thursday, after a months-long review and lobbying campaign by patient advocacy groups, the Centers for Medicare and Medicaid Services (CMS) said it would only pay Biogen Inc’s Aduhelm, and other drugs that work similarly to patients enrolled in validated clinical studies, unless the treatments clearly demonstrate benefit to the patient.
Medicare covers nearly 64 million Americans aged 65 and older, so the coverage decision could affect 85% of people who might otherwise use drugs for the age-related condition.
Eisai’s lecanemab and Lilly’s donanemab, like Aduhelm, are monoclonal antibodies designed to eliminate beta-amyloid, a type of protein fragment that accumulates in the brains of patients with Alzheimer’s disease . Both drugmakers said they expect results from upcoming phase III trials to eventually validate data from earlier stages under review by the US Food and Drug Administration.
A fourth plaque-targeting antibody, gantenerumab, is in late-stage development at Roche Holding AG, which is not seeking accelerated review by the FDA.
In June, the FDA cleared Biogen’s Aduhelm – the first drug in its class and the first treatment for Alzheimer’s disease approved in the United States in 20 years – under the agency’s fast-track, based on the drug’s ability to clear plaque, rather than evidence that it slows cognitive decline in patients with Alzheimer’s disease.
Medicare, however, decided to allow standard reimbursement only for Alzheimer’s drugs approved under the traditional FDA process, based on “direct measurement of clinical benefit.”
Eisai, which is partnered with Biogen, said it aims to complete an FDA rolling application for lecanemab, under the fast track, by the middle of the year. The Japanese drugmaker said it is also expecting results from its Phase III trial in 1,800 patients this fall.
If those results are positive, Eisai said he thinks this large study could meet the “high level of evidence” criteria set by Medicare in its coverage decision.
The study is designed to show that lecanemab can slow the rate of cognitive and functional decline by at least 25%.
“It’s a disease-modifying drug,” Ivan Cheung, president of Eisai in the United States, said in a recent interview with Reuters. “You would expect to see a separation between treated and untreated groups that improves over time.”
Roche also plans to report the results of the phase III trial for gantenerumab later this year.
Lilly, in a statement, said it intends to complete its current and continuing application for expedited FDA approval of donanemab this year. It does not expect to have the results of a phase III trial of the drug until mid-2023.
The Indianapolis-based company said it believes Medicare coverage restrictions are “unnecessary, restrictive and inappropriate” for FDA-approved drugs.
The idea that the removal of amyloid plaques is reasonably likely to slow cognitive and functional decline in people with early Alzheimer’s disease is known as the “amyloid hypothesis”, a theory that has led a long history of drugs that have tried and failed to eliminate ailments or help patients.
Greg Rippon, medical officer for neuroscience and Alzheimer’s disease at Roche’s Genentech unit, explained in a recent interview that this theory is supported by analysis of inherited forms of Alzheimer’s disease. , all of which are caused by mutations in the processing of amyloid.
He added that more recent studies have shown that amyloid buildup is a precursor to other brain dysfunctions that accelerate neurodegeneration in patients with Alzheimer’s disease.
“Obviously it comes down to the clinical data and the demonstration of that clinical benefit and that’s where a lot of the skepticism is concentrated,” Rippon said. (Reporting by Deena Beasley; editing by Bill Berkrot)