The Centers for Medicare and Medicaid Services said Tuesday they would only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease in approved randomized controlled trials. by CMS.
The draft national coverage decision, which insurers nationwide are likely to follow, makes it clear that CMS research randomized controlled trials which “demonstrate a clinically significant advantage on cognition and function”. This will be a difficult task for Biogen, which has previously shown conflicting benefits from previous Aduhelm trials that were initially halted due to futility, then resurrected for expedited approval.
“None of the trials completed at the time of publication of this NCA has convincingly demonstrated that the use of anti-amyloid mAbs results in significant improvement in the health outcomes of patients with AD,” CMS said in its draft decision, which is now open for 30 more days. comments and will be finalized in April.
Holly Fernandez Lynch, professor of bioethics at the University of Pennsylvania, said Terminal news that she thinks CMS made the right decision in going for cover with the development of evidence (EDC) and demanding the trial, although she has noted how odd it is that the government is essentially paying Biogen to conduct a trial.
“I didn’t think an outright denial of all coverage was really on the table. That’s a pretty strong rebuke from the FDA the way it is, ”she said.
The announcement is a blow to Biogen, which will likely need to design and run another clinical trial in addition to its confirmatory trial to meet FDA fast-track approval requirements. Biogen’s stock fell about 8% on Tuesday afternoon.
“This coverage determination project negates the daily burden on people living with Alzheimer’s disease,” Biogen said in an emailed statement to Endpoints, adding:
Coverage with developing evidence (EDC) in a randomized clinical trial will exclude almost all patients who could benefit from it. This will severely limit patients’ access to FDA-approved treatment, especially for underserved patients, as evidenced by other CED determinations. DACs can take months or even years to start, and hundreds of patients with Alzheimer’s disease – the majority of whom are Medicare beneficiaries – progress from mild to moderate stages every day, where treatment may fail. more be an option.
But Lon Schneider, who heads the University of California’s Southern California State Alzheimer’s Center, told Endpoints that 80% of patients in Biogen trials had prodromal AD, which is ” definitely, virtually no daily burden (some memory impairment, yes, some amyloid? yes). And the trials have not been able to show that a single patient has benefited from aducanumab, so who does not? “
He called the decision a “smackdown” because CMS “also told Lilly that the FDA could give you AA [accelerated approval] or BTD [breakthrough designation] but we will wait to see the positive results of the tests.
Industry group PhRMA proposed a similar characterization, noting: “With this proposal, CMS is canceling an entire class of drugs even before several products have been reviewed by the FDA, positioning itself and not the FDA as the key arbiter of drugs. clinical evidence ”.
The FDA declined to comment on CMS’s decision.
CMS also sets out very specific testing requirements that Biogen, Roche, Eli Lilly and any other amyloid candidate will have to meet, which is not what CMS has done in previous national coverage decisions, noting that he is aware of at least three other anti-amyloids mAbs are currently approaching phase 3 trials.
“The diversity of patients included in each trial should be representative of the national population diagnosed with AD,” says CMS, noting that the trials should determine, “at a minimum,” whether the use of mAbs directed against amyloid results in a statistically significant result. and a clinically significant difference in the decline of cognition and function, and whether they address the adverse events associated with the use of these anti-amyloid mAbs.
The news comes as Biogen recently halved the price of Aduhelm, having sold just $ 300,000 of Aduhelm in the first few months, and has come under heavy criticism for the $ 56,000 price tag which forced CMS to prepare for record premium increases for the elderly. Earlier this week, HHS Secretary Xavier Becerra called on CMS to reconsider these steep premium increases now that Aduhelm’s price has been slashed.
Senator Bernie Sanders of Vermont said in a statement that CMS’s decision “is an important step forward, especially following Secretary Becerra’s decision yesterday to order Medicare to reconsider a premium rate hike caused in part by the outrageous price of Aduhelm “.
Others who believe Aduhelm works have been disappointed with CMS.
Harry Johns, CEO of the Alzheimer’s Association, said in a statement: “Today’s draft decision from the Centers for Medicare & Medicaid Services (CMS) is shocking discrimination against all people with dementia. Alzheimer’s, especially those already disproportionately affected by this deadly disease, including women. , Blacks and Hispanics.
George Vradenburg, president and co-founder of UsAgainstAlzheimer’s, added: “This is absolutely unacceptable. If this ruling is upheld, for the first time in history, millions of Americans will be denied coverage not just for one drug, but for an entire class of drugs, not by the agency that regulates the drugs. , but by the federal insurance bureaucracy.
Editor’s Note: Article updated with additional comments.