UPDATED: CMS to restrict coverage of Biogen's controversial Alzheimer's drug to only clinical trials
The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.
The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.
“None of the trials completed as of the publication of this NCA has convincingly demonstrated that use of antiamyloid mAbs result in a meaningful improvement in health outcomes for AD patients,” CMS said in its draft decision, which is now open for 30 days of more comments, and will be finalized in April.
Holly Fernandez Lynch, a bioethics professor at the University of Pennsylvania, told Endpoints News that she thinks CMS made the right decision in going for coverage with evidence development (CED) and requiring the trial, although she noted how odd it is that the government will be essentially paying Biogen to run a trial.
“I didn’t think outright refusal of any coverage was genuinely on the table. This is a pretty strong rebuke of FDA as it is,” she said.
The announcement is a blow for Biogen, which will likely have to design and run another clinical trial in addition to its confirmatory trial to satisfy the FDA’s accelerated approval requirements. Biogen’s stock dropped about 8% Tuesday afternoon.
“This draft coverage determination denies the daily burden of people living with Alzheimer’s disease,” Biogen said in a statement emailed to Endpoints, adding:
Coverage with evidence development (CED) under a randomized clinical trial will exclude almost all patients who may benefit. This will significantly limit patient access to an FDA-approved treatment, especially for underserved patients as evidenced in other CED determinations. CEDs can take months to years to initiate, and hundreds of Alzheimer’s patients – the majority of whom are Medicare beneficiaries – are progressing each day from mild to moderate disease stages, where treatment may no longer be an option.
But Lon Schneider, who directs the University of Southern California’s State of California Alzheimer’s Disease Center, told Endpoints that 80% of patients in the Biogen trials had prodromal AD, which is “definitionally, virtually no daily burden (a bit of memory impairment, yes, a bit of amyloid? Yes). And the trials couldn’t demonstrate that any one patient benefitted from aducanumab, so who’s being deprived benefit?”
He called the move “a smackdown” as CMS “also told Lilly that FDA might give you AA [accelerated approval] or BTD [breakthrough designation] but we’ll wait to see positive trials results.”
Industry group PhRMA offered a similar characterization, noting, “With this proposal, CMS is writing off an entire class of medicines before multiple products have even been reviewed by FDA, positioning itself and not FDA as the key arbiter of clinical evidence.”
The FDA declined to comment on the decision by CMS.
CMS also lays out very specific trial requirements that Biogen, Roche, Eli Lilly and any other amyloid contenders will have to meet, which is not what CMS has done in previous national coverage decisions, noting that it’s aware of at least three other anti-amyloid mAbs currently approaching Phase 3 trials.
“The diversity of patients included in each trial must be representative of the national population diagnosed with AD,” CMS says, noting that the trials must address, “at a minimum,” whether the use of mAbs directed against amyloid result in a statistically significant and clinically meaningful difference in decline in 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 cut Aduhelm’s price in half, after selling just $300,000 of Aduhelm in the early months, and coming under heavy criticism for the $56,000 price tag that forced CMS to prep for record-high premium increases for seniors. HHS Secretary Xavier Becerra earlier this week called on CMS to reconsider those steep premium increases now that Aduhelm’s price has been reduced.
Sen. Bernie Sanders of Vermont said in a statement that CMS’ decision “is an important step forward, especially following Secretary Becerra’s move yesterday to direct Medicare to reconsider a premium rate hike caused in part by the outrageous price of Aduhelm.”
Others who believe that Aduhelm does work were disappointed by the decision from CMS, which will likely mean only a limited number of patients are covered under a CMS-approved clinical trial.
“This is clearly disappointing news, as CED would significantly limit the number of patients who can access Aduhelm and other anti-Abeta mAbs, and in this scenario, we would expect a prolonged process to initiate and enroll patients into the CED clinical trials which need to be reviewed and approved by CMS,” SVB Leerink told investors.
Advocacy organizations were similarly not pleased.
Harry Johns, Alzheimer’s Association CEO, said in a statement, “Today’s draft decision by the Centers for Medicare & Medicaid Services (CMS) is shocking discrimination against everyone with Alzheimer’s disease, especially those who are already disproportionately impacted by this fatal disease, including women, Blacks and Hispanics.”
George Vradenburg, chair and co-founder of UsAgainstAlzheimer’s, added, “This is absolutely unacceptable. If this decision stands, for the first time in history, millions of Americans will be denied coverage not just to a drug, but to a whole class of drugs—not by the agency that regulates drugs but by the federal insurance bureaucracy.”
Editor’s note: Article updated with additional comments.