The CMS proposed a plan that could strip Alzheimer's drug Aduhelm of public Medicare coverage. The result was widespread confusion. We clarify some aspects of their proposal toward answering the big question: Will Aduhelm be covered by Medicare?
“It’s been very confusing,” said Pam Montana, an advocate for Alzheimer’s who has participated in several clinical trials herself, adding that the recent memo by the Centers for Medicare and Medicaid Services (CMS) limiting coverage of Alzheimer’s drug Aduhelm “isn’t written very well.” Even after carefully re-reading it, Montana had trouble understanding what it meant for her and others living with Alzheimer’s. And she was far from alone.
Earlier this month, the CMS released a proposal to dramatically restrict insurance coverage for the anti-amyloid drug developed by Eisai and Biogen. Aduhelm was publicly available following the Food and Drug Administration’s (FDA) approval in summer 2021, but due to controversy around its efficacy, patients have hit snag after snag in terms of both access and insurance coverage.
The new CMS regulations suggest that Medicare won’t cover the drug for the general public. In addition, the agency’s plan appeared to stipulate that Aduhelm and future anti-amyloid monoclonal antibody treatments might need to undergo additional clinical trials, even after FDA approval, before Medicare coverage would be green-lit.
Would a new round of CMS- or NIH-approved drug trials be required? How does the plan affect Aduhelm, considering that the drugmakers of which already have planning underway for a mandated Phase 4 trial? How does it affect future anti-amyloid drugs, currently in Phase 3 trials and awaiting their own FDA approval processes?
And, Global Alzheimer’s Platform Foundation President John Dwyer added, how does the CMS throwing its weight around on Aduhelm affect the authority of future FDA decisions? “Why did this administration’s CMS decide to replicate a process that has been undertaken by the Food and Drug Administration for tens and tens of years and with reasonable results?” he asked, in a conversation with Being Patient. “It’s very discouraging, very wasteful. We estimate that the randomized controlled trial structure CMS is proposing would delay access to a drug somewhere between six and nine years.”
FAQ: What drugs does the new rule apply to?
Short answer: All the anti-amyloids from here on out.
“If finalized, the proposed Medicare National Coverage Determination (NCD) would cover any FDA-approved monoclonal antibodies (mAbs) directed against amyloid for the treatment of certain conditions,” a CMS spokesperson wrote to Being Patient. “This applies to Alzheimer’s Disease for Medicare beneficiaries with Mild Cognitive Impairment (MCI) due to Alzheimer’s Disease or mild Alzheimer’s Disease, dementia, and evidence of amyloid pathology consistent with Alzheimer’s disease under Coverage with Evidence Development (CED), when the conditions specified in the proposed decision are satisfied.
FAQ: How will this affect Aduhelm’s Phase 4 trials? Do those meet the CMS/NIH standards referred to in the proposal?
Short answer: It’s too soon to tell, because the CMS doesn’t have any authority to weigh in on Aduhelm’s Phase 4 trials — at least, not yet.
“It is possible that a trial that is already planned could satisfy the requirements specified in the proposed NCD,” the CMS spokesperson told Being Patient. “However, because this is a proposed decision, protocols are not being evaluated at this time.”
After reviewing all comments received on the proposed determination, CMS will announce its final decision by April 11, 2022. (Comments will be accepted until February 11.)
FAQ: How would the plan, if approved, affect the Alzheimer’s drug development process moving forward?
Short answer: It could mean drugmakers need to conduct additional rounds of trials before Medicare coverage is granted, or run more thorough, more diverse trials to start with.
Aduhelm’s trial was notoriously inconclusive when it came to demonstrating the drug’s efficacy. Controversially, it was approved anyway. But for future drugs, existing trials that show clear “statistically significant and clinically meaningful” outcomes — in other words, they show cognitive improvements in participants — could “trigger expedited reconsideration of insurance coverage for the drug,” according to the CMS.
“If you have an anti-amyloid that works in the same way,” the spokesperson said, “and their Phase 3 trial shows there is cognitive improvement, it would bypass the NCD, and it would be covered. “
FAQ: Who’s evaluating future Alzheimer’s anti-amyloid drug trials, then? The FDA? The CMS? The NIH?
Short answer: All of the above.
“Trials are to be designed by external investigators, who would submit trial protocols,” the spokesperson said. Then, a multidisciplinary team from the CMS, the National Institutes of Health, and the Agency for Healthcare Research and Quality would assess the protocols.
FAQ: OK, great, but ultimately, is Aduhelm going to be covered by Medicare, or not?
Short answer: At least for now, it will not. As for the future, it’s too soon to know.
Medicare coverage won’t be determined until the CMS gives a final ruling on the NCD, and that’s expected on April 11, 2022. At that point, one of three things will happen: The CMS will decline to extend Medicare coverage to Aduhelm; the CMS will approve Medicare coverage for Aduhelm; or, there could be more waiting: If the CMS enacts its proposed plan, it would reserve the right to conduct its own evaluation of Aduhelm’s Phase 4 drug trial protocols before offering Medicare coverage for Aduhelm.
In a statement released January 11, 2022, CMS Administrator Chiquita Brooks-LaSure said that the agency is “committed to providing the American public with a clear, trusted, evidence-based decision that is made only after a thorough analysis of public feedback on the benefits and risks of coverage for Medicare patients.”
“Medicare will issue guidance surrounding coding details and payment rates, including required beneficiary cost-sharing, upon the publication of the final NCD,” the CMS spokesperson said.
FAQ: So, what happens next?
Short answer: It will go through its Phase 4 trials as planned. Patients who are not participating in trials will pay full price for the drug.
Until the NCD is issued, the CMS has no authority over the evaluation of these protocols: Their proposal is still just a proposal — not official policy. So, when it comes to who will be evaluating Phase 4 trials and for what purpose, at this point, only the FDA will be weighing in.
The CMS hasn’t said whether it hopes to eventually review Biogen’s Phase 4 trial protocols, and Biogen and Eisai don’t know, either. “We cannot speculate CMS requirements until the final National Coverage Determination is announced,” a Biogen spokesperson wrote to Being Patient.
However, following the CMS’s memo, the drugmakers did move to expand the size of the FDA-required Phase 4 confirmatory trial in order to “strengthen the data” on Aduhelm’s efficacy. They aim to start the trial in May, pending approval of the trial protocol by the FDA. The drugmakers announced that they will improve diversity in trial enrollment.
For this trial, Medicare is a non-issue: The drugmakers are paying for the drug that will be administered to trial participants. And, as this is typically the case, it’s unclear why trial participants have been a part of the conversation about Medicare coverage at all.
There’s more to Aduhelm coverage than just the price of the drug.
The CMS’s memo also doesn’t specify the amount of insurance coverage for expensive imaging procedures that are required for screening participants to determine whether they qualify for the ongoing Aduhelm clinical trials.
There are lingering concerns about racial and economic equity in limiting Aduhelm access to just trial participants.
Montana worried that limiting coverage may make it more challenging for many individuals in rural areas to participate. “Most of us living with Alzheimer’s disease do not have facilities nearby,” she said. “Please change your decision and allow those of us living with Alzheimer’s hope for our future.”
Right now, all the general public can do is share their opinions with the CMS as it evaluates the best course of action.
According to the CMS, the agency is committed to transparency and wants everyone to understand how decisions regarding Medicare coverage are made. To support that goal, they’re keeping a running list of all organizations they met with regarding FDA-approved monoclonal antibody drugs for Alzheimer’s on their website.
“This list has been updated and will continue to be updated throughout the analysis process,” the CMS spokesperson told Being Patient.
These details will be shared in the final version of the memo, which is expected on April 11, 2022, according to the CMS spokesperson. In the meantime, the CMS is gathering public comments toward a review period before their final decision. The deadline for comments is February 11; learn how to your own comments on the determination here.