Medicare to broaden coverage of Alzheimer's drugs after full FDA approval, but with some restrictions

The Centers for Medicare & Medicaid Services (CMS) outlined plans Thursday to broadly cover a new class of Alzheimer’s drugs once the medications get full approval from the Food and Drug Administration (FDA). The new proposal does require patients to participate in registries that collect data.

The first drug potentially covered by the expanded plan, Eisai’s Leqembi, could be granted full approval from the FDA as soon as this summer.

CMS issued a statement Thursday reiterating its national coverage decision which said it would cover the costly drugs for patients enrolled in Medicare Part B who meet the criteria for coverage. That criteria includes patients' physicians and clinical teams participating in registries to collect evidence about how the drugs work in the real world.

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will discuss the results of a confirmatory trial of the Eisai product Leqembi on June 9, with a potential decision on traditional approval possible within weeks. Broader Medicare coverage would begin on the same day the FDA grants traditional approval, CMS said.

Under CMS’ current coverage policy, if FDA grants traditional approval to other drugs in this class they would also be eligible for broader coverage. Currently, two drugs in this class, Leqembi and Biogen’s Aduhelm, have received accelerated approval from the FDA, but no product has received traditional approval.

A year ago, CMS issued a narrow and controversial Medicare coverage proposal for Biogen’s Alzheimer’s disease drug Aduhelm. The agency said drugs approved under the FDA's accelerated pathway will only be covered when administered in eligible clinical trials.

The agency has faced intense pressure from lawmakers, patient advocacy groups like the Alzheimer's Association and pharmaceutical companies to change its restrictive national coverage decision that limits coverage to patients in clinical trials in the absence of full FDA approval. 

Back in April, CMS Administrator Chiquita Brooks-LaSure testified before Congress and said Medicare will cover the new Alzheimer’s treatment Leqembi for all patients eligible under the medication’s label if the FDA fully approves the drug in July, CNBC reported

The Alzheimer's Association on Thursday called the registries "an unnecessary barrier" and creates extra red tape for patients to get access to the drug.

"Registries are important tools to gather much-needed real-world evidence to transform and improve patient care. But, registries should not be a requirement for coverage of an FDA-approved treatment," the organization said in a statement. "We remain concerned that the requirement of clinicians to register and enter data will create unnecessary hurdles."

Rob Egge, the Alzheimer’s Association chief policy officer, said the registry system will limit patient access and make CMS’ promise of broad coverage an “empty proclamation," CNBC reported back in April.

The Veterans Health Administration has agreed to cover Leqembi for veterans who are over age 65 and meet other eligibility criteria.

In its statement, CMS officials defended the coverage requirements, writing that "registries are common tools in clinical settings that have successfully gathered information on patient outcomes for decades."

"There is strong precedent for using registries to gather more information on a newly-approved treatment. For example, when the FDA granted traditional approval to the transcatheter aortic valve replacement (TAVR), CMS required the clinicians and hospitals to participate in a registry to track outcomes. Information gathered from that registry has since been used to further inform Medicare coverage determinations, and to help people and their clinicians make better decisions about whether TAVR is the best treatment for them," CMS officials said.

Clinicians will be able to submit data on the drugs through a nationwide, CMS-facilitated portal that will be available when any product gains traditional approval, the agency said in its statement. But the registries still need to be set up for patients.

"Additional information about the data submission, registry requirements and details about how patients and physicians can enroll is still needed," the Alzheimer's Association said in its statement.

CMS said it is working with "multiple organizations that are getting ready to open their own registries." And th agency said "more information will be released as they come online" but provided little details.

“If the FDA grants traditional approval CMS is prepared to ensure anyone with Medicare Part B who meets the criteria is covered," Brooks-LaSure said in a statement. "I’m pleased to make this announcement today as part of CMS’ mission to help improve the lives of Americans we serve. I hope we see more private sector partners in this work making their own announcements soon.”

Broader CMS reimbursement will be a tailwind for Eisai and Biogen but also Eli Lilly and earlier-stage players like Prothena among others in the anti-amyloid space given over 5 million Medicare beneficiaries suffer from Alzheimer's disease, noted William Blair analysts in a note issued Thursday.

"We believe that registries at specialist centers where Leqembi is likely to be initially administered will not be a substantial hurdle. Patients on anti-amyloid therapy will be high touch with required MRI monitoring as per label and clinicians wanting to track cognitive outcomes in the real world given the class is not without safety risks," the William Blair analysts wrote.

According to analysts with GlobalData Healthcare, Eisai and Biogen, who collaborated on the development of Leqembi, are poised to realize $12.9 billion in revenue from the drug through 2028. The data analytics company estimates sales in the United States will grow at an annual rate of 106%, totaling $7.7 billion during the period.