The complete announcement is below.
The short summary: This refers specifically refers to drugs which have received full FDA approval, not just accelerated approval. Currently that includes only Aduhelm (aducanumab) although as the press release notes, full approval for Lekembi (lecanemab) may come as early as July--for very specific groups with MCI/Mild AD, confirmation of PET amyloid, ability to tolerate MRIs and PET scans and comply with biweekly infusions for 96 weeks, with monthly infusions for an indeterminate time after that. Access to a medical setting with the staffing and infrastructure to follow patients closely and report results to a national registry will be needed, and those with a variety of medical, psychiatric or risk factors are recommended for exclusion in a recently published "appropriate use recommendation" document. Donanemab is likely to seek full approval this fall.
FWIW, I've seen articles predicting that this would bankrupt government or insurance companies here or in Europe. I think that's not realistic for this class of drugs.Centers for Medicare & Medicaid Services (CMS) Administrator Chiquita Brooks-LaSure announced today how people can get drugs that may slow the progression of Alzheimer’s disease covered by Medicare. If the Food and Drug Administration (FDA) grants traditional approval, then Medicare will cover it in appropriate settings that also support the collection of real-world information to study the usefulness of these drugs for people with Medicare.
“Alzheimer’s disease takes a toll on not just the people suffering from the disease but also on their loved ones and caregivers in a way that almost no other illness does. CMS has always been committed to helping people obtain timely access to innovative treatments that meaningfully improve care and outcomes for this disease,” Brooks-LaSure said. “If the FDA grants traditional approval CMS is prepared to ensure anyone with Medicare Part B who meets the criteria is covered. 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.”
he 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. 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 have received accelerated approval from the FDA, but no product has received traditional approval.
Medicare will cover drugs with traditional FDA approval when a physician and clinical team participates in the collection of evidence about how these drugs work in the real world, also known as a registry. Clinicians will be able to submit this evidence through a nationwide, CMS-facilitated portal that will be available when any product gains traditional approval and will collect information via an easy-to-use format. This method, and any others that may follow, will adhere to robust privacy protections in accordance with applicable federal laws and regulations, including HIPAA. Researchers will have access to the information to conduct studies, furthering knowledge of how these drugs can potentially help people. CMS is working with multiple organizations that are getting ready to open their own registries. More information will be released as they come on line.
The approach is consistent with CMS’ National Coverage Determination. To get Medicare coverage people will need to 1) be enrolled in Medicare Part B, 2) be diagnosed with mild cognitive impairment or early dementia caused by Alzheimer’s disease, and 3) have a qualified physician participating in a registry, including the options above, with an appropriate clinical team and follow up care. These criteria are in addition to any label requirements the FDA specifies.
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.
Other registries for drugs that may slow the progression of Alzheimer’s disease may become available in the coming weeks and months. Information will be available at https://www.cms.gov/Medicare/Coverage/C ... evelopment as studies come on line.
In the AHEAD clinical trial (people with amyloid and no MCI or dementia), 75-90% of people seeking to enroll at sites did not end up starting on the trial because they did not have "enough" amyloid, were unable or unwilling to have multiple MRIs and PET scans, or unable to manage biweekly visits, including travel time, with the 90-minute infusion + monitoring, plus time for the on-site pharmacist to fill the order (doses are 10 mg/kg of body weight, weighed each time) and an RN to review the recent medical history, note vital signs (BP and pulse) and set up the infusion IV.
Researchers, including those who designed these trials, believe this is a first-in-class decision for a few drugs that will accelerate development of combined amyloid-tau drugs (already starting in small trials), anti-inflammatory drugs, oral anti-amyloid drugs like ALZ-801, etc.
It's notable that CMS recognizes the value of having a national registry of ALL sites administering these drugs to gain much more understanding of real-world barriers to treatment, benefits, side effects and serious adverse events, including deaths.