Almost immediately following the regular approval by the Food and Drug Administration of the Alzheimer’s disease biologic Leqembi (lecanemab), the Centers for Medicare and Medicaid Services announced it will cover most patients eligible for the therapeutic. This includes patients with mild cognitive impairment or mild dementia with confirmed amyloid plaques.
Amyloid plaques are accumulations of beta amyloid proteins which clump together between the neurons in the brains of Alzheimer’s disease patients. Physicians confirm the existence of beta amyloid plaque in patients by performing brain scans.
CMS is requiring that Medicare beneficiaries who take Leqembi enroll in a patient registry to collect more data on the drug. The patient registry requirement is also a prerequisite for reimbursement of Leqembi.
In April 2022, CMS issued a National Coverage Determination which severely restricted access to all beta amyloid directed monoclonal antibodies unless they had regular, as opposed to accelerated, approval by the FDA and were deemed to offer at least some clinically meaningful benefit.
In November 2022, researchers presented Phase 3 data for lecanemab, which showed modest clinical benefit. Accelerated (January 2023) and then regular (July 2023) FDA approval followed. Correspondingly, CMS relaxed the conditions of reimbursement for Leqembi.
While the revised NCD does not open the floodgates to coverage for millions of Medicare recipients, it does offer access to an estimated 1.2 million U.S. beneficiaries. These are approximate numbers of patients with mild cognitive impairment and confirmed amyloid plaques who are eligible for Leqembi based on its labeled indication.
The CMS press release issued in the wake of Leqembi’s FDA approval raises a number of questions. While it’s clear that coverage of Leqembi is broadened considerably, it’s unclear how the patient registries will work, how comprehensive the coverage of brain scans and genetic testing will be and whether regional carriers (Medicare Administrative Contractors and Medicare Advantage Plans) have discretion with respect to which other conditions of reimbursement they can impose.
Patient Registries
For Leqembi, CMS has established a patient registry on its website and will also provide access to registries established by other organizations on the same site.
As part of Medicare’s longstanding coverage with evidence development program, patient registries are supposed to inform answers to questions about how a medical technology is working, which includes whether it improves health outcomes based on real-world evidence. Practically all registries which Medicare has established in the past were for diagnostics and medical devices. Nonetheless, the tool been available for Medicare to use for pharmaceuticals for quite some time.
It’s not yet clear, however, how much data collection and analysis will cost and who will bear those costs. More importantly, without a control group and therefore nothing to compare it to, the registry data being collected about Leqembi’s benefits and risks can’t properly address the question of how well the drug does in terms of slowing cognitive deterioration.
Genetic Testing And Brain Scans
The Leqembi label contains a boxed warning about the risks of brain bleeding and swelling known as amyloid-related imaging abnormalities. The FDA requires testing for the ApoE genetic biomarker before initiating Leqembi to inform patients and physicians of the risk of ARIA. Individuals with two copies of the ApoE mutation, which is roughly 15% of Alzheimer’s patients, are at especially high risk of brain swelling and bleeding.
Apparently, Medicare will cover genetic testing to screen for and monitor side effects. Detection of beta amyloid plaque is also a labeled prerequisite for prescribing. Currently, Medicare covers one amyloid PET scan per lifetime, called Amyvid (florbetapir F 18). Medicare has proposed changing its policy on coverage of Amyvid, namely, leaving it up to Medicare Administrative Contractors at the local level to decide. As such, it’s unclear how much coverage this will entail, for example, how many scans in a given time period? And it’s unknown whether certain Medicare contractors will favor the cheaper cerebrospinal fluid tests over, say, Amyvid.
Regional Or Local Medicare Coverage
Practically all Medicare Part B (physician-administered) drugs, such as injectables given in a doctor’s office, are covered at the local or regional level by Medicare Administrative Contractors and Medicare Advantage Plans. The former make what are called local coverage determinations, which the latter often abide by. In addition, Medicare Advantage Plans have additional formulary management tools at their disposal, such as the use of step edits.
But in the case of beta amyloid directed monoclonal antibodies, CMS pursued an NCD, which is technically binding on all Medicare carriers at the local level.
Under the NCD, the Medicare Part B plans must follow the requirements of the NCD and generally have little if any discretion on general coverage criteria.
What’s left undetermined is whether local Medicare carriers can impose any additional conditions of reimbursement at a more granular level. For example, could they expand exclusions, such as the ones referred to in the Department of Veterans Affairs formulary guidance? It excludes anyone with a medical, neurological or mental health condition that may be a contributing or primary cause of cognitive impairment. This could be a person who has had a stroke, transient ischemic attack or seizure within the last year, as well as any person with brain scan findings that show evidence of micro hemorrhages, aneurysms, lesions or tumors. Also excluded in the VA guidance are patients with certain untreated bleeding disorders, thyroid stimulating hormone above the normal range and any uncontrolled immunological disease. The formulary protocol goes on to warn that the combined use of lecanemab with anti-platelet or anti-coagulant drugs may increase the risk of cerebral macro hemorrhage.
Patient Out-Of-Pocket Costs
Then there’s Leqembi’s annual price tag of $26,500. In Medicare Part B (physician-administered drugs) patient co-insurance is 20% of this list price, though many Medicare beneficiaries will have paid for supplemental insurance, such as MediGap, to cover at least a portion of the patient cost-sharing. But there will be additional costs for patients, including co-payments for the biweekly physician visits. This is because Leqembi needs to be administered every two weeks.
Adoption will also depend on how healthcare systems cope with the registries, as well as the need for screening and administration of the drug in infusion clinics.
Perhaps because at this point in time there are no other disease-modifying agents targeting Alzheimer’s disease, one of Leqembi’s two sponsors, Eisai, has estimated that roughly 100,000 patients will be on the drug by year three post approval. Eisai’s goal may be achievable, but it will face challenges, such as overcoming the hurdle of patient cost-sharing.
Aside from the out-of-pocket cost barrier, it may also be difficult to convince doctors to prescribe and patients to take the medichine. There isn’t yet a consensus among clinical neurologists that the treatment is clinically meaningful for patients. Leqembi modestly slows the rate of continuing decline in cognitive function compared to placebo. Notably, however, it does not improve cognitive function; it slightly reduces the rate of deterioration as measured by validated cognitive testing instruments to a degree that may not be evident to patients and their families or caregivers. Moreover, the drug’s side effect profile may be considered problematic by many potential patients.