The Food and Drug Administration’s expected approval of Biogen and Eisai’s Alzheimer’s drug Leqembi, or lecanemab, is set to broaden access and accelerate a new class of treatments for the degenerative disease.
A number of patient advocacy groups are eagerly awaiting the decision, which would trigger broader access under Medicare. But the drug still faces coverage restrictions, along with critics who don’t believe it should be on the market at all.
The FDA is expected to decide by Thursday whether to fully approve the monoclonal antibody, which previously received accelerated clearance based on its ability to reduce amyloid beta plaque buildup in the brain. A small group of external advisers unanimously recommended the approval in May in a dramatic reversal from the reception the companies received for their last Alzheimer’s drug Aduhelm, or aducanumab.
“We finally are at the doorstep of being able to deliver what we hoped for for so long,” said Robert Egge, chief public policy officer for the Alzheimer’s Association. “Even as we recognize there are a lot of limitations to this.”
Leqembi is only indicated for early-stage Alzheimer’s. Clinical data show the drug slowed the progression of disease by 27 percent in an 18-month long trial when measured on a standard cognitive scale. But the information used to pinpoint a patient’s location on the scale is subject to interpretation from the doctor and the patient’s caregiver, and the rate of progression along the scale differs as the disease worsens.
Public Citizen, a consumer advocacy group that doesn’t accept government or corporate money, argued against the drug’s approval at the May advisers’ meeting, noting that the drug’s actual impact on the cognitive scale only amounted to a 0.46 percent difference.
That’s not enough to justify Leqembi’s serious risks of bleeding and swelling in the brain, Health Research Group Director and Yale Internal Medicine Adjunct Professor Robert Steinbrook said.
“We just don’t think this drug is the answer,” he said.
Lawmakers are also weighing in, with members on both sides of the aisle asking the administration to roll back restrictions on coverage.
Senate Health, Education, Labor and Pensions Chairman Bernie Sanders, I-Vt., also wrote to Health and Human Services Secretary Xavier Becerra in June, urging him to lower Leqembi’s $26,500 list price by either breaking the companies’ patents or testing ways to tie the price to the drug’s effectiveness.
The Centers for Medicare and Medicaid Services limited coverage of the entire drug class to participants of clinical trials when authorized via accelerated approval, after Aduhelm’s accelerated approval sparked a public uproar in 2021.
But CMS recently unveiled plans to broaden access to fully approved drugs by only requiring data collection through patient registries. The agency is launching its own free central database, aiming to reach doctors in small practices and underserved areas.
Registries are historically pricey, with registration fees alone reaching as much as $25,000 per hospital. CMS said it is also working with external groups that are planning separate registries, though it’s unclear how much they will cost.
“CMS encourages any organization interested in developing a registry to ensure cost is not a barrier to participation,” a spokesperson said.
Sean Tunis, a principal with health care consultancy Rubix Health and the former chief medical officer for CMS, said the agency’s coverage restrictions made sense for accelerated approval drugs. But the registry is not likely to achieve its goals because of its simplified data questions and lack of collaboration from industry groups, he said.
Tunis helped establish the coverage determination process under former CMS Administrator Mark McClellan in 2005. Last month he outlined open questions about the process for Leqembi in the health policy journal Health Affairs.
“I just think if it’s actually going to accomplish the goals that it was set out for — which is to allow for Medicare to pay for promising therapies while meaningful evidence is generated — it’s just, I think it’s going to take a more careful, thoughtful approach and working with the other groups to figure out a way that accomplishes both those things,” he said.
But the price of the drug makes coverage decisions harder, he said, even though CMS doesn’t have authority to consider price as a factor.
“This whole area of policy would be a lot easier if Medicare actually could take price into account,” he said. “Part of the reason that they have to do everything through the coverage policy is that at the end of the day, they don’t have any discretion about price. If they’re going to cover something, they essentially have to pay list price.”
Another big question mark still exists — whether CMS will broaden coverage of PET scans needed to monitor changes and risks in the brain under Leqembi.
Medicare currently covers just one scan per dementia patient in their lifetime. The agency revisited the policy last June, but delayed a decision in December, citing the need to consider “newly published evidence.”
The CMS spokesperson said the agency expects to propose a decision “soon.”
The debates could intensify as research progresses. Scientists are pursuing the drug’s effects in patients who have amyloid plaque but no outward signs of dementia, and also in patients with protein tangles known as tau — another associated marker in the brain.
At least some of these studies are supported through the National Institute on Aging, which also helped establish the science undergirding both Leqembi and PET scans.
Inflammation is another area of interest, Egge said. The Alzheimer’s Association is the largest nonprofit funder of research in the space (and also accepts donations from pharmaceutical companies, including Biogen Inc. and Eisai Inc.)
Current consensus is that a multi-pronged assault is the best form of attack.
“Just like so many other diseases, from HIV/AIDS to cancer and others,” he said, “it almost always is a combination approach that over time yields the best results.”