Celine Castronuovo, Bloomberg News, June 15, 2023
Expanded access to the Alzheimer’s drug that Eisai Co. developed with Biogen Inc. is unlikely to happen quickly even if the FDA decides follow-up studies confirm the drug helps slow the disease, health policy analysts and researchers say.
Patient advocacy groups are anxiously awaiting what they hope will be a favorable decision by the Food and Drug Administration on Leqembi’s traditional approval application, which six independent advisers to the agency voted unanimously in support of last week.
The FDA’s final decision is expected by early July on the pricey drug, which was previously cleared under the agency’s accelerated approval pathway in January as an early treatment option to slow the progression of the disease that impacts more than 6 million Americans. It could open the door to expanded coverage for Medicare patients, but could also increase federal spending for the infusion therapy that hasn’t been widely prescribed outside of clinical trials.
Only patients whose physician participates in a new registry that can collect additional data on the drug’s performance in the real world would be allowed to access the drug, said the Centers for Medicare & Medicaid Services. The CMS currently only covers the drug during clinical trials.
The Medicare agency said in a press release earlier this month that “broader” coverage would begin “on the same day the FDA grants traditional approval.” But patients, doctors, and analysts are all doubtful, citing the limited information publicized by the agency on how the registry will work.
[….]
Leqembi is currently priced at $26,500 a year, and a study published in the Journal of the American Medical Association estimated that coverage could cost Medicare up to $5 billion a year.
The CMS said in an email that it hopes to make the registry as user-friendly as possible, and that it plans to release more information soon on what information providers will need to enter.
“If the FDA grants traditional approval, CMS is prepared to ensure anyone with Medicare who meets the criteria is covered,” said Sara Lonardo, senior adviser at the CMS Office of Communications.
How Will It Work?
Medicare’s plans for a patient registry are in line with its 2022 coverage decision for amyloid-targeting therapies aimed at slowing the progression of Alzheimer’s disease. The CMS said at the time it needed additional evidence on the new class of therapies before deciding if it’s reasonable and necessary to provide broader coverage.
Under the CMS coverage plan, Medicare coverage for Leqembi would be available for Part B beneficiaries diagnosed with mild cognitive impairment or early dementia caused by Alzheimer’s disease once FDA granted traditional approval. Patients must also “have a qualified physician participating in a registry,” with “an appropriate clinical team and follow up care,” the CMS said.
The registry will be designed to answer questions around whether the amyloid-targeting therapy meaningfully improves health outcomes, such as by slowing the decline of cognition and function for patients, the CMS said in an email.
The registry will also measure whether harms such as brain hemorrhage and edema associated with the use of the therapy change depending on a patient’s characteristics or where it’s administered, and if any benefit or harm changes over time, the CMS said.
Diana Zuckerman, president of the National Center for Health Research, said the CMS patient registry is a “reasonable strategy that will maximize the number of patients who can get access.”
“If you don’t collect information about it, patients and family members can’t make informed decisions,” Zuckerman said, adding that understanding how the therapy works among diverse populations is especially important.
Black Americans made up just 2.3% of the study population in Leqembi’s phase 3 clinical trial, despite being disproportionately impacted by Alzheimer’s disease compared with White Americans.
[….]
“Approval doesn’t necessarily mean instantaneous prescribing,” said James Galvin, chief of cognitive neurology and director of the Comprehensive Center for Brain Health at the University of Miami Health System.
“There’s a lot of logistics that need to be figured out, not just with the registry, but also, where do we send them to be infused? Are the outpatient infusion centers equipped to do this?” Galvin said.
[….]
To read the entire article, click here.