The Food and Drug Administration on Thursday fully approved the Alzheimer’s treatment Leqembi, a pivotal decision that may expand access to the expensive drug for older Americans.
Medicare announced shortly after the FDA approval that it’s now covering the antibody treatment for patients enrolled within the insurance program for seniors, though several conditions apply.
Leqembi is the primary Alzheimer’s antibody treatment to receive full FDA approval. Additionally it is the primary such drug that to receive broad coverage through Medicare.
Leqembi shouldn’t be a cure. The treatment slowed cognitive decline from early Alzheimer’s disease by 27% over 18 months during Eisai’s clinical trial. The antibody, administered twice monthly through intravenous infusion, targets a protein called amyloid that’s related to Alzheimer’s disease.
Medicare coverage is a vital step to assist older Americans with early Alzheimer’s disease pay for the treatment. With a median income of about $30,000, most individuals on Medicare cannot afford the $26,500 annual price of Leqembi set by Eisai without insurance coverage.
Medicare had previously only agreed to cover Leqembi for patients participating in clinical trials after the treatment received expedited approval in January. This policy had severely restricted access to the drug.
To be eligible for coverage, patients should be enrolled in Medicare, diagnosed with mild cognitive impairment or mild Alzheimer’s disease, and have a health care provider who’s participating in a data-collection system the federal government has established to watch the treatment’s advantages and risks.
Joanna Pike, president of the Alzheimer’s Association, the lobby group that advocates on behalf of individuals living with the disease, said although Leqembi shouldn’t be a cure, it can help patients within the early stages of the disease maintain their independence, conduct their every day lives, and spend more time with their families.
“This provides people more months of recognizing their spouse, children and grandchildren,” Pike said in an announcement Thursday. “This also means more time for an individual to drive safely, accurately and promptly deal with family funds, and participate fully in hobbies and interests.”
However the treatment carries serious risks of brain swelling and bleeding. Three patients who participated in Eisai’s study died. FDA scientists have said it’s unclear if Leqembi played a task in these deaths.
Alzheimer’s disease is essentially the most common explanation for dementia amongst older adults and the sixth leading explanation for death within the U.S., based on the FDA.
Dr. David Knopman, a neurologist who makes a speciality of Alzheimer’s disease on the Mayo Clinic in Minnesota, said Leqembi clearly demonstrated a profit to patients in Eisai’s trial, though he cautioned the efficacy of the treatment was modest.
Knopman said appropriately diagnosed and informed patients should give you the chance to make a decision for themselves whether or not they need to take Leqembi after weighing the advantages and risks of the treatment in addition to the potential logistical challenges of finding a spot to receive the twice-monthly infusions.
Medicare coverage
To receive coverage, Medicare is requiring patients to seek out a health-care provider participating in a registry system that collects real-world data on the drug’s advantages and risks. The system is controversial. The Alzheimer’s Association and a few members of Congress are fearful this requirement will create barriers to treatment.
There are concerns that the variety of health-care providers participating in such registries will likely be limited, and that folks in rural towns and other underserved communities could have to travel long hours to seek out such a provider.
The Centers for Medicare and Medicaid Services has arrange a nationwide portal to make it easy for health-care providers to submit the required data on patients receiving Leqembi. The free-to-use portal went live moments after the FDA decision on Thursday.
CNBC Health & Science
Read CNBC’s latest health coverage:
Rep. Anna Eshoo of California, the rating Democrat on the House Subcommittee on Health, and Rep. Nanette Barragan, D-Calif., raised concerns in a letter to CMS last month that patients could struggle to seek out a health care provider participating within the system.
Alzheimer’s is often diagnosed with the assistance of a PET scan to detect the amyloid protein related to the disease or in some cases with a spinal tap. Medicare currently only covers one PET scan per lifetime for dementia. It’s unclear if this system plans to alter that policy.
There’s also concern that there may very well be too few specialist physicians and locations to manage the infusions if Leqembi is broadly embraced as a treatment and patient demand for the antibody is high.
Some studies have estimated that wait times for antibody treatments like Leqembi could range from months to even years over the following decade depending on demand.
Tomas Philipson, who advised the FDA commissioner and CMS administrator through the second Bush administration, said the registry is an unnecessary hurdle and Medicare should drop it, but he doesn’t consider the requirement will create an insurmountable barrier to patients accessing Leqembi.
If demand for Leqembi is high, doctors could have an incentive to take part in the registry and the drug corporations will need to help, said Philipson, an authority on health-care economics on the University of Chicago.
How high demand will likely be for Leqembi is uncertain, he said. Families fearful concerning the serious unwanted side effects may opt to not take the treatment, while others will determine the advantages outweigh those risks, he said.
High cost
Leqembi’s price tag and the treatment’s benefit-risk profile are controversial.
Medicare patients treated with Leqembi can pay 20% of the medical bill after they meet their Part B deductible, based on CMS. Costs may vary depending on whether the patient has supplemental Medicare coverage or other secondary insurance, based on the agency.
Patients could withstand $6,600 in annual out-of-pocket costs for Leqembi even with Medicare coverage, based on a study published within the journal JAMA Internal Medicine. The treatment could cost Medicare as much as $5 billion a yr depending on how many individuals receive the infusions, the study estimated.
Sen. Bernie Sanders, I-Vt., chair of the Senate Health Committee, has called Leqembi’s price “unconscionable” and in a letter last month asked Health and Human Services Secretary Xavier Becerra to take motion to cut back the fee.
Sanders said patient out-of-pocket costs for Leqembi would amount to a sixth of many seniors’ total annual income and noted the high cost of the treatment could increase premiums for everybody on Medicare.
Eisai says its $26,500 annual list price for Leqembi is lower than the corporate’s estimate of $37,600 for the whole value of the treatment for every patient. The Institute for Clinical and Economic Review, a nonprofit that analyzes health-care costs, estimated in April it must be priced at $8,900 to $21,500 per yr.
Though Leqembi could prove costly to Medicare, Philipson said delaying coverage of the treatment would end in significant increased health-care spending as individuals with mild Alzheimer’s disease, which may be managed at home, progress to more serious disease that requires expensive nursing home care.
Philipson and his colleagues on the University of Chicago estimated that delaying Medicare coverage of Alzheimer’s antibody treatments by one yr would end in $6.8 billion in increased spending. By 2040, health-care spending would rise by $248 billion.
Clinical profit
Thursday’s full FDA approval comes after a panel of six outside advisors voted unanimously in June in support of the drug’s clinical profit to patients. The panel was unusually small because some members recused themselves on account of conflicts of interest.
The American Academy of Neurology stated in a February letter to CMS that there’s a consensus amongst its experts that Eisai’s clinical trial of Leqembi was well designed and the outcomes were “clinically and statistically significant.”
Some nonprofit groups resembling Public Citizen, a consumer advocacy organization, strongly opposed FDA approval of Leqembi. A representative from Public Citizen told the advisory panel that the evidence for the drug’s profit doesn’t outweigh significant risks of brain swelling and bleeding.
And representatives from the National Center for Health Research and Doctors for America, also nonprofits, told the panel that Eisai’s clinical trial didn’t include enough Black patients, who’re at higher risk for Alzheimer’s disease.
Leqembi has technically been approved for the U.S. market since January, when the FDA cleared the treatment under an accelerated pathway. The FDA uses expedited approvals to save lots of time and get drugs to patients affected by serious diseases more quickly.
But Medicare refused to cover the Leqembi at the moment, asking for more evidence that the expensive treatment had an actual clinical profit for patients that outweighed the risks.
This system’s cautious coverage policy stems from the FDA’s controversial 2021 approval of one other Alzheimer’s antibody treatment called Aduhelm, also made by Eisai and Biogen.
The FDA’s advisory committee declined to endorse Aduhelm because the info didn’t support a clinical profit to patients. Three advisors resigned after the agency’s decision to approve the treatment anyway.
Knopman is considered one of the advisors who resigned over the FDA’s decision on Aduhelm. He said the info for Leqembi is different. Eisai conducted a clean trial that showed the antibody had a modest clinical profit for patients, Knopman said.
An investigation by Congress subsequently found that the FDA’s approval of Aduhelm was “rife with irregularities.”
Sanders, in his letter to Becerra, said the FDA “has a special responsibility to revive the general public trust after its inappropriate relationship with Biogen through the agency’s review of a previous Alzheimer’s drug, Aduhelm.”