The Food and Drug Administration on Thursday gave full approval to the Alzheimer’s drug Rekhembi, a pivotal decision that expands access to expensive drugs for older Americans.
Medicare Shortly after FDA approval, it announced that it would target antibody treatments for patients enrolled in senior insurance programs, subject to some conditions.
Leqembi is the first Alzheimer’s disease antibody therapy to receive full FDA approval. It is also the first drug to be broadly applied through Medicare.
Rekembi is not a cure. The treatment slowed his cognitive decline from early Alzheimer’s disease by 27% over an 18-month period during Eisai’s clinical trial. The antibody, given twice a month as an intravenous infusion, targets a protein called amyloid that is associated with Alzheimer’s disease.
Medicare adoption is an important step in helping older Americans with early-stage Alzheimer’s disease pay for treatment. With an average income of about $30,000, most Medicare participants cannot pay the $26,500 annual price set by Eisai for Rekembi without coverage.
Medicare had previously agreed to cover only patients enrolled in clinical trials after the Rekembi treatment received accelerated approval in January. This policy severely restricted access to drugs.
To be covered, the patient must be enrolled in Medicare, be diagnosed with mild cognitive impairment or mild Alzheimer’s disease, and have a physician participating in a federally established data collection system to monitor benefits and risks of treatment. There is a need.
Joanna Pike, president of the Alzheimer’s Association, a lobby group that advocates for people living with Alzheimer’s disease, said that while Rekumbi is not a cure, it can help patients in the early stages of the disease to remain independent and carry on with their daily lives. said to help. , spend more time with family.
“This will allow people to recognize their spouses, children and grandchildren for more months,” Pike said in a statement Thursday. “This also means more time to drive safely, manage your finances accurately and quickly, and fully participate in your hobbies and interests.”
However, this treatment carries significant risks of brain swelling and bleeding. Three patients who participated in the Eisai study died. FDA scientists said it was unclear whether Lekembi had a role in these deaths.
According to the FDA, Alzheimer’s disease is the most common cause of dementia in the elderly and the sixth leading cause of death in the United States.
Dr. David Knopman, an Alzheimer’s disease neurologist at the Mayo Clinic in Minnesota, said Rekembi was shown to clearly benefit patients in Eisai’s trials, but the therapeutic effect was modest. warned.
Properly diagnosed and well-informed patients, Knopman said, after weighing the benefits and risks of treatment and the potential logistical challenges of finding a place to receive their twice-monthly infusions. He said he should be able to decide for himself whether or not to take Lekembi.
Medicare coverage
To get coverage, Medicare asks patients to find providers who participate in a registry that collects real-world data about the benefits and risks of their drugs. This system is controversial. The Alzheimer’s Association and some lawmakers fear the requirement creates barriers to treatment.
The number of health care providers participating in such registries is limited, and people in rural towns and other underserved areas travel long hours to find such health care providers. There is a concern that it may have to be done.
The Centers for Medicare and Medicaid Services have established a nationwide portal to make it easier for providers to submit the necessary data about patients on Rekumbi. The free-to-use portal went live shortly after the FDA’s decision on Thursday.
In a letter to CMS last month, California Rep. Anna Esheu and Democratic California Rep. expressed concern that it could suffer from
Alzheimer’s disease is usually diagnosed with the help of a PET scan to detect the disease-associated amyloid protein, or sometimes using a spinal tap. Medicare currently only covers once-in-a-lifetime PET scans for dementia. It is unclear if the program plans to change its policy.
There are also concerns that if Lekembi becomes a widely accepted treatment and patient demand for the antibody is high, there may be too few specialists and infusion sites available.
Some studies estimate that Wait times for antibody treatments like Lekhembi could range from months to years over the next decade, depending on demand.
Thomas Philipson, who advised FDA commissioners and CMS administrators during the second Bush administration, said the registry was an unnecessary hurdle and that Medicare should remove it, but this requirement would access Lekembi. He said he didn’t see it as an insurmountable barrier for patients.
Philipson, a health economics expert at the University of Chicago, said if demand for Rekumbi was high, doctors would be motivated to join the registry, and drug companies would want to help.
He said it was unclear how much demand for Rekembi would grow. Families worried about serious side effects may choose not to undergo treatment, he said, while other families will decide that the benefits outweigh the risks.
high cost
The price and benefit-risk profile of lekhembi are controversial.
Medicare patients treated with Rekumbi will pay 20% of their medical bills after meeting the Part B deductible, according to CMS. The agency said costs may vary depending on whether the patient has additional Medicare insurance or other secondary insurance.
Patients could pay up to $6,600 annually The out-of-pocket costs for Lekembi are high, even with Medicare coverage, according to a study published in JAMA Internal Medicine. Studies estimate that treatment could cost Medicare up to $5 billion a year, depending on the number of people receiving injections.
Senator Bernie Sanders (Berney, Barney), chairman of the Senate Health Committee, called the price of Rekumbi “unconscionable” and in a letter last month asked Health and Human Services Secretary Xavier Becerra to cut costs. requested to take measures.
Sanders said out-of-pocket costs for Rekumbi patients amounted to one-sixth of the total annual income of many seniors, noting that the high cost of treatment could raise premiums for all Medicare members. bottom.
Eisai said Rekembi’s annual list price of $26,500 is lower than the company’s estimate of $37,600 in total treatment costs for each patient. The Clinical and Economic Review Institute, a nonprofit that analyzes health care costs, estimated in April that medical costs should range from $8,900 to $21,500 a year.
Rekumbi can be expensive for Medicare, but delays in applying the treatment could result in patients with mild Alzheimer’s who can be managed at home progressing to more severe disease requiring expensive nursing home care. Phillipson said it would lead to a significant increase in health care costs.
Phillipson and colleagues at the University of Chicago estimated that a one-year delay in Medicare’s adoption of Alzheimer’s antibody treatments would increase spending by $6.8 billion. By 2040, he expects healthcare spending to increase by $248 billion.
clinical benefits
Full FDA approval on Thursday came after a panel of six outside advisors voted unanimously in June to endorse the drug’s clinical benefits for patients. The panel was unusually large as some members declined to participate due to conflicts of interest.
In a February letter to CMS, the American Academy of Neurology noted among experts that Eisai’s Rekembi clinical trial was well designed and that its results were “clinically and statistically significant.” He said there was consensus.
some non-profit organizations such as citizenConsumer advocacy groups strongly opposed the FDA’s approval of Rekumbi. A Public Citizen representative told the advisory panel that the evidence for the drug’s benefits did not outweigh the serious risks of brain swelling and bleeding.
Also, representatives of the National Center for Health Research and Doctors for America, also nonprofits, told the panel that Eisai’s clinical trials did not include enough black patients at high risk for Alzheimer’s disease. rice field.
Lekhembi has been technically approved in the US market since January when the FDA approved treatment based on the accelerated route. The FDA uses accelerated approvals to save time and get drugs to patients suffering from serious illnesses sooner.
But Medicare denied coverage for Lekembi at the time, asking for more evidence that the expensive treatment offered patients real clinical benefits that outweighed the risks.
The program’s prudent enforcement policy stems from the controversial 2021 FDA approval of another Alzheimer’s antibody treatment called Aduhelm, also made by Eisai and Biogen.
An FDA advisory panel declined to endorse Aduhelm because the data did not support clinical benefit to patients. Three advisers resigned after the agency’s decision to approve the treatment anyway.
Knopman is one of the advisers who resigned over the FDA’s decision on Aduhelm. He said the Rekumbi data differed. Knopman said Eisai has conducted clean trials that have shown that the antibody offers significant clinical benefit to patients.
A subsequent congressional investigation found that the FDA’s approval of Aduhelm was “rife with fraud.”
In a letter to Becerra, Sanders wrote that the FDA “has a special responsibility to restore public trust following its inappropriate relationship with Biogen during its review of the pioneering Alzheimer’s drug Aduhelm. There is,” he said.