As physicians and health policy experts debate the merits of Aduhelm, the first new drug for Alzheimer’s disease approved in 18 years, patients want to know:
“Will this medication help me — and how much?”
Doctors explaining the pros and cons of Aduhelm won’t have a definitive answer.
“On an individual basis, it will be absolutely impossible to predict,” said Dr. Allan Levey, director of the Goizueta Alzheimer’s Disease Research Center at Emory University.
Cognitive decline varies widely among people who have started experiencing memory and thinking problems or who are in the earliest stage of Alzheimer’s — the patients in whom Aduhelm was tested, Levey said.
“The nature and rate of progression varies tremendously,” he added, “and we’re not going to know when we treat somebody (with Aduhelm) if their progression will be fast or slow or average — we just won’t be able to say.”
Nor will it be possible to specify how much difference this drug would make for a given patient, experts have said.
Uncertainty about the potential benefits of Aduhelm, which received conditional approval from the U.S. Food and Drug Administration on June 7, is considerable. One phase 3 drug trial found that a high dose taken over the course of 18 months slowed cognitive decline by about four months; a second clinical trial failed to show any effect. The FDA is requiring a post-approval trial from drug makers Biogen and Eisai Inc. to supply more data. Final results might not be available until February 2030.
With many unanswered questions about Aduhelm’s approval, the House Committee on Oversight and Reform has opened an investigation. The FDA on Thursday revised the drug’s label to narrow its potential use.
“Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease,” the updated guidance says, “the population in which treatment was initiated in clinical trials.”
Besides uncertainty about the drug’s effective and potential benefits, experts, including Levey, said another important point for patients is that cognitive decline will likely continue even with the drug.
Here are some other key points experts aim to explain:
Potential side effects are common
Brain bleeds and swelling occurred in 41% of patients treated with the highest dose (10 milligrams) of Aduhelm, the subgroup that showed some response in one clinical trial, according to a document released by the FDA. Brain scans identified these as mild in 30% of cases, moderate in 58% and severe in 13%. Most cases resolved, without serious incident, within three months.
The FDA recommends that patients taking Aduhelm get at least three MRI scans of the brain in the first year to check for side effects. Physicians and health systems are discussing what kind of safety protocols are needed beyond these scans.
Other tests will be necessary
Aduhelm was tested on patients with beta-amyloid deposits in their brain that had been confirmed by positron emission tomography brain imaging.
In clinical practice, only patients who have those deposits should take Aduhelm and imaging to confirm that should be required, experts agreed.
But that presents a problem for many patients. Because of their age, most are covered by Medicare, which doesn’t pay for PET imaging outside of research settings. Instead, most medical centers will rely on amyloid cerebrospinal fluid tests, obtained through spinal taps.
There are also genetic tests, which can determine if a person is at greater risk of Alzheimer’s, but those can be expensive, aren’t covered by Medicare and could have psychological consequences for immediate family — who could find out themselves that they are predisposed to the disease.
Therapy will be expensive
Medicare and private insurers have not yet decided whether to impose restrictions on who can get coverage for Aduhelm, which will be administered through monthly infusions at medical centers. With an annual list price of $56,000 for the drug alone, KFF researchers estimate, some Medicare beneficiaries could pay as much as $11,500 out-of-pocket to cover their coinsurance obligation.
Brain scans, monthly infusions, physician services, and amyloid and genetic testing could bump total expenses toward $100,000 a year, some experts have said.
“The most pressing thing we need is an understanding of payment for this medication,” said Dr. Aaron Ritter, a dementia expert with the Cleveland Clinic Lou Ruvo Center for Brain Health in Las Vegas. “Many patients are going to be on a fixed income with limited capacity to pay large amounts.”
Specialists will not prescribe to all Alzheimer’s patients
Although physicians can prescribe an approved drug to whomever they think it will help, dementia experts say Aduhelm should be considered only for patients similar to those in the clinical trials: individuals with mild cognitive impairment (memory and thinking concerns that don’t interfere with their functioning) and with early-stage Alzheimer’s (when symptoms are still mild but functioning becomes impaired).
“We are going to start small and go slow until we understand more” about the medication and how patients respond, said Dr. Maria Torroella Carney, chief of geriatrics and palliative medicine at Northwell Health, New York’s largest health care system.
Since Aduhelm was not tested on people with moderate or severe Alzheimer’s disease, it shouldn’t be given to these patients, several experts said.
Physicians will respect patients’ wishes
Even physicians who worry that Aduhelm’s potential harms might outweigh potential benefits said they will still prescribe the medication, though with caution and careful consideration. Dr. Jason Karlawish, a professor at the University of Pennsylvania Perelman School of Medicine, is among them.
“Now that this medication is available, I have to adhere to a core ethic of the practice of medicine, which is respect for patient autonomy,” Karlawish, co-director of the Penn Memory Center, said. “If patients and family caregivers ask for Aduhelm after a thorough discussion, I’ll be a reluctant prescriber.”
Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation, which is not affiliated with Kaiser Permanente.
Source: Orange County Register