'You reach for anything': Doctors must decide whether patients should get the new $56,000 Alzheimer's drug, Aduhelm

• Biogen's drug Aduhelm is the first Alzheimer's drug on the market in nearly two decades
• Doctors must decide whether to prescribe the $56,000-a year treatment that has shown little clinical benefit.
• The drug's $4,312 per treatment cost has sparked affordability concerns among consumer advocates

The first new Alzheimer’s drug in nearly two decades will be shipped to health providers in the next two weeks, and it couldn't come soon enough for New York resident Gregory Guercio.

Guercio’s wife, Marie, 72, was diagnosed with Alzheimer’s more than two years ago. Treatments have done little to slow the march of the incurable disease. His hope rests with Aduhelm, a pricey drug that targets sticky clumps of protein in the brain suspected of scuttling memory and thinking in Alzheimer's patients.

“When something like this comes out, you reach out for anything,” said Guercio of Long Island, New York. “You don’t have any expectations. You just hope.”

Biogen said it will begin shipping the drug in two weeks to about 900 health facilities nationwide, a mix of facilities that served as clinical trial sites as well as clinics set up for care of Alzheimer’s patients. The drug is administered by IV once every four weeks.

This week, the Food and Drug Administration gave broad approval for Aduhelm for Alzheimer’s patients, but doctors must decide whether to prescribe the $56,000-a- year treatment that has shown little clinical benefit and significant side effects to patients.

The agency granted accelerated approval of the drug, a standard to provide earlier access to patients with serious diseases. Its maker, Biogen, must conduct a post-approval study to monitor the drug's effectiveness.

Clinical trials show Aduhelm is effective at removing amyloid beta, a sticky protein that forms clumps and plaques in the brains of Alzheimer’s patients. Two large trials reported mixed results on whether the drug effectively slowed clinical decline from Alzheimer’s, a disease that afflicts 6.2 million Americans. 

The trials showed patients treated with the monoclonal antibody had side effects such as brain swelling, minor bleeding and headaches while taking the medication. 

'A stampede to the gate'

Gayatri Devi, a neurologist at Lenox Hill Hospital in New York City, fielded a flurry of queries this week from families of loved ones desperate for a chance at getting the drug. 

She said the drug has a real chance at slowing memory, thinking and functional problems in some patients who can do daily tasks but are at risk of cognitive decline over the next year. But she's wary of the drug's side effects

"In a situation like this where there is almost a stampede to the gate, caution is the most important thing," Devi said.

In the two clinical trials, 41% of participants on the drug experienced side effects such as painful brain swelling, headaches and dizziness, compared with 10% who took a placebo. A smaller percentage of patients who received the drug in clinical trials had small bleeds in their brain.

The drug's label requires patients to obtain MRIs before their seventh and 12th treatment to monitor for such side effects. Patients are allowed to continue treatment after a clinical evaluation and if brain imaging shows swelling has not increased, according to the label.

Devi said the high percentage of patients reporting side effects in clinical trials is a "significant concern," and she plans to order MRIs if her patients or their caregivers report ailments such as headaches or confusion. She said she is more likely to prescribe if a patient has support from caregivers who can monitor and report side effects.

"That’s my big concern as a physician – first, do no harm," Devi said. "There is a lot of desperation involved with this condition."

Dr. Pierre Tariot, director of the Banner Alzheimer's Institute in Phoenix, said Aduhelm gives doctors who treat Alzheimer's patients the first new approved drug in 18 years. It's the first drug to attack amyloid beta plaques that are a hallmark of the disease.

"So it is certainly a big deal," Tariot said.

More:FDA approves new Alzheimer's drug, Aduhelm, the first in nearly two decades

More:FDA will decide fate of contentious Alzheimer's drug. The stakes 'could not be higher.'

A 'poster child' of high cost concerns

Though the drug has raised hopes for doctors and patients, its $4,312-per-treatment price tag has sparked affordability concerns among consumer advocates. 

A Biogen representative said in a statement that co-pay and infusion cost-assistance programs might reduce out-of-pocket costs for privately insured patients.

Medicare, the government-run insurance program for adults 65 and older, generally covers FDA-approved injectable or infused drugs but typically requires beneficiaries pay 20% of the drug's cost after meeting a deductible.

Medicare supplement plans can further reduce the cost of co-pays or co-insurance, but consumers must pay the premiums for such plans, said David Mitchell, founder of Patients for Affordable Drugs and a consumer representative on the FDA's Oncologic Drugs Advisory Committee.

Mitchell said Biogen should have priced the drug lower because the company must complete a post-approval study to prove the drug provides a clinical benefit.

The nonprofit Institute for Clinical and Economic Review, which analyzes drug prices, reported last month a fair and cost-effective price would range from $2,500 to $8,300 per patient each year. In a statement released this week, ICER said the FDA's approval failed to protect patients, and Biogen could collect more than $50 billion per year "even while waiting for evidence to confirm that patients receive actual benefits from treatment."

Aduhelm's price underscores the need for Medicare to negotiate a better deal for beneficiaries, Mitchell said.

"I am concerned that we are watching our system bend, break, buckle under the weight of this kind of drug and this kind of pricing," he said. "This drug is the poster child for why Medicare needs to be able to negotiate prices."

Alzheimer's Association officials said the organization will work to ensure the drug and related testing is covered by public and private insurance, so it is widely available to patients and families who might benefit.

Harry Johns, president and CEO of the Alzheimer's Association, acknowledged that some scientific experts called for another clinical trial to show the drug works. Last November, the FDA's Peripheral and Central Nervous System Drugs Advisory Committee of outside experts recommended the agency not approve the drug. 

Johns said the agency struck the right balance to allow patients access to the drug while requiring a post-approval study measuring the drug's effectiveness. 

"We believe this is so important because it gives people opportunity for access today and confirms efficacy in the process,"  he said. 

At home in New York, Guercio said he closely monitored the news about Aduhelm's clinical trials and this week's FDA approval.

His law firm provides health insurance coverage for him and his wife. He said he does not know what his out-of-pocket costs might be to pay for the drug. The 73-year-old will continue to work and earn money from his law practice, which represents school districts on Long Island and at the state's capital.

"You are not going to turn to your spouse and say I am going to deny you that hope because of the money," he said.

As the primary caregiver for his wife, he has witnessed the devastating effect Alzheimer's has had. He has modest expectations.

"You try to stay realistic because they’re not holding it out as any wonder drug, that’s for sure," Guercio said. "Whatever benefits you get has got to be a plus for us, that’s the way we look at it. We are married for 50 years. We’re hoping for more."

Ken Alltucker is on Twitter at @kalltucker, or can be emailed at alltuck@usatoday.com