TALLAHASSEE — Monday’s announcement gave hope to a community desperate for it.
For the first time in nearly two decades, the U.S. Food and Drug Administration approved a drug, aducanumab, to treat Alzheimer’s disease, the horrific ailment that strips people of their cognitive function.
Well over 500,000 Floridians suffer from Alzheimer’s, and many of those patients and families were on the front lines pushing for the drug’s approval.
“It’s a game-changing, celebratory victory because it’s the first drug we’ve had that treats the disease itself,” said Michelle Branham, the Gov. Ron DeSantis-appointed chair of the Florida Alzheimer’s Disease Advisory Committee.
Branham is also the vice president of public policy for the Florida arm of the nonprofit Alzheimer’s Association, which lauded the federal government’s decision this week. (The Association discloses on its website that it accepts hundreds of thousands of dollars from pharmaceutical companies annually, including from Biogen, the manufacturer of this new drug.)
But the approval comes with significant potential downsides for Florida, too. In a state where prescription drug affordability is already a major issue, the infusions are expected to cost about $56,000 per patient per year before insurance, according to Biogen.
Critics say that’s a lot of money for a drug that may not work as advertised: A Food and Drug Administration advisory panel voted overwhelmingly against recommending the drug in November because it said the treatment was not proven to treat Alzheimer’s.
Here are answers to four key questions about aducanumab, which will go by the brand name Aduhelm.
1. How does aducanumab work?
The concept behind aducanumab is at the cutting edge of Alzheimer’s research. The drug is a monoclonal antibody that targets the amyloid plaque buildup on the brain that is a characteristic of Alzheimer’s disease. It’s given to patients via monthly injections.
In one of the trials conducted by Biogen, aducanumab appeared to slow cognitive decline in patients.
“For people living with this disease, that’s huge. It could mean a delay in putting a loved one in an assisted living facility,” said Dr. Amanda Smith, director of clinical research at the University of South Florida’s Byrd Alzheimer’s Center and Research Institute, who worked on a clinical trial. “It could mean that for two, three, four extra years, they can go use the toilet by themselves and you don’t have to take them.”
In another clinical trial, however, aducanumab appeared to have little effect on its patients. Smith said the reasons for this were complex, but they likely had to do with the relatively small dosages given to patients in that trial. Still, for a drug with such severe side effects — brain bleeding occurred in about one in six high-dose patients in one of the trials — critics argue the benefits have to be worth the considerable risks.
2. Why was aducanumab’s approval so controversial?
In short, critics argue aducanumab’s benefits are not worth the risks. The federal advisory panel’s ruling from last year, for example, dealt a blow to the drug’s credibility as an Alzheimer’s treatment.
Others have noted the exorbitant cost. Not only will patients have to either get the treatment covered by insurance or find a way to pay for it out of pocket, but there will be other costs: paying for procedures to detect amyloid plaque, paying for the costs of a monthly infusion and follow up tests, and more. (A Biogen spokesperson said in a statement that the company plans to help patients through “co-pay and infusion cost assistance programs.”)
Then there’s the broader cost to the state. Floridians — even those with insurance — already have so much trouble affording prescription drugs, DeSantis has made a plan to import treatments from Canada a major part of his health care policy agenda.
Some experts worry aducanumab could make drugs less affordable overall and worsen the national debt. Medicare would likely cover at least a portion of the drug’s cost to individuals because many of those plans come with limits to out-of-pocket prescription drug costs, a Biogen spokesperson noted in an email. Depending on what Medicare decides to cover, taxpayers could be on the hook for billions.
Some experts also worry a rash of new prescriptions for the pricey drug could drive up health care premiums for everyone.
When asked whether DeSantis is concerned about the cost of the drug to the consumer or federal government, a statement emailed by a spokesperson did not directly address the cost issue.
“Governor DeSantis wants every Alzheimer’s patient in Florida to have access to safe and effective treatments,” the spokesperson, Christina Pushaw, said in part in the statement, noting DeSantis had nothing to do with the drug’s approval.
3. Who could benefit from this drug?
Although the drug has only been studied in patients with, at worst, mild cognitive impairment due to Alzheimer’s, the federal government approved the drug for general treatment of the disease.
Smith said the decision to grant such broad approval concerned her more than anything else about the process. Advocacy groups pushed harder than anyone for the drug’s approval, Smith noted. What will happen when families are told this revolutionary treatment may not be right for their loved one?
“I think there’s going to have to be some hard conversations between patients and their families and providers,” said Dr. James E. Galvin, a professor of neurology at the University of Miami Miller School of Medicine. (Galvin noted he worked a clinical trial for the drug, and also consulted for Biogen as they reviewed the data.)
The Food and Drug Administration approved aducanumab under its ”accelerated approval pathway.” The agency gives this approval when a drug has been shown to improve one aspect of the disease even if the drug’s ultimate benefit is more uncertain. In this case, the agency says that even if aducanumab’s overall effect on the progression of Alzheimer’s is unproven, the drug has been shown to reduce amyloid plaque.
This approval poses an important challenge for physicians. How will doctors determine whether a patient has a buildup of amyloid plaque? There is no simple test for amyloid; only a PET scan, a spinal tap or an expensive blood test can reveal the presence of the plaque.
And even more broadly, if a doctor refuses to prescribe the drug to a patient, what’s to stop the patient from doctor shopping until they find a physician that will prescribe the treatment?
“I think there’s a lot of challenges and unanswered questions that we need to address,” Galvin said.
For Biogen’s part, the company spokesperson said it will continue to focus on patients “in the earlier stages of Alzheimer’s disease.”
4. What else about the drug is unknown?
Almost everything about how the drug will perform in a clinical population is unknown. Patients don’t yet know which insurance plans will cover the aducanumab. It’s unclear how doctors will prescribe it, given the federal government’s broad approval. It’s unclear what percentage of Florida’s Alzheimer’s patient population will have access to the drug. It’s likewise unclear who in that population would benefit from the drug.
As part of its accelerated approval, the Food and Drug Administration has required Biogen to conduct a phase four clinical trial to demonstrate the drug’s benefit. If the drug doesn’t pass muster, the agency could pull it from the market. But it’s unclear when that could happen.
Despite all of this uncertainty, patient advocates know their next move.
“The next priority as an association is going to be to advocate heavily for access to the drug,” Branham said.
Biogen says it will start shipping the treatment to clinics within two weeks.