What’s next for the new Alzheimer’s drug? Lots of thorny issues ahead

Biogen said it will start shipping Aduhelm to treatment sites “within about two weeks.”Adam Glanzman/Bloomberg

Despite all the excitement surrounding the FDA’s approval on Monday of a controversial treatment for Alzheimer’s disease, the drug won’t be flowing into a lot of patients’ arms anytime soon.

Aduhelm, made by Cambridge-based Biogen, poses an array of thorny issues that have yet to be untangled, including how to provide the monthly hour-long infusions and whether insurers will pay for the drug despite the $56,000 annual price tag. And some consumers may face hefty out-of-pocket charges, which could make them think twice about taking a drug with known risks and uncertain benefits.

Getting started with Aduhelm will take time, doctors said.

“We’re not prepared to deliver this tomorrow,” said Dr. Brian Silver, interim chair of neurology at UMass Memorial Medical Center, in Worcester. “I think this is going to be a few months at least before we’re set up to do this.”

For example, UMass Memorial has to follow a procedure for adding new drugs to its formulary, and figure out how to integrate Alzheimer’s patients into its infusion center, he said. Doctors will likely wait for guidelines from the American Academy of Neurology, which Silver said is just starting to look into it. And the hospital will need to know what policies Medicare and insurers put in place.

At Tufts Medical Center, Dr. Richard M. Dupee, chief of geriatrics, said he is discussing with the pharmacy department whether the hospital will offer Aduhelm.

“The decision will be based on how many patients want this done,” he said. “I have yet to receive a phone call saying, ‘I want my mom or dad to do this.’ ”

Still, Biogen said it will start shipping Aduhelm to treatment sites “within about two weeks” and it believes that “over 900 sites will be ready to treat a patient shortly.”

The places most likely to be first out of the gate offering treatments are the hospitals that participated in the clinical trials. Biogen identified Mass General Brigham in Boston and Butler Hospital in Providence as two study sites it expects to also administer the drug. Mass General did not reply to several requests for comment.

At Butler Hospital, Dr. Stephen Salloway, director of the Memory and Aging Program, who has studied the drug for many years, said that more than 100 patients had expressed interest before the FDA approval, and other inquiries “are just beginning to come in.” It will take about two weeks for the hospital pharmacy to be able to access the medication, and he said infusion centers throughout the region are also poised to offer it.

But, Salloway added, “We are waiting to hear about coverage from Medicare and other carriers.”

For private insurers in Massachusetts, it will take “weeks to months” to review the data and consult experts, a process that is now underway, said Lora Pellegrini, chief executive of the Massachusetts Association of Health Plans.

Aduhelm is the first new drug for Alzheimer’s disease in nearly 20 years, and the first ever to attack the underlying disease process. But the Food and Drug Administration approved it despite limited data on whether it slows cognitive decline and despite the risk of brain swelling, a side effect that 30 percent of study participants experienced. The approval applies to all people with Alzheimer’s, even though the research studies enrolled only patients with early onset of the disease.

So each insurer will weigh whether the benefits justify the risks and costs, and may decide to limit coverage to people with early-stage illness or require prior authorization.

But the vast majority of people with Alzheimer’s are 65 and older and thus covered by the federal Medicare program. With FDA approval, the drug is automatically covered under Part B, although some administrative steps such as establishing a billing code still have to take place.

But it’s possible some restrictions will be put in place that will slow access, industry experts said. The regional contractors that process Medicare claims could move to limit coverage. And the Centers for Medicare and Medicaid Services, or CMS, may be asked to conduct a study to establish nationwide standards for Aduhelm, a process called the National Coverage Determination, which could take months or even a year to complete.

Theoretically, CMS could determine that Aduhelm is not medically necessary. More likely is that the agency would restrict which patients will receive the drug or require that only those willing to participate in research studies receive it, said Thomas Barker, cochair of the health care practice at the Foley Hoag law firm and former general counsel for CMS.

Still, the agency cannot control how much it pays for the drug, he said. Medicare will pay Biogen the average market price, plus 6 percent.

Barker thinks it’s likely that CMS will pursue a National Coverage Determination, and the decisions that result would be copied by private insurers.

An additional issue for both insurers and Medicare is the costly testing that occurred before and during treatment in the research studies. To confirm that patients have amyloid plaques, the sticky brain proteins that are the hallmark of Alzheimer’s, doctors typically perform a type of imaging called a PET scan. Will Medicare and insurers pay for that? Will they pay for MRIs needed to monitor for side effects?

Another unanswered question: How long should people continue taking Aduhelm?

“There is no end date for the treatment,” said Salloway, the Butler Hospital researcher, “and we do not know yet if the interval between infusions can be lengthened or the medication discontinued.”

That means a lot of expense for individual patients and taxpayers as a group, said Dr. Steven D. Pearson, president of the Institute for Clinical and Economic Review, a drug-pricing watchdog group in Boston. ICER has sharply criticized the FDA decision and estimated that, because of the questionable benefits, Aduhelm should cost only $2,500 to $8,300 a year.

Medicare has no limit on out-of-pocket payments, and will cover only 80 percent of the $56,000 cost, leaving patients with bills in excess of $10,000 a year, Pearson said. While many people have supplemental insurance to pick up some of those copays, that just means further inequities for those who can’t afford such coverage, he said. Plus there will be copays on the PET scans and MRIs, he added.

Pearson called Aduhelm “a clarion example” of the need for a systematic way to manage innovation and cost in drug development.

Despite the controversies, Mark Miller, the former executive director of the Medicare Payment Advisory Commission, thinks Aduhelm will be widely used and make billions for Biogen.

Infusion centers used for cancer patients and others can accommodate Aduhelm patients, said Miller, now the executive vice president at the Arnold Ventures philanthropy. Doctors will make good money providing the drug. Most Medicare patients have supplemental coverage that will pick up the 20 percent copay.

“There’s not a lot of friction here to say, “Don’t do this,’ ” he said.

Anissa Gardizy of the Globe staff contributed to this report.