Biogen's New Alzheimer's Drug Faces Hurdles Reaching Patients, Even if It's Approved

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Across the U.S., millions of families are anxiously awaiting a  decision on a new drug to treat Alzheimer’s disease. An approval would provide hope for families eager for anything that might improve their loved ones’ lives. Yet if the drug reaches the market, the U.S. health system may have a hard time getting it to the people who might benefit.

The Food and Drug Administration is expected to decide by June 7 if the drug, called aducanumab and made by Biogen Inc., works well enough to allow it on the market. If they do, it will trigger a cascading series of questions about who will get the new drug, how they’ll get it, how much it will cost and who will pay for it.

Aducanumab, delivered in monthly intravenous infusions, is thought to slow the progression of Alzheimer’s in people who show early signs of cognitive impairment. No drug has yet been shown to do that. Patients need to get it early: If their dementia progresses too far, they’ll no longer qualify for the drug.

A 2017 Rand Corp. analysis estimated that 88 million Americans 55 and older may be eligible for initial screening for a drug like aducanumab that targets the early stages of decline. Of those, 2.4 million might ultimately be candidates for treatment. Biogen hasn't shared plans for pricing, but one analyst estimated the drug could cost $30,000 per year.

Among the questions ahead: How will Medicare and private health plans reimburse for the medication? How will eligible patients be identified? Will there be enough capacity at infusion sites and imaging centers to meet demand? And how will doctors assess patients’ progress on a drug whose only claim is to slow the rate of disease, when that rate can vary naturally?

“This is a pretty tall order for health systems to tackle,” said Soo Borson, professor emerita of psychiatry and behavioral sciences at the University of Washington.

Choke Points

Although similar therapies are under development, aducanumab would be the first drug authorized to alter the course of the most common type of dementia. It has a controversial history, yet families that have been waiting for an Alzheimer’s treatment are eager for it.

“We’ve had people calling on the phone already,” said Christine Mann, chief operating officer of Dent Neurologic Institute in Western New York, a large outpatient neurologic center. 

If it reaches the market, a series of potential choke points could block the medicine from the people it might help.

First, patients will need screening to determine if they have the early signs of cognitive trouble that may signal Alzheimer’s but could also have other causes. Primary care doctors may detect the first indications, but the U.S. has a limited pool of dementia specialists needed to confirm a diagnosis of mild cognitive impairment.

The next step requires confirming whether patients have amyloid plaques, a tell-tale marker of Alzheimer’s in the brain, typically through a positron emission tomography (PET) scan. Depending on the FDA’s judgment, a test to measure amyloid levels in spinal fluid may be an easier and less expensive alternative. 

Those who qualify for the drug would need monthly visits to infusion centers, spending an hour tethered to intravenous drips. The U.S. only has so many infusion chairs. Dent Neurologic and other centers are contemplating extending their hours and adding capacity, including building new infusion  facilities, if aducanumab is approved.

Biogen's New Alzheimer's Drug Faces Hurdles Reaching Patients, Even if It's Approved

The Rand analysis projected that when all constraints around diagnosis and delivery are accounted for, average wait times for a new Alzheimer’s drug could be 18 months, a period when some patients might deteriorate to the point when they no longer qualify. It called the U.S. “ill-prepared” for the volume of patients. (Biogen sponsored the analysis, but the study’s author says the results were independent.)

“We know that the availability of specialist and diagnosis capabilities are a bottleneck,” Biogen Chief Executive Officer Michel Vounatsos said on a call with Wall Street analysts in April. The company expects 600 sites around the country to be ready to administer the drug immediately, with more to follow. Biogen didn’t respond to requests for comment.

Once in treatment, patients on aducanumab may need ongoing evaluation to assess their cognitive function. They’ll also likely need regular scans to monitor for brain swelling, a frequent side effect that appeared in trials and can be dangerous in severe cases.

Health insurers and drug benefit managers are preparing for a possible influx of patients.

“We anticipate that this will be an expensive therapy for a previously unaddressed problem, and so demand will be great,” said Steve Miller, chief clinical officer at Cigna Corp.

He expects health plans will require that patients who get the therapy strictly match the indication specified by the FDA. “You’re going to have probably a very narrow use group,” Miller said.

Heightened Scrutiny

Aducanumab’s unusual path to the FDA review has heightened scrutiny. Biogen called off two large randomized trials, after concluding the drug appeared to not work. The company later reversed course, applying for approval after analysis of further data from one of the two trials appeared to show some effect.

A panel of outside advisers to the FDA last year voted against approval, a judgment the agency isn’t bound by, though in previous instances it’s tended to follow the lead of its advisory panel. A draft report from the Institute for Clinical and Economic Review, a nonprofit that assess the value of new medicines, cited “substantial uncertainty” about aducanumab’s benefits.

Since Alzheimer’s primarily affects older people, how Medicare handles reimbursement for the drug and associated care will play a large role in determining its affordability. The U.S. health program for seniors currently only covers amyloid PET scans in limited circumstances. A spokesperson for the Medicare agency said it’s evaluating payment and coverage rules for aducanumab in the event the FDA approves it.

Borson of the University of Washington said inadequate screening and late diagnosis have long been problems for dementia care. Patients often don’t get the help they need, like backup support for family members and links to social services, she said. The long-awaited arrival of an Alzheimer’s medication could change that. “If aducanumab or any of these other agents is approved, it’s going to add fuel to all of these efforts.”

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