Across the United States, millions of families are anxiously awaiting a decision on a new drug to treat Alzheimer’s disease. An approval would provide hope for people eager for anything that might improve their loved ones’ lives. Yet if the drug reaches the market, the US health system may have a hard time getting it to those who might benefit.
The Food and Drug Administration is expected to decide by June 7 if the drug, called aducanumab and made by Cambridge-based Biogen, works well enough to allow it on the market. But approval would trigger a 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.
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 neurological 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 United States 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 telltale 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 country has only 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.
The Rand analysis projected that when all constraints on 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 they no longer qualify. It called the United States ’'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’s chief executive, Michel Vounatsos, said on a call with Wall Street analysts in April. The company expects 600 sites 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 are also likely to 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.
Aducanumab’s unusual path to FDA review has heightened scrutiny. Biogen called off two large randomized trials after concluding the drug appeared not to 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 the past it has tended to follow the lead of advisory panels. 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.