Geri Taylor knew, perhaps better than most, that what she was experiencing was cause for alarm. A former nurse and healthcare executive, Taylor had come face to face with Alzheimer's disease throughout her career and recognized its early signs. She was having a tough time remembering, and not just things like where she set her keys. In a defining moment in 2012, Taylor, then 69, looked in the mirror and didn't recognize her reflection.
She and husband Jim Taylor soon went to see a neurologist, who gave a diagnosis of early cognitive impairment. But it wasn't until they tried enrolling her in a clinical trial for an experimental drug that Geri learned she indeed had Alzheimer's.
Much has changed in the years since. To Geri, the disease's most noticeable effect is that words now remain just out of reach. "I have these wonderful thoughts. And on my way to putting words together, I can't remember what they were about." Jim, meanwhile, admits he's had a "much more challenging" time adjusting to this new normal than his wife.
Geri is one of roughly 6 million people in the U.S. living with Alzheimer's, a disease that steadily breaks down memory as well as the brain and body. Until this week, patients and their caregivers had no treatments for the disease, only a handful of drugs that could help alleviate some of its symptoms.
But in a historic and controversial decision, the Food and Drug Administration approved a new medicine on Monday that's meant to slow the cognitive decline caused by Alzheimer's, despite conflicting evidence about whether it's truly effective. Developed by the companies Biogen and Eisai, the medicine — now known as Aduhelm — is the first new Alzheimer's treatment approved in nearly 20 years.
For the Taylors, Aduhelm has long been a source of hope. The trial Geri applied to, the one that confirmed her Alzheimer's, was an early study of Aduhelm, and Geri ended up on the high dose that would ultimately gain FDA approval.
Aside from about an 18-month period between when that study ended and a different, so-called "extension" one began, Geri has been receiving hour-long infusions of Aduhelm each month. She believes it's helping combat her disease.
"It’s the lifeline," she told BioPharma Dive. "Every time I get a shot, as soon as it's over, I’m sure I feel better. That's what I've got. That's all I've got."
Many patients feel the same. Yet, with Aduhelm now headed to market, there are outstanding questions about how they'll be able to afford and access the drug. Biogen set the average price for Aduhelm at $56,000 per year, which is far above what some stakeholders had expected. According to The Wall Street Journal, Cigna, one of the nation's largest health insurers, estimates some eligible patients may end up paying $10,000 or more in annual out-of-pocket costs.
Sticker shock could be felt widely, as the FDA cleared Aduhelm with hardly any restrictions to its use. The Institute for Clinical and Economic Review, a prominent watchdog group for drug pricing, sharply criticized Biogen for the price it set as well as the FDA for approving a drug with known safety concerns but unclear benefits to such a large population.
"Alzheimer's disease has a tremendous impact on patients and loved ones, and no one can be insensitive to the hopes and the fears that are part of their daily lives," ICER said in a statement. "But no one should assume that approving a drug with such conflicting and uncertain evidence will necessarily help patients and families."
In conference calls with analysts and in an interview with BioPharma Dive, Biogen executives defended the drug's price, which they compared to specialty drugs for cancer and inflammatory diseases.
"We believe that this price is really fair," said Chirfi Guindo, head of global product strategy and commercialization, in an interview. "It is sustainable and we remain open-minded."
More broadly, there aren't enough neurologists to diagnose what could be a dramatic influx of patients looking to be treated. Biogen estimates between 1 million and 2 million people could test positive for the toxic protein its drug targets, a figure that some providers fear would overwhelm treatment centers.
"They're aware of the amount of trouble this will cause if it becomes FDA-approved, with hundreds of thousands of patients calling their clinics to get the treatment," said Thomas Wisniewski, director of the Center for Cognitive Neurology at NYU Langone Health, in an interview early this year.
Even if there were enough specialists, one of the tests used to diagnose Alzheimer's — known as a positron emission tomography, or PET, scan — is costly, and often not covered by insurance. The FDA is also requiring that patients on Aduhelm be monitored via magnetic resonance imaging, or MRI, for symptoms of the drug's more worrisome side effects, which may create another logistical challenge.
"I'm quite concerned that the data doesn't support its use, and even practically, that we don't have a good plan for how it could be used safely," said Mary Sano, director of the Alzheimer's Disease Research Center at Mount Sinai School of Medicine.
For Biogen, addressing these concerns will critical. The Cambridge, Massachusetts-based company bet heavily on Aduhelm, hoping that a first-of-its-kind Alzheimer's drug could offset problems elsewhere in its business. Now that approval is in hand, the potential insurance and diagnosis obstacles must be addressed for Aduhelm to live up to Biogen's multibillion-dollar sales expectations.
The company has been preparing for Aduhelm's debut for months. Executives have laid out plans to spend $600 million on its launch, and in a presentation Tuesday, they said 900 or so Alzheimer's treatment centers appear ready to administer the drug.
Biogen also said it's been pressing insurers to cover PET scans, while at the same time running its own testing program for that crucial protein, called amyloid. The company announced Monday a partnership with CVS Health that will make cognitive screenings available in 14 metropolitan areas, through the latter's Project Health initiative.
The Taylors noted to BioPharma Dive that one of the major advantages of participating in Biogen's studies has been the required brain scans are paid for.
"We personally aren't concerned. We're assuming we will continue on in the trial, because they’ll want to know about the long-term impacts," Jim said. "But it is something to be concerned about."
Another new partnership, with Cigna, will attempt to improve access by tying costs to how well a patient performs on Aduhelm.
Biogen claims it's trying to reach underserved communities, too, with other programs promoting equitable access and financial assistance. Recent reporting from STAT has detailed how, despite being more likely to develop Alzheimer's and other dementias, Black and Hispanic people are far less likely to be diagnosed and, in turn, properly treated.
"The expense is really quite high, and I'm not even talking about the drug," said Sano. "Just to have something delivered the way this is delivered, and require the imaging that it requires. It's really expensive."
Aduhelm's cost is all the more in focus given the continued debate about whether the drug's effect on amyloid actually translates to cognitive and functional benefits.
The main clinical trial supporting Aduhelm's approval found patients on the high dose declined about four-tenths of a point less on a cognitive scale compared to those who got placebo. Doctors who spoke to BioPharma Dive said that, to have a meaningful impact on patients' daily lives, a drug would likely need to at least slow cognitive decline by one or two points.
Those findings, however, contrasted with results from a separate, identically designed trial that saw placebo-treated patients do marginally better than those given high-dose Aduhelm. The contradictory results, combined with the ways Biogen analyzed them, led FDA advisors and the agency's own statisticians to strongly oppose Aduhelm's approval.
That the FDA still cleared Biogen's drug was a surprise to many, among them Michael Sherman, chief medical officer of the New England-based insurer Harvard Pilgrim.
Sherman told BioPharma Dive ahead of Monday's decision that, unlike with other drugs with the potential to have large budget impacts, his team wasn't doing as much modeling for Aduhelm, in part because they didn't expect it to get approved.
Sherman noted, though, that in the event of an approval, he'd consider a price tag of $10,000 or more to be "expensive." ICER, the nonprofit cost watchdog, previously estimated a price between $2,500 and $8,300 a year could be considered cost effective, if the drug's apparent benefits were assumed to be true and not a false positive.
"It's hard to answer what I think the drug is worth if we don't think it should be approved in the first place," Sherman said.