jOH REINSTEIN remember where you were when you heard that the Food and Drug Administration (FDA) had approved Aduhelm (aducanumab), a new drug for Alzheimer’s. It was last June and I was at Kohl’s, a department store, when she started getting news alerts on her phone. She asked a stranger outside the store to take a photo of him jumping in the air, which she immediately posted on Facebook to share the news with family and friends. “It was an amazing feeling because I really felt like there was hope for me and so many others,” he says. “I remember going home and my wife was crying.”
After being diagnosed with Alzheimer’s disease four years ago at the age of 56, Mr. Reinstein thought the wait for treatment was finally over. “I was ready to roll,” he remembers. Alzheimer’s is a brutal and unrelenting degenerative disease that gradually robs patients of their memories and devastates families. But in January the Centers for Medicare & Medicaid Services (cms) proposed that only the drug costs of patients participating in clinical trials be covered. If he cms confirms this decision in April, it will greatly limit who can have the drug.
Despite Aduhelm being heralded as the first treatment to modify the course of Alzheimer’s disease, taxpayers of all stripes have been cool about the drug. Weak evidence of efficacy, troublesome side effects, and a sky-high price are all to blame. The drug has also had a checkered history. In early 2019, Biogen, the biotech firm behind it, stopped its development, saying it didn’t work. Months later, the firm made a youHe turned and said that a new analysis showed that the drug worked after all.
The scientists disapproved of the way the data was reanalyzed. And skeptics pointed to the huge financial gains that approval would bring to the company. A scientific advisory panel of the FDA said the drug should not be approved. But the agency overruled his committee.
To further complicate matters, the company priced the drug at $56,000 a year. The Institute for Clinical and Economic Review estimates that the drug should be priced between $3,000 and $8,400 a year if it works (something that hasn’t been proven). Although the list price has since dropped to $28,200 a year, taxpayers have so far been extremely reluctant to hit it. Many private health insurers have said they want more proof of effectiveness before paying for it, and there are concerns about adverse effects such as brain swelling and bleeding.
Biogen urgently needs the government to purchase the drug. It recently emerged that the struggling company is cutting around 1,000 jobs (more than 10% of its workforce). In January, during a call with investors, Biogen boss Michel Vounatsos urged Aduhelm’s supporters to fight the CMS’decision of s. And UsAgainstAlzheimer’s, an advocacy group that receives funding from Biogen, recently launched a major ad campaign aimed at influencing the final decision.
“When we got the CMS’s decision, we were actually a bit surprised. I used a lot of swear words,” says Reinstein. Like most patients, he cannot afford to pay for the medicine out of his own pocket. Patients argue that cms has exceeded his or her authority by denying coverage for a FDA-Approved drug. Some also worry that the decision will restrict the market for other drugs in development that work in the same way but could be more effective.
Patients argue that Aduhelm is much cheaper than expensive cancer therapies or genes. However, the prevalence of Alzheimer’s disease, which affects 6 million people in the United States, means that annual use of such an expensive drug will soon become unaffordable. One study estimated that if 1 million patients were given Aduhelm, the annual cost would be $73 billion by 2028. Although Biogen has lowered its price since this analysis was done, the widespread use of this drug presents a major challenge for Medicaid and Medicare.
As discussions continue, those who are missing out on treatment fear it may be too late for them to benefit from the drug. “I am 60 years old. I want to spend time with my grandchildren and my family,” explains Mr. Reinstein. “And the longer they delay, the longer than this [disease] can progress. I am the perfect candidate for this. Wait another two years, it may not be.” ■
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This article appeared in the US section of the print edition under the headline “Ancient Problem.”