At least half-a-dozen private health insurers in some of the nation’s largest states are balking at covering Biogen’s controversial drug for Alzheimer’s disease, saying it is an experimental and unproven treatment despite being approved by the federal government one month ago.
Six affiliates of Blue Cross and Blue Shield in Florida, New York, Michigan, North Carolina, and Pennsylvania indicated in policies posted online they will not cover the Cambridge biotech’s drug, Aduhelm, because they consider it “investigational” or “experimental” or because “a clinical benefit has not been established.” Aduhelm, which is priced at $56,000 a year, is intended to slow cognitive decline in patients with early Alzheimer’s symptoms, regardless of their age.
James Chambers, an associate professor of medicine at the Tufts Medical Center Institute for Clinical Research and Health Policy Studies, said that insurers have occasionally opted not to cover expensive specialty medicines for rare diseases, but that he’s never seen firms refuse to pay for an approved drug that could be prescribed to millions of people.
“This is unprecedented,” said Chambers, who has spent seven years studying how private insurers decide which drugs to cover. “Maybe it’s not entirely surprising given the controversy surrounding the drug’s approval, but it’s not something I’ve seen before.”
The Food and Drug Administration approved Aduhelm on June 7 over the objections of its independent advisory committee and some medical experts who said the results of clinical trials were muddled at best.
Helen Stojic, a spokeswoman for Blue Cross Blue Shield of Michigan, acknowledged on Wednesday that the insurer’s website says Aduhelm was “considered investigational/experimental” due to “insufficient evidence of a clinical benefit.” But, she said, the insurer hasn’t decided yet whether to cover it.
Analysts say that insurers sometimes say they won’t cover a drug and then reverse themselves because of a backlash from patients or because rival companies do cover it.
So far, no private insurer in Massachusetts has said whether it will cover the medication. The largest, Blue Cross Blue Shield of Massachusetts, with 2.8 million members, is reviewing the trial results and conferring with doctors, and “will likely have a final decision” by the end of July, said spokeswoman Amy McHugh.
The state’s second-largest insurer, Point32Health ― which was formed by the recent merger of Tufts Health Plan and Harvard Pilgrim Health Care and has 2.2 million customers ― is also scrutinizing the drug. Its chief medical officer, Michael Sherman, last month accused Biogen of putting “excessive corporate profits” ahead of patients in the pricing of its medication. He also said Biogen should cut the cost of the drug to $5,400, given its questionable benefits and potential risks, or the insurer might not cover it.
A Biogen spokeswoman, Allison Parks, said Tuesday that the drug maker was “disappointed” by the out-of-state insurers’ decisions and that only a “very small portion” of potential patients would be affected. She said the insurers were mischaracterizing Aduhelm.
“Typically, an experimental drug is one that has not yet entered clinical trials, whereas an investigational drug is one that is being studied in trials but has not yet received marketing approval from the FDA,” she said. “Aduhelm is approved by the FDA and is neither experimental nor investigational.”
The vast majority of the roughly 6 million Americans with Alzheimer’s disease — 96 percent by one estimate — are over age 65 and would rely on Medicare as their primary insurer for coverage of Aduhelm. Biogen estimates there are 1 million to 2 million people who have early symptoms and might benefit from the drug, which the FDA last week recommended that doctors start patients on only if they have mild cognitive impairment. Nonetheless, many Medicare recipients would rely on secondary private insurance for other costs associated with Aduhelm.
On Monday, the Centers for Medicare & Medicaid Services, or CMS, announced it has begun reviewing Medicare coverage for Aduhelm. The outcome of that process — formally known as a “National Coverage Determination” — will have far greater impact for potential recipients — and for Biogen. A preliminary decision is expected within six months, with a final ruling coming within nine months.
The federal agency’s decision will also have an enormous effect on the Medicare budget. Analysts have predicted that Medicare and its enrollees, who pay a share of their prescription drug costs, could spend billions of dollars on Aduhelm annually, possibly more than what the US government spends on some agencies, such as NASA.
Chambers, the Tufts researcher, said private insurers typically wait for CMS to decide on coverage and often follow the government’s lead. “Medicare is the gorilla in the room,” he said.
But, by jumping out ahead of the federal decision, he said, some private insurers are “drawing a line in the sand and making a point about a very expensive drug.”
To justify their decision not to cover Aduhelm, some of the insurers cited withering assessments of the drug, including a May article co-written by Dr. Aaron Kesselheim, a Harvard Medical School professor, in the journal JAMA.
Kesselheim was one of three members of the FDA advisory panel who resigned last month after the agency brushed aside its recommendation in November that regulators reject Aduhelm. He has called the approval “perhaps the worst” drug authorization in the FDA’s recent history.
And, Blue Cross and Blue Shield of North Carolina referenced a June statement by a Boston drug-pricing watchdog group, the Institute for Clinical and Economic Review, which said Aduhelm merited an annual price of $2,500 to $8,300, given its modest benefits and the risks of side effects.
Even if a health insurer decides not to pay for a drug, customers can typically file an appeal and sometimes get it covered.
One of the most closely watched drugs in recent history, Aduhelm is a monoclonal antibody made from the immune cells of older people with no or uncommonly slow cognitive decline. It is administered in monthly intravenous infusions.
Aduhelm generated contradictory results in two late-stage clinical trials. In one study, Biogen said, a high dose could delay cognitive decline by 22 percent or about four months over 18 months, an encouraging result. The other trial failed to prove the drug was effective.
Critics have also noted that the drug caused side effects in some patients, including brain swelling, although they usually were not serious.
Aduhelm is the first new drug for Alzheimer’s approved since 2003. The FDA cleared it under an “accelerated approval pathway” reserved for medicines that fill a serious unmet need.
The FDA uses that approach when regulators are uncertain about the clinical benefits of a drug and rely on another measure to determine whether it might help patients. In this case, that was Aduhelm’s reduction of a sticky substance called amyloid that clumps into plaques in the brains of people with Alzheimer’s. That reduction, Biogen says, is “reasonably likely to predict” that the drug will benefit such patients. Some experts say that hasn’t been proven.
Last Friday, acting FDA commissioner Dr. Janet Woodcock asked the Department of Health and Human Services’ independent Office of the Inspector General to investigate the process that led to the drug’s approval. She cited interactions between Biogen representatives and drug regulators, first reported by the news site STAT, that “may have occurred outside of the formal correspondence process.”
Two powerful House Democrats are also leading a congressional investigation into Aduhelm’s approval.
Jonathan Saltzman can be reached at email@example.com.