Inside the tactical tug of war over the controversial Alzheimer’s drug

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The drug industry, patient advocates, and congressional Republicans have all attacked federal officials’ decision to decline routine Medicare coverage for a controversial Alzheimer’s drug. They’ve gone as far as to accuse them of tacit racism, ageism, and discrimination against the disabled — and hinted at a lawsuit — over the decision to pay only for patients taking the drug in a clinical trial.

The drug, Aduhelm, with a listed price tag of $28,200 a year, has had few takers in the medical world. Brain doctors are leery of administering the intravenous drug because it appears dangerous and largely ineffective. Many of the nation’s most prestigious hospitals — such as the Cleveland Clinic, Johns Hopkins Hospital, and Massachusetts General in Boston — have declined to offer it to patients.

While groups representing the pharmaceutical industry and patients press to undo Medicare’s decision, industry critics applaud the Centers for Medicare & Medicaid Services for throwing obstacles in the way of a drug they think the FDA should never have approved in the first place.

For the industry, the campaign has a broader existential target: to prevent CMS from using its payment decisions to keep FDA-approved drugs off the market. In recent years, FDA programs to speed approval of new drugs have led to a rash of entries with often minimal scientifically sound evidence to prove they work, critics say.

The FDA’s own expert panel recommended against approving Aduhelm for that reason. Last June, the agency approved it anyway.

CMS then announced Medicare would pay only when the drug was used in further clinical trials to assess its true benefit. That Jan. 11 announcement has drawn more than 9,000 comments to the agency’s website — a tsunami compared with most approval decisions. The remarks are roughly divided among pros and cons, and many appear to be organized by groups on the pro side of the debate (such as the Alzheimer’s Association) or those opposed (such as the nonprofit More Perfect Union). The agency could change or even reverse its decision, though experts believe the latter is unlikely.

“If the FDA were doing its job, CMS wouldn’t have had to step in. But good for the CMS, they are helping to protect the public from drugs whose harms outweigh benefits,” said Dr. Adriane Fugh-Berman, a Georgetown University professor of pharmacology who directs PharmedOut, a group that publicizes what it sees as poor industry practices.

Aduhelm is the first FDA approval for a class of laboratory-made antibodies designed to clear away so-called amyloid plaques, which gradually accumulate in the brains of people with Alzheimer’s disease.

In clinical trials, Aduhelm did well dissolving the plaques, but its impact on the functioning of patients in earlier stages of Alzheimer’s was so meager that an expert panel voted 10-0 (an 11th panelist was uncertain) in November 2020 to advise FDA to reject it. The science is unclear about whether the presence of such plaques — a so-called surrogate marker — correlates with the mental functioning of patients.

As such, the FDA gave “provisional approval” to Biogen, the maker of Aduhelm, allowing it nine years to provide evidence that the drug slows the progression of Alzheimer’s. In that period, Biogen would make far more money than if the application had been rejected. Even under the CMS decision, it would reap Medicare payments from whatever is used in clinical trials, which would need to include thousands of participants to assess the drug’s performance.

Drug companies and pharma investors have responded to CMS’ ruling with special alarm because they have spent decades improving their relationships with the FDA, only to have CMS seemingly pull the rug out by exerting its own power over an expensive drug.

“The drug companies are worried that this could be a precedent for other drugs. And it should be,” Fugh-Berman said. “This isn’t just about money; it’s about protecting the public.”

This “accelerated approval” employed for Aduhelm got its start in 1992 and is aimed at moving promising new classes of drugs to the public faster. Companies whose drugs go through the process — more than 250 drugs or vaccines have been approved so far — are supposed to quickly gather evidence that the products likely improve health once they’re on the market. But such follow-up studies often lag or are never performed. For example, the makers of the Duchenne muscular dystrophy drug eteplirsen, approved in 2016, didn’t start recruiting patients into a post-marketing trial until 2020 and don’t expect results until 2026.

Biogen originally said it would get confirmatory results for Aduhelm within seven years of approval. The company later trimmed that to four years. It also hinted that it might sue CMS, calling its decision “arbitrary and capricious.”

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