Medicare will cover the controversial Alzheimer's drug Aduhelm only if a patient is enrolled in a clinical trial, the Centers for Medicare & Medicaid Services (CMS) proposed Tuesday.
The proposed decision means that CMS would only cover Aduhelm and similar types of drugs if the trial were approved by the agency and conducted in a hospital outpatient setting.
Aduhelm, manufactured by Biogen, is intended for patients with early-stage Alzheimer's disease.
The drug has drawn controversy for its price and because the Food and Drug Administration (FDA) approved it despite doubts from experts about its effectiveness and safety concerns. Medicare coverage is based only on whether the drug is "reasonable and necessary" and does not take price into consideration.
The CMS review process found that while “there may be the potential for promise with this treatment, there is also the potential for harm to patients. This harm may range from headaches, dizziness, and falls, to other potentially serious complications such as brain bleeds,” CMS Chief Medical Officer Lee Fleisher said in a statement.
The treatment, which is delivered intravenously, is intended to reduce the amount of plaque in the brains of Alzheimer's patients.
Aduhelm is not a cure for Alzheimer's, and the FDA made its decision without clear evidence that the drug can even slow the progression of the disease. Instead, the agency based its approval on how well it reduces brain plaque, which is thought to contribute to Alzheimer's disease, but has not been definitively proven.
FDA granted accelerated approval for the drug on June 7. Aduhelm was the first new Alzheimer's drug approved in nearly 20 years, but the approval came over the near-unanimous objections of the FDA's expert advisory committee. Three of the agency's outside advisers resigned over the decision.
In the months since, FDA and Biogen have narrowed the original approval, a government watchdog and two House committees have opened investigations into FDA's approval process, and Biogen slashed the price amid fears of a massive Medicare premium spike.
Amid lagging sales, Biogen halved the price of the drug. Aduhelm will cost $28,200 per year for an average-weight patient, down from the $56,000 a year when the drug first launched last summer. Sales of the drug yielded only $300,000 for Biogen between July and September after forecasts had predicted revenue to reach $12 million.
The decision announced Tuesday is not final and is open for comment for the next 30 days. A final policy is expected in April, which could be significantly different.
In a statement, Biogen expressed disappointment in the decision. The company said restricting coverage only to clinical trials will severely limit access to the patients who need it the most.
“This draft coverage determination denies the daily burden of people living with Alzheimer’s disease. Coverage with evidence development (CED) under a randomized clinical trial will exclude almost all patients who may benefit," the company said. "This will significantly limit patient access to an FDA-approved treatment, especially for underserved patients as evidenced in other CED determinations."
About 6.2 million people in the United States have Alzheimer’s disease, according to the Alzheimer’s Association. But the company has estimated only about 1.5 million people will be eligible.
Updated at 6:19 p.m.