The proposal by the Centers for Medicare and Medicaid Services (CMS) to limit coverage for the Alzheimer’s drug Aduhelm to patients in clinical trials drew criticism as an unusual step for a drug granted accelerated approval from the FDA.
Biogen’s Aduhelm, the first Alzheimer’s drug in nearly 20 years approved by the FDA, would be only the 22nd treatment to have coverage restricted to clinical studies by the CMS if the proposal is finalized, Forbes reports.
The decision was “crushing news” for Alzheimer’s patients, according to Bloomberg. Around 6 million Americans, including 200,000 under the age of 65, have Alzheimer’s, and half of them “may have mild symptoms, the target group for Aduhelm.”
“Aduhelm is the first drug on the market able to remove amyloid, the sticky substance that builds up in the brains of Alzheimer’s patients,” according to NPR. While it is not supposed to be able to stop Alzheimer’s, it is believed the drug may slow cognitive decline associated with the advance of the disease, according to The Washington Post.
The CMS explained its decision to limit coverage of the Aduhelm in its National Coverage Determination (NCD) report on the drug: “None of the trials completed as of the publication of this NCA [National Coverage Analysis] has convincingly demonstrated that use of antiamyloid mAbs result in a meaningful improvement in health outcomes for AD [Alzheimer’s disease] patients.”
Aduhelm was granted accelerated approval by the U.S. Food and Drug Administration (FDA) last summer. Accelerated approval can be given to a drug “for a serious or life-threatening illness that may provide meaningful therapeutic benefit over existing treatments.” FDA uses this pathway to approve a drug when “it is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients and there remains some uncertainty about the drug’s clinical benefit.”
The possibility that the drug may slow cognitive decline is enough for the Alzheimer’s Association to cheer its approval. But some experts have questioned the untried nature of the drug, potential side effects, and its current high cost: $28,200 a year.
Patients would struggle to afford the drug
Given the cost, many patients would not be able to take the drug if the CMS finalizes its decision to limit coverage to clinical trials.
“Limiting coverage only to patients enrolled in government-mandated randomized clinical trials means that most of those suffering from Alzheimer’s would be denied access to an approved medicine simply because of where they live or where they go for care,” BIO President and CEO Dr. Michelle McMurry-Heath said in a statement.
She said the CMS is “pronouncing its disagreement with FDA’s gold-standard, science-based, safety and efficacy standards” and added: “The CMS proposal also would require post-approval randomized controlled trials for all future drugs in this class, regardless of the type and volume of evidence demonstrated during FDA-required trials and regardless of whether future drugs are approved during an accelerated pathway or through traditional channels.”
Given this stipulation in the proposal, “the biggest losers may actually be other drug makers readying treatments for the debilitating disease,” according to Stat. “The question is whether they encounter such difficulties and, if so, to what extent?”
Aduhelm maker Biogen argues that the policy criteria are too narrow and announced plans to make a formal response to CMS. In their official statement on the decision, Biogen underscored that CMS policy would delay Aduhelm treatment for many Medicare beneficiaries “potentially to the point where treatment may no longer be an option for them.”
After a month of hearings, the CMS said it will make a final decision in April on whether to cover the cost of Aduhelm for all eligible patients or only those in clinical trials.