Krista Solano

Associate Editor

Loyola University Chicago School of Law, JD 2023

For the first time in approximately twenty years, the U.S. Food and Drug Administration (FDA) approved a drug to combat the progression of Alzheimer’s. The newly approved drug is manufactured by Biogen and will be called Aduhelm. The FDA granted fast track designation of the drug to speed up access to patients. While Aduhelm will not reverse already developed Alzheimer’s symptoms, it will slow down the advancement of the disease by removing deposits of beta-amyloid, a protein found in early-stage Alzheimer’s patient’s brains.

It remains unclear who will bear the burden of Aduhelm’s hefty price tag, as the Centers for Medicare and Medicaid Services (CMS) and many insurance carriers have not made a final decision. Even for patients willing to pay for Aduhelm out-of-pocket, some hospitals refuse to administer the drug.

Alzheimer’s background

According to the FDA, Alzheimer’s is the sixth-leading cause of death in the United States and currently affects 6.2 million Americans. The devastating illness progressively destroys the brain’s memory and thinking skills and eventually prohibits patients from completing daily tasks. Alzheimer’s not only affects patients’ lives but their loved ones as well, as they often become the caretakers to those that fall ill to the disease.

The exact cause of Alzheimer’s disease is unknown, but many scientists believe genetics, lifestyle, and environmental factors cause the debilitating disease. On the foundational level, essential brain proteins fail to function normally, resulting in the damage and death of neurons. Research is focused on the role of two proteins: beta-amyloid and tau. Tau proteins play an essential part in a neuron’s internal support and transportation of materials. During Alzheimer’s disease, tau proteins organize themselves in neurofibrillary tangles that are deadly to cells. Beta-amyloid buildup, also called amyloid plaque, has a toxic effect on neurons. Some scientists believe the accumulation of beta-amyloid is the primary cause of Alzheimer’s, otherwise known as “the amyloid hypothesis.”

What is fast track designation?

Fast track designation is the expedited approval process utilized by the FDA for drugs used to combat serious conditions or to fill an unsatisfied medical demand. Alzheimer’s, cancer, and AIDS are just a few of the very serious medical conditions that the FDA considers when evaluating whether to use fast track designation on approval. Fast track designation does not mean that there is less research conducted, but rather, can allow drug companies access to more frequent meetings and communication with the FDA and can allow for eligibility for accelerated approval, priority review, or rolling review. Drug companies must request fast track designation to be considered under the expedited process, for which the FDA makes a decision on approval within sixty days.

How does Aduhelm work?

While Aduhelm is not a cure for Alzheimer’s, the newly approved drug seeks to slow down the progression of the disease by reducing amyloid plaques in the brain. Amyloid is a protein often found in Alzheimer’s patients’ brains that blocks cell pathways, leading to cell death. The monthly intravenous infusion of Aduhelm will work by reducing the amyloid plaque buildup in the brain. The reduction in amyloid plaque buildup was seen in participants by using positron emission tomography (PET) imaging.

The FDA has narrowed its recommendation for possible patients to only those in the early stages of the disease. Patients with significant issues of memory loss and thinking will not receive an Aduhelm recommendation because there is no beneficial research to support administration.

Who will bear the cost of Aduhelm?

Of the 6.2 million Americans suffering from Alzheimer’s disease, it is estimated that almost 90% of patients are covered by Medicare. Biogen announced that the drug’s list price will be $56,000 a year, with additional bills in the thousands for diagnostic testing and brain imaging. Federal officials have still not decided whether Medicare will cover the steep cost, forcing many clinics to be hesitant in prescribing Aduhelm.

In late July, the Centers for Medicare and Medicaid Services began a nine-month payment review of Aduhelm to determine whether the drug is a “reasonable and necessary” treatment for Alzheimer’s. If categorized as so, Medicare is legally obligated to cover the cost of the drug but can still place restrictions on patient criteria. The initial six months of the payment review include staff review of the research, evaluating the benefits of the proposed drug, external technology assessments, and drafting the decision memorandum. The following three months of the process allow thirty days for public comments on the proposed decision memorandum. Within sixty days of the public comment phase, the final decision memorandum and implementation instruction must be completed.

Medicare’s lack of coverage decision has led to some hesitancy or outright denial of coverage by insurance carriers. The Department of Veterans Affairs (VA) and many regional Blue Cross Blue Shield companies have omitted Aduhelm from the list of readily available treatments. As the most expansive integrated healthcare system in the United States, the VA covers more than nine million veterans, for which 150,000 are diagnosed with Alzheimer’s. If covered, Aduhelm is estimated to cost the VA more than $4 billion annually.

Who will administer Aduhelm?

Two of the most prominent hospitals in the US have said they will not administer Aduhelm. Cleveland Clinic and Mount Sinai of New York have announced Aduhelm infusion will not occur at these hospitals, but doctors can prescribe the drug to patients to be administered elsewhere. This has not only led to patient frustration but indicates health system distrust in the FDA. Patients are agitated with the administration logistics since the disease is severely progressive and administration is only recommended to early-stage sufferers. As such, the future of Aduhelm is uncertain, but patients and their loved ones will be eagerly awaiting its fate.