Unmet Need and Science Warrant Approval of Aducanumab

Editor’s note: With all the recent news on the FDA’s approval of Aduhelm (aducanumab), the new drug used to treat Alzheimer’s, ASA wanted to give our readers informed opinions on the topic from experts in the aging sector. Below is a piece arguing in favor of the FDA approval of Aduhelm. Click here to read the piece arguing against the FDA approval.

The opinions expressed in this article are those of its author. They do not necessarily reflect the opinions or views of ASA management or its members.

Let me start with the most essential point first. The Alzheimer’s Association fully supports the Food and Drug Administration’s (FDA) recent accelerated approval of Aduhelm (aducanumab).

As the world’s largest nonprofit funder of Alzheimer’s disease research and a relentless advocate for more than 6 million Americans living with the disease and their caregivers—we believe the science, combined with the unmet need, warranted accelerated approval. And because the approved therapy—the first for Alzheimer’s disease in nearly two decades—may help some people living with early-stage Alzheimer’s and mild cognitive impairment.

I offer this critical takeaway upfront to ASA members and stakeholders because I don’t want it lost amid headlines surrounding the drug’s approval.

We Support the Drug for What it Is and What it Can Be

The Alzheimer’s Association supports the FDA’s accelerated approval of Aduhelm for what it is—the first treatment for Alzheimer’s disease since 2003 and the first to address the underlying biology of Alzheimer’s, in people with Mild Cognitive Impairment (due to Alzheimer’s) and early-Alzheimer’s dementia. And for what it can be—a pivotal milestone which begins a new era in Alzheimer’s treatment and research where the same progress we have witnessed in combating many other major diseases may finally develop for the millions now facing Alzheimer’s, a terminal disease.

While not a cure, this new drug is the first FDA-approved treatment to reduce one of the defining brain changes of Alzheimer’s disease—the clumping of amyloid plaques in the brain—that disrupts brain function. According to the FDA, this reduction in amyloid plaques is reasonably likely to lead to a reduction in the clinical decline due to Alzheimer’s disease.

This could mean more time for individuals to actively participate in daily life, have sustained independence and hold on to memories longer. For individuals living with Alzheimer’s and their families these simple daily activities—that many of us take for granted—mean everything.

We recognize the drug may work differently for everyone, and may not work for some individuals. The Alzheimer’s Association has been consistent in its position that Aduhelm be made available specifically to the population represented in the clinical trials—people with mild cognitive impairment (MCI) due to Alzheimer’s disease and early Alzheimer’s dementia. Experts from the Alzheimer’s and scientific community expressed widespread agreement on this important point during a recent scientific meeting convened by the Alzheimer’s Association to discuss steps forward in wake of the FDA’s approval, attracting more than 2,700 registrants from 90 countries.

For millions more living with Alzheimer’s who fall outside this category, Alzheimer’s and dementia researchers must leverage this important milestone, using it as a springboard to accelerate new advancements. With more than 100 therapies in the current Alzheimer’s pipeline—exploring multiple pathways for combating the disease—the opportunity for new and potentially better treatments has never been more promising. This is critical because there is consensus in the Alzheimer’s research community that slowing and stopping the disease may not come from a single therapy, but rather by combining proven treatments into powerful combination therapies that have worked well in combating other disease conditions.

Without a First Approval, There Can't Be a Second or Third

History has shown that approvals of the first drug in a new category will invigorate research, increase investments in new treatments and generate greater innovation. We have seen it happen for heart disease, cancer and HIV, where the first treatment for these disease conditions spurred additional treatments and new advancements that have benefited patients. We must do the same for Alzheimer’s.

However, the promise and potential of this new treatment—or any future treatment—will never be fully realized unless it is affordable and accessible for all who could benefit. At a cost of $56,000 per year, the current price is unacceptable. This price poses an insurmountable barrier to access for too many families impacted by Alzheimer’s. It complicates and jeopardizes sustainable access to this treatment, and may further deepen issues of health equity. The Alzheimer’s Association has called on drugmaker Biogen to change its price.

Families impacted by Alzheimer’s already face significant challenges, accessing treatments that could help them should not be another. Eliminating barriers to access is the Alzheimer’s Association highest priority and we will do everything in our power to ensure access to the drug, any tests needed during the treatment process and other associated costs for all who will benefit. We’re committed to working with the Centers for Medicare & Medicaid Services (CMS)—and with the private payer community—to remove barriers to coverage for those who would benefit from this treatment.

The Alzheimer’s Association and many others in the scientific community see the approval of Aduhelm as an important step forward. Without the first approved treatment, there cannot be the second or third or fourth, each improving on earlier treatments. There is much more work to do in our fight to end Alzheimer’s and all other dementias and the Alzheimer’s Association remains steadfast in our work.

Kristen Clifford, MBA, is the chief program officer at the Chicago-based Alzheimer’s Association and lives in Austin, Texas.