The Food and Drug Administration (FDA) is the governing body of prescription medications in the United States. In order for a drug to gain FDA approval, it needs to demonstrate safety and efficacy. A thorough review process is conducted in order to assure this safety and efficacy. In June 2021, however, that process was questioned by the scientific community. In June of 2021, the FDA approved aducanumab (Aduhelm) for the treatment of Alzheimer’s disease. This is the first approval of a medication to treat Alzheimer’s disease since 2003. Although this medication was highly anticipated by the neurodegenerative disease community, the data was less than ideal. This medication is indicated in mild or moderate dementia or cognitive impairment. It utilizes a novel mechanism of action to aim to treat this devastating disease. Abucanumab is an amyloid beta-directed antibody.1
The recent FDA approval was based on data that suggests that aducanumab reduced the levels of cerebral amyloid plaques on PET scan.2 Many providers within the healthcare community are not convinced that amyloid plaques are characteristic of Alzheimer’s disease. The FDA granted approval based on the conclusion that the reduction of amyloid plaques is reasonably likely to cause some disease improvement. Amyloid plaques are not a validated endpoint in determining effects from Alzheimer’s disease. There have also been theories that decreases in the amyloid plaque is due to advanced cerebral disease, indicating more severe disease.2 The FDA advisory committee recommended against the approval of aducanumab on the basis of insufficient evidence. The FDA required the product’s manufacturer, Biogen, conduct more long term studies over the next 9 year in order to determine long-term clinical benefit. If these trials do not meet acceptable endpoints, the FDA could withdraw approval.3
On top of the lack of efficacy data, aducanumab is extremely expensive. It is estimated to cost over $56,000 per year to treat one patient. Being that most patients with dementia and Alzheimer’s are enrolled in Medicare services, the decision on whether CMS will cover the medication or not is a very important one. It is estimated that if Aduhelm is added to the CMS formulary, it will add billions of dollars to the federal government cost. Usually, FDA approval provides sufficient evidence to guarantee the safety and efficacy for CMS approval. Given the high critique of this medication, that is not enough. CMS would leave this coverage to the state Medicaid programs if they deny coverage of Aduhelm, which will drain funding. For legal reasons, Medicaid must cover most FDA approved medications. There are discussions on how to limit patients’ access to this medication. It has not been shown, concretely, to be beneficial. While there are still discussions on how to navigate this situation, there is also coverage for reform in the healthcare system. There are calls for changes in the laws that require CMS to cover medications if the states cannot afford to cover a medication. Especially with a medication that has no concrete benefit and a high cost, we are seeing how detrimental this could be on the healthcare systems.3
Aduhelm. Package insert. Biogen; 2021.
Høilund-Carlsen PF, Alavi A. Aducanumab (Marketed as Aduhelm) Approval Is Likely Based on Misinterpretation of PET Imaging Data. J Alzheimers Dis. 2021 Oct 10.
Sachs R, Bagley, J. Medicare Coverage of Aducanumab — Implications for State Budgets. NEJM. 2021 Nov 20.