Gaining a Better Understanding of Clinical Benefit in Alzheimer Disease: Ronald C. Petersen, MD, PhD


The director of the Alzheimer’s Disease Research Center at Mayo Clinic discussed a recently published paper focused on the expectations and clinical meaningfulness of randomized controlled trials in Alzheimer disease. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

"We’re trying to put the clinical change, the clinical response of these drugs into a perspective that is reasonable for clinicians, patients, families, public stakeholders to say ‘Yeah, that makes sense. I see the point you’re trying to make. And it makes sense.’"

There has been a considerable buzz among clinicians in the Alzheimer disease (AD) field as conversations of new treatments and potentially disease-altering approaches enter the fold. After not having any approved therapies since 2003, the toolbox began to expand with the approval of aducanumab (Aduhelm; Biogen) in June 2021, and most recently with lecanemab (Leqembi; Eisai) in January 2023. Both antiamyloid therapies, these options are only a part of the solution to treating AD, a complex multifactorial disease.

While the approvals of these agents signified progress for the community, questions remained regarding the process of demonstrating clinically meaningful benefit and its true definition. With new clinical trial data expected soon on emerging therapeutics, the Alzheimer’s Association convened a work group of experts to highlight key considerations for interpreting data from cognitive and functional measures. In the recently published paper, the group stressed the importance of implementing treatment strategies early in the disease, as well as to ass change over the course of a relatively brief 18-month trial, to better understand the value of an effective intervention.

Following the publication, lead investigator Ronald C. Petersen, MD, PhD, sat down to discuss the motives behind the paper, and why this is such a critical topic of concern. Petersen, director of the Alzheimer’s Disease Research Center at Mayo Clinic, also provided perspective on new, adaptive trials, the need to incorporate biomarkers, and how clinicians should handle observing early, significant treatment effects.

1. Peterson RC, Aisen PS, Andrews JC, et al. Expectations and clinical meaningfulness of randomized controlled trials. Alzheimers Dement. 2023;1-7. doi:10.1002/alz.12959
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