In the early stages of Alzheimer's disease (AD), treatment effects on the core areas of cognition and function can be small initially. Due to this, researchers have proposed looking more holistically at the benefits of disease-modifying therapies (DMTs) through a framework they are calling "meaningful benefits".
Meaningful benefits covers three dimensions:
Multidimensional outcomes assessing not only cognition and function, but also patient- and caregiver-reported outcomes, health and economic outcomes, and neuropsychiatric symptoms
Complementary analyses that contextualize and expand the understanding of the outcomes, such as number-needed-to-treat or time-to-event analyses
Assessment of both cumulative benefit and predictive benefit, where early changes on cognitive, functional, or biomarker assessments predict longer-term clinical benefit
The concept of meaningful benefits emphasizes the importance of multidimensional reporting, while advancing our understanding of treatment effects in preclinical AD and mild cognitive impairment due to AD. The authors propose that such an approach will help bridge the gap between the emergence of DMTs and their clinical use.
You can review the entirety of their proposal at BioMedCentral.com.