Abstract
There has not been a new treatment for Alzheimer's disease (AD) for over a decade, with a large number of Phase II/III randomized clinical trials failing. Randomized clinical trials examine group effects that may be difficult to extrapolate to the individual patient given the multifactorial pathogenic processes associated with AD, and are increasingly long in duration, expensive to run, requiring large sample sizes that are difficult to recruit. An alternative approach is to consider N-of-1 trial designs. The N-of-1 trial is ideal to evaluate effectiveness of interventions for chronic conditions combining the rigor of a randomized trial with the tailoring of therapy to an individual. This review examines the N-of-1 design, its benefits and limitations, and how it could be implemented to investigate new therapies for AD.
Study funding
This study was supported by grants from the National Institute on Aging (R01 AG0402-11-A1) and the Harry T Mangurian Foundation.
Financial disclosure & competing interests disclosure
The author has received funding from NIH grant R01 AG040211A1. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
No writing assistance was utilized in the production of this manuscript.