N-of-1 Trial Designs in Alzheimer's Disease

What is it about?

For the past 2 decades, nearly every clinical trial in Alzheimer's disease has failed, yet subgroup analyses suggest that certain individuals do respond. An alternative approach to clinical trial design, the N-of-1 design, may help fast track medications through the testing process and identify within subject response to study medications.

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Why is it important?

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. With the stated goal of finding a cure for AD by the year 2025, if we continue on the same path of 18–24 month RCTs, there are simply not enough agents or enough time to reach this target. Instead, if we attempted N-of-1 trials in AD, we may very well have the possibility of offering patients and their families a selection of newly approved therapies and giving them new hope well before the target date.

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