Presented at the 27th International Symposium on Amyotrophic Lateral Sclerosis and Motor Neurone Disease, Dublin Ireland, December 2016.
Objectives: We asked, for what types of ALS human clinical trials can concurrent controls, historical controls, and virtual controls be most efficient.
- Historically Controlled Trials (HCTs) are an efficient design when large treatment effects are expected, e.g., >50% improvement
- The threshold at which HCTs are more efficient than Concurrently Controlled Trials (CCTs) will change as improved prediction of outcomes reduces unexplained trial-to-trial variation
- Use of HCTs will also be aided by accumulation of additional historical control data as secular trends in ALS care may bias older trials
- Historically controlled trials are an appropriate design when studying intensive treatments that would require a large treatment effect to be practical
Authors: David A Schoenfeld, Robert Küffner, Eric A Macklin, David L Ennist, Dan H Moore, Neta Zach, Nazem Atassi