Presented at the 31st International Symposium on ALS/MND, December 9, 2020.
Background: ALS drug development has been plagued by high clinical trial failure rates. Subgroup analysis is a key tool used to account for patient heterogeneity, but current methods fall short. DEC analysis systematically groups and analyzes patients based on predicted disease path, creating more homogeneous patient subgroups with reduced noise around the endpoint.
Conclusions: DEC analysis organizes clinical trial participants in an unbiased way into subgroups that are more homogeneous than the full trial population
- Method reveals “hot spots” of detectable treatment effects that could form the basis for a subsequent successful clinical trial
Numerous ways to implement this approach are envisioned:
- “Rescue” of drugs that failed late-stage clinical development
- Application to all-comers trials with the goal of identifying patients with detectable effects to seamlessly expand into a fully powered trial
Authors: Albert A. Taylor, Danielle Beaulieu, Jonavelle Cuerdo, Mark Schactman, Dustin Pierce, Mike Keymer, David L. Ennist