What was your consortium's most significant achievement from the past year?
Michael Benatar, MD, PhD, Principal Investigator, CReATe: Changing the dialogue around the best approach to early/mid-stage ALS clinical trials.
What did this achievement accomplish?
Dr. Benatar: Historically, early and mid-stage ALS clinical trials have relied too heavily on underpowered clinical outcome measures. Through the consortium’s work to clinically validate biomarkers (e.g. neurofilament light chain) for defined contexts of use (e.g. prognostic and response), encourage a focus on biological (rather than clinical) measures of response, highlight the problems of multiplicity, and encourage better go/no-go decision making at the end of phase 2, CReATe investigators are slowly shifting the community’s approach to early drug development.
How will this impact the rare disease community?
Dr. Benatar: The impact should be more efficient clinical trials that are more likely to be informative and more likely to correctly identify promising therapeutics.
How will this advance the goals of your consortium and/or the RDCRN at large?
Dr. Benatar: Experience of the ALS clinical trials field is directly relevant to the efforts of other rare disease communities to identify and develop therapeutics that deliver meaningful benefits to patients.