Is Enrichment Beneficial for ALS Research? We Say “Yes!”
Earlier this year, the FDA released updated guidelines on the types and use of study population enrichment methods. They believe the use of enrichment can potentially provide increased efficiency in the clinical trial process. Our experts at Princeton Pharmatech have proven these benefits, at least in the development of Edaravone, a product approved in 2017 for treatment of amyotrophic lateral sclerosis (ALS).
Development of biologics for rare diseases, such as ALS, can be difficult when patients present with heterogeneous symptoms and progression rates. By enriching the study population with those patients most likely to demonstrate the effect of the drug, if there is one, you can more conclusively show the efficacy of your treatment. During the development of edaravone, we assisted our client in designing a randomized control trial with an enriched population using the results of our previous trial. Post-hoc analyses of the effects of this strategic study design resulted in a reduction of minimally progressing patients included in the population, establishing support for the detection of treatment effect as an effectual method of enrichment.
We are proud to announce our latest research paper published in the Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration journal, an open-access publication with the Taylor & Francis Group. If you are interested in reading more about our methods and results, check out the article for yourself!