Key points
Demographics of Registry participants were compared with enrollees of PRO-ACT database. Clinical trial participants were younger and had shorter disease duration. Race information was largely missing in the PRO-ACT database. Addressing the discrepancy of the demographic niche in the ALS population may increase the generalizability of study outcomes.
Affiliate
Moon Han, Jaime Raymond, Theodore C. Larson, Paul Mehta & D. Kevin Horton
1. Centers for Disease Control and Prevention/Agency for Toxic Substances and Disease Registry, Office of Innovation and Analytics, National ALS Registry (CDC/ATSDR), Atlanta, GA, USA
Summary
This study from the National ALS Registry aims to compare the participant demographics between the Registry and the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT). The primary objective of the study was to identify any potential demographic differences between patients being recruited for clinical trials in PRO-ACT compared to the Registry’s database, who are more likely to represent the epidemiological population of ALS patients. Common demographic characteristics were compared: age, sex, ethnicity, and race (when possible); alongside comparisons of ALS-symptom related variables: age of diagnosis, age of symptom onset, familial history of ALS, etc.
The study found that the median age of participants in the PRO-ACT database was younger than that of the Registry’s (57 vs 61). Race category differences were observed, with several race groups being underrepresented in the clinical trial database. A statistically significant portion of PRO-ACT recruits had missing or unknown race information compared to the Registry (31.9% vs 2.1%). Addressing the discrepancy of the demographic niche in the ALS population may increase the generalizability of study outcomes. Further efforts are needed to target understudied portions of the population.