Comparison of Demographics: National Amyotrophic Lateral Sclerosis Registry and Clinical Trials Data

TL;DR

This study compares the demographics of participants in an ALS registry and clinical trials data to identify differences in age and diversity.

Contribution

The study highlights demographic disparities in ALS research populations to improve patient inclusion and study generalizability.

Findings

PRO-ACT had more younger participants and fewer older ones compared to the Registry.

The Registry had higher minority representation, while PRO-ACT had significant missing race data.

Registry participants had a longer delay between symptom onset and diagnosis compared to PRO-ACT participants.

Abstract

To characterize the participant demographics in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database compared with the web-portal National Amyotrophic Lateral Sclerosis (ALS) Registry (the Registry).

Demographics and ALS symptom information were compared between the self-reported registrant data in the Registry web portal (2010–2021) and the latest available PRO-ACT data (updated August 2022), which is a collection of clinical trials data.

Greater percentages of younger (≤ 59 years old) but smaller percentages of older (60 + years old) participants were represented in PRO-ACT compared to Registry. Enrollment for minority race groups was greater in the Registry portal data, but race information was largely missing/unknown in PRO-ACT database. Median age at the time of diagnosis and age at the time of symptom onset were significantly higher for Registry enrollees compared to the participants of PRO-ACT. Symptom onset sites were similarly reported, but duration between self-noted symptom onset and diagnosis was slight, but significantly longer for the Registry enrollees (11 vs. 9 months). Hispanic were as likely as non-Hispanic to participate in research studies, based on the Registry data.

There was a notable difference in the age distribution and minority representation of enrollees between the PRO-ACT and Registry study populations. Age distribution in the PRO-ACT database skewed to a younger and less diverse cohort. Despite the clinical heterogeneity and complex disease mechanism of ALS, identifying the underrepresented demographic niche in the PRO-ACT and Registry study populations can help improve patient participation and criteria for patient selection to enhance generalizability.

The online version contains supplementary material available at 10.1007/s40615-024-02047-4.