US clinical trials lack diverse representation of ALS patients
Study finds women and non-white patients are underrepresented
- U.S. ALS clinical trials lack diverse representation.
- Women and non-white patients are significantly underrepresented.
- Broader enrollment is crucial for generalizable findings and drug development.
In the U.S., clinical trials for amyotrophic lateral sclerosis (ALS) do not include enough non-white patients and women to fully represent the disease’s landscape in the real world, a study found.
This lack of representation of certain groups may prevent researchers from generalizing clinical trial findings to the overall ALS population.
These findings provide “a starting point” to develop “effective approaches to broaden enrollment across all groups of people with ALS,” researchers wrote in “Participation in U.S.-Based ALS Clinical Trials by Sex and Race,” which was published in Muscle & Nerve.
Racial, ethnic disparities appear to exist in global ALS trials
ALS damages the nerve cells in control of voluntary movement, causing muscle weakness that worsens over time. Symptoms vary from patient to patient, making diagnosis and treatment challenging. To develop strategies that work well for everyone, clinical trials should include a diverse patient population that reflects the disease’s real-world prevalence.
In global ALS trials, disparities appear to exist by race and ethnicity, but it remains unknown whether specific populations are also underrepresented in ALS clinical trials in the U.S.
To learn more, researchers examined data from 11 recent Phase 2 and Phase 2/3 clinical trials involving a total of 1,153 patients. Fourof those trials ran as part of the HEALEY ALS platform trial.
They then compared these data with the National ALS Registry and with a surveillance project that covered three states and eight metropolitan areas between 2009 and 2011 to calculate the participation-to-prevalence ratio (PPR). A PPR of less than 0.8 indicates underrepresentation in a trial, while a PPR of more than 1.2 indicates overrepresentation.
Of the 1,153 patients, 424 (36.8%) were women. The trials that reported race included 1,122 patients. Twenty (1.8%) were Asian, 26 (2.3%) were Black, and 1,057 (94.2%) were white. No patients were Native American or Hawaiian/Pacific Islander. Four (0.4%) were of other or mixed races, and 15 (1.3%) did not report race.
Results showed that women had a PPR of 0.76, meaning fewer women joined clinical trials than expected based on how many women are estimated to have ALS in the U.S. Indeed, the proportion of women in the National ALS Registry (46%) or the surveillance project (44%) was higher than in the clinical trials analyzed (36.8%).
Racial disparities did not change over the years
In terms of race, white patients were overrepresented, with a PPR of 1.26 compared with National ALS Registry data, while Black patients had a low PPR of 0.34. Patients of other or mixed races, including Asian, were also underrepresented, with a PPR of 0.35.
Hispanic ethnicity was not reported in the National ALS Registry, but it was underrepresented based on the surveillance data, with a PPR of 0.44.
These racial disparities did not change over the years, showing that there has been no meaningful improvement in the participation of different groups. These disparities were also present in the four largest trials, indicating that greater participation does not ensure a better racial representation of the real-world population.
The findings overall show that ALS clinical trials still do not reflect the full diversity of the disease, with non-white patients and women particularly underrepresented.
However, “racial diversity in trials is important because it may improve generalizability and the trial’s ability to predict safety in clinical practice,” the researchers wrote. A broad patient representation can also boost patient enrollment and accelerate drug development, they added.
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