Scientists in NYC aim to increase diversity in CF clinical trials
Fewer than 1 in 4 minority patients seen to take part in trials
Scientists in New York are calling on the cystic fibrosis (CF) research community to take proactive steps to increase diversity in clinical trials — which now typically include a disproportionate number of non-Hispanic white people, and fewer patients from other racial and ethnic backgrounds.
“Ensuring that pwCF [people with CF] from diverse backgrounds have access to and participate in clinical trials is key to advancing health equity,” the team wrote in a report, “Center-level self-study identifies opportunities to advance equity in cystic fibrosis clinical trial participation,” published in the Journal of Cystic Fibrosis.
For their study, the researchers looked at participation by patients from a “minoritized racial or ethnic group” at their own center in New York City.
“Efforts to improve the racial and ethnic diversity of [people with] CF in clinical trials, both onsite and offsite, will require a shift in how recruitment opportunities are identified and communicated to [people with] CF, particularly those historically underrepresented in clinical trials,” the team wrote.
Researchers looked at diversity in clinical trials at their own center
Clinical trials are rigorously designed studies used to collect data about CF and test potential treatments. For trials to provide the most meaningful data possible, the participants in them need to be representative of the broader population of people living with CF.
Now, a team of scientists in New York City conducted an evaluation at their own center to see whether individuals participating in trials were representative of the total group of patients followed at the center.
The analysis included data on 200 people with CF seen at the center between 2016 and 2020. Among them, 145 identified as non-Hispanic white people, while the other 55 identified as being of other racial or ethnic backgrounds, mostly as Hispanic and Black individuals.
Participation was assessed for 24 clinical trials conducted at the center — 14 studies testing new therapies, and 10 observational studies collecting data on patients or expanded access programs to CFTR modulators. These are a medication used to treat the genetic disorder.
All of the trials enrolled adolescents and/or adults; none were open to children younger than age 12. The researchers noted that, among the 48 children younger than 12 who were included in this analysis, the proportion of patients from racial or ethnic minorities was higher than in the overall group (64.6% vs. 27.5%). Also, more than half of all minority patients were children younger than 12.
Additionally, trials of medications were limited to patients carrying at least one copy of F508del, the most common CF-causing mutation, which is generally more common in white populations. Among the 200 patients in this analysis, 73.1% of those who identified as non-Hispanic white individuals were positive for F508del, as compared with 60% of those with other racial/ethnic backgrounds.
The results showed that slightly more than a third of non-Hispanic white patients (35.9%) had participated in one or more of the two dozen clinical trials at the center. By comparison, less than 1 in 4 patients from other backgrounds — specifically, 21.8% — had participated in a trial.
“Through our center-level self-study, we found an underrepresentation of [people with] CF identifying as part of a minoritized racial or ethnic group participating in onsite clinical and pharmaceutical clinical trials,” the researchers wrote.
CF centers urged to add metrics to improve clinical trial diversity
The team noted that, among non-Hispanic white patients, more than half (63.4%) were 12 and older and carried at least one copy of F508del — the group most likely to be eligible for these trials. By comparison, only 11 of the 55 patients from minority backgrounds (20%) met these criteria.
Considering only this subset of patients, the proportion who participated in trials was actually somewhat higher among the 11 who were not non-Hispanic white individuals.
“When the study population was limited to the [people with] CF most likely to be eligible for a CF pharmaceutical clinical trial … we found an overrepresentation of [patients] identifying as part of a minoritized racial or ethnic group participating in onsite pharmaceutical clinical trials,” the researchers wrote.
“While it is tempting to attribute this finding to the concerted effort of our research staff to engage [patients] from diverse backgrounds in research, there are other influencing factors,” they added, stressing that the small number of likely eligible patients makes it hard to make reliable assessments.
In addition to the 24 trials conducted at the clinic, records also indicated that seven patients had participated in clinical trials at other sites. While there are standardized procedures at the clinic to assess eligibility in interest in trials there, the researchers realized while doing this study that they did not have a similar system for trials at other locations.
We encourage CF centers to add clinical trial participation metrics to their quality improvement efforts, evaluating for concordance with center patient diversity and assessing implemented recruitment strategies.
“In undertaking a center-level self-study to explore the concordance of racial and ethnic diversity in clinical trials with our overall patient population, we reflected on our recruitment and referral processes,” the researchers wrote.
“For offsite clinical trials, it became evident that there is no standardized referral process at our CF Center; many of these opportunities either filled specific diagnostic voids or were brought to our attention by the [people with] CF themselves,” the team wrote, adding that they are “in the process of creating a more strategic approach to offsite clinical trial referrals.”
All seven patients who had participated in off-site trials were non-Hispanic white, which the researchers noted as a concerning discrepancy.
“We encourage CF centers to add clinical trial participation metrics to their quality improvement efforts, evaluating for concordance with center patient diversity and assessing implemented recruitment strategies,” the researchers concluding, noting, “Successful approaches may then be shared across CF centers to ensure equal opportunity and participation in clinical trials.”
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