Hispanic Population Appears Underrepresented in Pediatric Cancer Trials

“Fewer Hispanic participants were enrolled in the trials we reviewed compared with the representation of Hispanic patients in the National Childhood Cancer Registry [NCCR]; however, this discrepancy may be partially explained by the large proportion of participants with unknown information regarding ethnicity,” according to the study authors.

Although representation of racial and ethnic minority groups in pediatric cancer trials appear to be consistent with the racial distribution of childhood cancer across the United States, Hispanic patients seem to be underrepresented with respect to enrollment, according to findings from a retrospective study published in JAMA Oncology.¹

Twenty-two pediatric exclusivity requests were written for 40 studies that included 2025 patients. Across the trials, the most represented racial group was White patients (64.3%) followed by Black patients (11.3%), those of other races (9.6%), patients with unknown data (8.0%), Asian patients (4.6%), Native Hawaiian or other Pacific Islander patients (1.6%), and American Indian or Alaska Native patients (0.6%). Additionally, most patients were male (58.8%).

With respect to ethnicity, the most represented groups included those with unknown data (69.1%), non-Hispanic patients (22.5%), Hispanic patients (5.7%), and those of other ethnicities (2.8%). Most studies were phase 2 trials (48%) followed by phase 1 (35%), phase 1.5 (13%), and phase 3 (5%).

“Fewer Hispanic participants were enrolled in the trials we reviewed compared with the representation of Hispanic patients in the National Childhood Cancer Registry [NCCR]; however, this discrepancy may be partially explained by the large proportion of participants with unknown information regarding ethnicity,” the study authors wrote. “The reasons for the high degree of missing ethnicity information are unclear and should be studied.”

Investigators used the FDA’s Document Archiving Reporting and Regulatory Tracking System to assess exclusivity request submissions for oncologic drugs receiving pediatric exclusivity from 2001 to 2021. Investigators acquired study location information from the ClinicalTrials.gov database and evaluated complete study reports that accompanied exclusivity request submissions.

Investigators analyzed data from 2022 to 2023. The main outcomes included representation in pediatric clinical trials by race, sex, and ethnicity that were initiated in response to written requests from the FDA.

“The FDA must consider study participant representativeness during the review of a request for pediatric exclusivity, but this measure may have limited effectiveness given the retrospective nature of this assessment,” the study authors wrote.

“Implementing requirements for study participant representativeness may be most effective when the [written request] is issued but can be challenging, due in part to variable interpretation of what constitutes study population representativeness. In most cases, as shown in our analysis, the initial study conducted within a [written request] in oncology is a small, dose-finding, safety, and activity–estimating study, and subsequent studies may also enroll small numbers of patients due to the rarity of pediatric cancers.”

According to the investigators, the analysis did not include a review of individual protocols for each study included in the pediatric exclusivity requests, which may have provided details regarding potential plans to enroll a representative study population. Another limitation included the large number of patients who lacked information related to ethnicity.

“In April 2022, the FDA issued a guidance² recommending that sponsors develop diversity plans as a measure to ensure that historically underrepresented populations are adequately represented in the clinical trials. In December 2022, the Food and Drug Omnibus Reform Act was enacted to amend to the Food, Drug & Cosmetic Act, providing FDA the authority to require diversity action plans that specify participant enrollment goals disaggregated by race, ethnicity, sex, and age for phase 3 and other pivotal trials,³” the authors wrote.

“These measures will potentially provide a mechanism to prospectively set goals for study participant representativeness in applicable pediatric clinical studies and specify measures to ensure success in meeting these goals.”

References

1. Fashoyin-Aje LA, Akalu AY, Boehmer J, Pazdur R, Donoghue M, Reaman GH. Review of racial and ethnic representation of participants enrolled in pediatric clinical trials of oncology drugs conducted through FDA written requests. JAMA Oncol. Published online January 4, 2024. doi:10.1001/jamaoncol.2023.5781
2. Diversity plans to improve enrollment of participants from underrepresented racial and ethnic populations in clinical trials: draft guidance for industry. FDA. April 2022. Accessed January 24, 2024. http://tinyurl.com/3w2be42f
3. Food and Drug Omnibus Reform Act of 2022 (FDORA) as part of the Consolidated Appropriations Act. US Congress. 2023;section 3601-3604. Pub Law No. 117-328. Accessed January 24, 2024. http://tinyurl.com/2vx5ez9c