In this issue of Pediatrics, Brewster et al evaluate progress in the reporting of race and ethnicity and representation of diverse populations in US pediatric clinical trials between 2007 and 2020.1 The authors identify that, although the proportion of race and ethnicity reporting increased from 28% to 87% over the investigated 13-year period, there remain critical gaps in representation of Asian, Black, American Indian, and Hispanic children. Reporting was also skewed toward government-funded trials, rather than academically supported or industry-funded trials. Furthermore, enrollment patterns were overall stagnant and not aligned with population growth of historically underrepresented groups.
Injustices in health care and the significance of racism and other social determinants of health (SDoH) on clinical outcomes has been long established.2–4 In recent years, the US health care system has made concerted efforts to address the inequities in our health care provisions. This includes identifying and proposing solutions to rectify the lack of racial and ethnic diversity in clinical research studies. Diversity in clinical research is commonly hampered by mistrust that has resulted from current and historic misconduct and abuse ranging from the perpetuation of race-based medicine and other forms of discriminatory care to atrocities such as the Tuskegee Experiments.5 Systemic racism has limited access to clinical trials and precipitated an imbalance toward enrichment of predominantly white, non-Hispanic participants in clinical research.5–8 Such lack of diversity in medical advancements effectively preclude the generalizability of research to all groups. Further, inclusion of homogenous participants prevents identification of SDoH barriers to care and isolation of therapeutic efficacy, adverse events, and molecular characteristics of disease that may differ based on ancestral background.9–11
The US Food and Drug Administration (FDA) has aimed to address gaps in diversity in clinical trials specifically through development of guidelines for researchers and industry alike. Over the past decade, the FDA has provided guidelines and ultimately requirements for investigational drug and device applications and approval. These efforts have ranged from standardized reporting of safety data and outcomes across individual racial and ethnic groups to recommendations to improve diversity, such as inclusive eligibility criteria, decreased burden of trial requirements, and availability of study documents in multiple languages.12–14 Most recently in 2022, the FDA recommended investigators and industry develop “Race and Ethnicity Diversity Plans” early in study development and within phase 1 and 2 applications, far beyond the previous phase 2 and 3 recommendations.12
As we embark on recognizing and righting historic abuses, we must be aware of the current state of reporting and inclusion in clinical trials. Researchers and clinicians must be cognizant as to how race and ethnicity data are defined, categorized, and collected15 and how the data are used and analyzed,16 so that practices are inclusive and transparent for patients and reviewers. Given the number of pediatric clinical trials that are centered within tertiary or quaternary referral hospitals, it is important that these institutions invest the time and resources needed to ensure accuracy of the race and ethnicity data collected. Through accurate data reporting, we can better improve equitable access to trials and the downstream applicability of results to the broadest population of patients.
Ensuring equitable inclusion in pediatric clinical trials cannot be done without multidisciplinary and cross industry support. We must create enrollment criteria that are not excessively restrictive and accommodate for normative values that vary in range across diverse populations. We need to provide opportunities for participation that decrease patient and family burden, such as telemedicine and remote monitoring options and inclusive opportunities for patients’ understanding of trials, such as study materials in multiple languages. Financial incentives to help families meet the study requirements such as travel to and from clinic, overnight stays in hotels, and timely reimbursement to complete study-related needs should always be considered.17 Investigators should seek long-term engagement with community advocates and other stakeholders from underrepresented groups to identify needs and provide input in the design of educational resources for potential research participants. In parallel, we must advocate for systemic change.17,18 Repeat longitudinal assessments will be important as constructs of race and ethnicity evolve and as SDoH at the individual level change over time. Finally, we cannot overlook the significance of creating a diverse workforce that is as representative as the patients we serve. We must be as equally proactive about changing the culture of the institutions in which we work as we are about developing pipeline programs, opportunities for advancement, and retention practices that expand inclusion in our workforce.
Brewster and coauthors set the stage on the current state of diversity in pediatric clinical trials and the need for ongoing improvements. Our job is to translate such work to the development of sustainable practices that address persistent health care inequities in clinical research and beyond, ultimately bringing medical developments to the forefront of care in ways that effectively reach all children.
Dr Kline drafted the initial manuscript and critically reviewed and revised the final manuscript. Dr Ellison cowrote and critically reviewed and revised the manuscript for important intellectual content. Both authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.
COMPANION PAPER: A companion to this article can be found online at www.pediatrics.org/cgi/doi/10.1542/peds.2022-058552.
FUNDING: No external support.
CONFLICT OF INTEREST DISCLOSURES: The authors have indicated they have no conflicts of interest to disclose.
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