Key Considerations Around Underrepresented Populations in Clinical Trials

Increasing diversity and accessibility in clinical trials is critical to ensure that all populations can equitably benefit from clinical research and advancements in healthcare. Excluding specific population groups can compromise the validity and generalizability of research results, making it essential to address these disparities.  

In our white paper, “Underrepresented Populations in Clinical Trials: Considerations, Diversity, Accessibility, and Patient Centricity,” we describe the current landscape of diversity and accessibility in clinical research. 

Here’s a brief overview of key considerations for including underrepresented populations in clinical research, and regulatory guidance around the topic: 

Minors and the elderly 

Clinical research involving infants and older adults carries inherent risks due to age-related physiological differences, which need to be considered when designing such trials.¹  

Specific ethnic and racial groups 

Marginalization of certain ethnic and racial groups in clinical trials continues to be a significant issue. Understanding the historical context of unethical research is essential for researchers to understand the mistrust that may exist towards trial participation within specific population groups.1 Additionally, recognizing genetic and disease predisposition differences among specific patient groups is an important consideration, with trial participation of such minority groups needed to be able to aid in uncovering population-specific health data.^1,2 

Rural communities 

Rural communities face logistical, financial, and educational barriers to trial participation.³ Efforts to address these barriers can make clinical trials more accessible and representative. 

People with disabilities 

Individuals with disabilities often experience more adverse health outcomes⁴ yet are frequently excluded from the clinical research from which they could benefit. Providing appropriate accommodations and assistive technologies is essential for their inclusion.⁵ 

International participants  

Drugs approved in one country may not be accessible in others where trials were initially conducted, raising ethical concerns. Researchers must consider the implications and strive for equitable access to treatments.6 

Regulatory viewpoints 

The FDA emphasizes the importance of avoiding unnecessary exclusions and broadening eligibility criteria to ensure trial participants reflect the intended drug users.² Recent guidance also promotes Diversity Action Plans to foster inclusion.⁷ Furthermore, the FDA’s patient-focused drug development (PFDD) guidances highlight the importance of incorporating patient perspectives into drug development.^8,9,10,11 These guidances provide strategies for collecting and using patient experience data and ensuring that clinical trials are designed with patient needs in mind.  

Conclusion 

Increasing diversity in clinical trials is paramount for advancing healthcare and providing equitable outcomes. Ensuring diversity and inclusion in clinical trials is essential to provide equitable healthcare and improve health outcomes for all. 

Download the full white paper to read a more comprehensive exploration and learn how we can work together to promote diversity, accessibility, and patient- centricity in clinical trials.  

Authors

Joan Busner, PhD has more than 35 years of experience as an academic psychiatric researcher and psychopharmacology principal investigator and founded and directed two university psychiatric clinical trials units. Dr. Busner served continuously on University Institutional Review Boards for 20 years. At Signant she has scientific and clinical responsibility for studies in mood, anxiety, pediatrics, and rare/orphan diseases. Dr. Busner is currently Affiliate Associate Professor of Psychiatry at Virginia Commonwealth University.

Bill Byrom, PhD has over 30 years’ experience as a clinical development specialist, an eClinical product strategy leader, and an industry expert in clinical outcome assessments.  He has authored 80+ publications and two industry textbooks on electronic patient-reported outcomes (ePRO), and his recent research includes the use of wearables and bring-you-own-device (BYOD) eCOA in clinical trials. At Signant he provides eCOA science expertise into customer projects and company strategy.

Greta Marie de Waal, PhD provides scientific expertise and guidance relating to the implementation of electronic clinical outcome assessments (eCOAs) and has a special focus on eDiary design and accessibility of eCOA best practices. She completed her PhD in Physiological Sciences at Stellenbosch University, which focused on the bacterial and inflammatory involvement in colorectal carcinogenesis, and has 3+ years’ experience in the clinical research industry. She has given scientific consultation for projects across a wide range of therapeutic areas, including  oncology, infectious disease, dermatology, and gastroenterology.

Lauren Crooks, MSc has comprehensive cross-functional experience within the life sciences and information technology sectors. At Signant, she combines this experience to provide scientific consultation and support to clients on the implementation of eCOA to optimize patient care and outcomes in clinical trials. She has supported eCOA projects across multiple therapeutic areas, including dermatology and neurology, with a current focus on respiratory diseases. 

References 

1. Winter, S. S., Page-Reeves, J. M., Page, K. A., Haozous, E., Solares, A., Nicole Cordova, C., & Larson, R. S. (2018). Inclusion of special populations in clinical research: important considerations and guidelines. Journal of clinical and translational research, 4(1), 56–69.
2. Final FDA guidance, 2020. Enhancing the Diversity of Clinical Trial Populations — Eligibility Criteria, Enrollment Practices, and Trial Designs.
3. Charlton, M., Schlichting, J., Chioreso, C., Ward, M., & Vikas, P. (2015). Challenges of rural cancer care in the United States. Oncology (Williston Park), 29(9), 633-640.
4. Iezzoni, L.I. (2011). Eliminating health and health care disparities among the growing population of people with disabilities. Health Affairs (Millwood), 30(10), 1947–1954.
5. DeCormier Plosky, W., Ne’eman, A., Silverman, B.C., Strauss, D.H., Francis, L.P., Stein, M.A., & Bierer, B.E. (2022). Excluding People With Disabilities From Clinical Research: Eligibility Criteria Lack Clarity And Justification. Health Affairs, 41(10), 1423-1432.
6. Miller, J., Mello, M.M., Wallach, J.D., Gudbranson, E.M., Bohlig, B., Ross, J.S., Gross, C.P., & Bach P.B. (2021). Evaluation of Drug Trials in High-, Middle-, and Low-Income Countries and Local Commercial Availability of Newly Approved Drugs. JAMA Network Open, 4(5), e217075.
7. Draft FDA guidance, 2024. Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies.
8. Final FDA guidance, 2020. Patient-Focused Drug Development: Collecting Comprehensive and Representative Input.
9. Final FDA guidance, 2022. Patient-Focused Drug Development: Methods to Identify What Is Important to Patients.
10. Draft FDA guidance, 2022. Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments.
11. Draft FDA guidance, 2023. Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints For Regulatory Decision-Making.