The FDA’s Recent Guidance on Diversifying Clinical Trials Can Revolutionize Cancer Care for Underrepresented Minorities

The Use of Real-World Evidence in the Community Health Setting Can Improve Access to Novel Therapies


The FDA’s Recent Guidance on Diversifying Clinical Trials Can Revolutionize Cancer Care for Underrepresented Minorities

Clinical research serves to grow our understanding of how to best diagnose and treat cancer. For diseases such as testicular cancer and pediatric leukemia, steady increases in survival rates continue due to advancements in clinical research; while diseases such as melanoma and chronic myeloid leukemia are revolutionizing through breakthrough clinical trials. Clinical trials offer an opportunity to understand which novel therapies may offer better outcomes than the standard of care treatments; especially critical for those patients with advanced cancers. 

All drugs must go through a clinical trial to be considered for approval however, diversity in trials has proven to not adequately represent the communities which rely on many of these novel therapies. In the FDA’s recent guidance, “Enhancing the Diversity of Clinical Trial Populations,” they share that though efforts are consistently made, challenges still arise in enrolling certain groups which perpetuates low enrollment of underrepresented minorities.

Historically, clinical trials have not reflected the diversity of the population impacted by cancer. The burden of cancer is disproportionately carried by people of color, and the system-level exclusion of these populations should be urgently addressed at every stage of the cancer treatment pipeline. Equity in representation in clinical trials is vital to creating meaningful advancements in clinical science to impact all populations affected by cancer. In addition to systematic exclusion of non-majority groups, personal distrust of the healthcare system by people of color due to racist and unethical conduct of historical studies such as the Tuskegee Study,[1] hinders accrual of diverse clinical trial participants.

The FDA’s guidance is also inclusive of underrepresentation of non-English speakers, those living in rural areas, and those with lower socioeconomic status as these populations may disproportionately suffer greater disease burden. Systematic exclusion of non-urban participants is also an obstacle; the physical location of most academic medical centers in urban areas creates the specific challenge of recruiting a geographically diverse population to clinical research[2]. Most research participants live near a major medical center or have the resources to travel. Competing commitments to employers or childcare commitments, biases trial participants to those with additional resources. Community health systems in rural areas need additional support to conduct robust clinical research programs.

This issue is not unique to cancer care; non-majority populations tend to have worse health outcomes in other major chronic diseases including hypertension, obesity, and diabetes[3]. Changing the way that clinical research is conducted may help in reducing systemic underrepresentation of some populations. Solutions, such as scalable technology and information tools, to bring greater clinical research capabilities to more geographic locations will help to diversify the participation in clinical research[4].

Additionally, as highlighted in the FDA guidance, real-world evidence in clinical research presents the opportunity to better understand the diverse populations impacted by cancer. By analyzing real-world data in the community health system setting, Syapse is able to capture a more comprehensive view of the patient journey and generate real-world evidence to inform treatment decisions of cancer patients. Our recent analysis of COVID-19 in cancer patients demonstrated that those who identify as Black, and low income groups, suffered from worse COVID-19 related outcomes[5]. As considerations are made to approve COVID-19 vaccines, it is important to consider how it will perform in the real-world setting across populations which traditionally haven’t been appropriately represented.

Lack of clinical trial diversity can lead to a lack of progress in improving outcomes for underrepresented groups. The recent FDA guidance for enhancing clinical trial participant diversity[6] provides important actionable steps. Ensuring the inclusion of people of every racial, socioeconomic, and geographic background in the cancer therapy development pipeline is a necessary step in addressing historical inequities, reshaping the clinical research and care ecosystem, and creating a socially just future for biomedical research.


Acknowledgments: Thank you to Matthew Rioth, MD, MS, FAMIA, Senior Director of Medical Oncology, for his support and thoughtful contributions to this important topic.


[1] J Natl Med Assoc. 2005 Jul;97(7):951-6.

[2] Cancer Epidemiol Biomarkers Prev 26:992-997, 2017


JAMA Oncol. 2019;5(10):e191870

J Health Care Poor Underserved. 2014 Feb; 25(1): 257–275.

[4] JCO Oncology Practice 16, no. 7 (July 01, 2020) 422-430.

[5]Clin Cancer Res 2020;26(18_Suppl):Abstract nr S10-02.