Diversity in Clinical Trials: How Pharmaceutical Companies Can Achieve Success Amidst Regulatory Focus

Diversity in clinical trials has been a long-standing issue across clinical research. Studies have historically failed to represent the patient population, with the majority of patients enrolled in trials being White, younger men. This has caused not only gaps in the industry’s understanding of how treatments in certain disease areas broadly affect diverse populations, but also inequitable access to treatments. As a result, lack of representation can also impede healthcare decision-making and future clinical development.[1]

To combat this, regulators have begun to place a greater focus on increasing diversity in clinical trials. Most recently, in 2022, the FDA renewed their guidance for the industry to support enrollment of more patients from underrepresented racial and ethnic populations. With this guidance, FDA Commissioner Robert M. Califf stated[2], “Going forward, achieving greater diversity will be a key focus throughout the FDA to facilitate the development of better treatments and better ways to fight diseases that often disproportionately impact diverse communities.”

Following the FDA’s guidance, President Biden signed the Consolidated Appropriations Act, 2023 into law, which includes the Food and Drug Omnibus Reform Act of 2022 (FDORA). As a part of FDORA, pharmaceutical companies must submit diversity action plans, which should include their diversity enrollment goals, rationale for goals, and how they intend to meet their goals, for all late-stage trials.[3] This requirement may be waived for certain trials where patient diversity would be unrealistic, such as rare diseases.

As these new rules go into effect, it’s important for pharmaceutical companies to start thinking now about how they will increase diversity in their clinical trial planning and execution. Below are two things pharma companies should consider.

1) Increase Understanding of Industry-wide Enrollment Rates

To determine reasonable diversity goals, it’s important for pharmaceutical companies to not only see how their clinical trials have performed in the past, but also understand how their performance has compared to that of the industry as a whole.

It will be especially useful to assess indication-specific enrollment rates. A recent study of clinical trials across the United States revealed that 14.9% of patients enrolled in clinical trials were Black, which is consistent with the 2020 U.S. Census. But when looking at the therapeutic area and indication level, there is actually substantial variation in Black participation. An example of this can be seen in lung cancer trials, where only 8% of participants were Black—even though the disease disproportionately affects Black patients.

Pharmaceutical companies can get a better idea of where their own clinical trials should be by diving into more granular industry-wide data on how trials have performed historically—keeping in mind the epidemiology of their targeted indications.

2) Identify Sites with Proven Track Records of Enrolling Diverse Patient Populations

In the aforementioned study, it was found that in certain indications the majority of Black patient enrollment was concentrated to only a fraction of clinical trial sites. Specifically in Alzheimer’s, migraine, lung cancer, and breast cancer trials, the majority of Black patients were enrolled by only 10% of the sites used.

To create diversity action plans, pharmaceutical companies will need to generate enrollment timeline estimates based on their target number and diversity of patients and choose the optimal sites that will meet their timelines. To do this, it will be critical for pharmaceutical companies to have visibility into and select sites with a proven record of enrolling diverse populations in target indications.

Next Steps: How to Take Action with Medidata Intelligent Trials

Under the new regulations, the FDA will be publishing further guidance on clinical trial diversity; pharmaceutical companies will only have a few months to adapt to new requirements. Pharma companies must start taking action to build a diversity strategy now in order to prepare for upcoming FDA race and ethnicity guidance.

With access to deeper insights and analytics, companies can make better-informed decisions, leveraging data to set reasonable diversity goals for their clinical trials and create realistic diversity action plans.

With Medidata AI Intelligent Trials, pharmaceutical companies can now leverage metrics from the industry’s largest scientific and operational dataset to baseline and benchmark the diversity of trials in a specific indication and identify sites that are more likely to enroll diverse patient populations quickly.

Watch our webinar and learn how to optimize site selection to enroll diverse populations and set up diversity action plans for clinical trials.

[1] National Institute on Minority Health and Health Disparities. Diversity and Inclusion in Clinical Trials. https://www.nimhd.nih.gov/resources/understanding-health-disparities/diversity-and-inclusion-in-clinical-trials.html#:~:text=It%20is%20essential%20to%20have,and%20produce%20more%20innovative%20science.

[2] U.S. Food and Drug Administration. FDA Takes Important Steps to Increase Racial and Ethnic Diversity in Clinical Trials. April 13, 2022. https://www.fda.gov/news-events/press-announcements/fda-takes-important-steps-increase-racial-and-ethnic-diversity-clinical-trials.

[3] Ropes & Gray. Congress Enacts Legislation Requiring Guidance on Clinical Research Diversity and Modernization. Jan 9, 2023. https://www.ropesgray.com/en/newsroom/alerts/2023/01/congress-enacts-legislation-requiring-guidance-on-clinical-research-diversity-and-modernization.