The Importance of Patient Diversity in Clinical Trials

Rose Blackburne, MD, MBA is Vice President and Global Head of Women’s Health/General Medicine at PPD, a part of Thermo Fisher Scientific, discusses the importance of diversity in clinical trials. This concept is not new, but it is gaining momentum. The industry can leverage its experience and expertise to ensure that diverse and inclusive practices are upheld in today’s clinical trials. By using a combination of senior leadership support, cross-functional teams and enterprise-wide education, contract research organizations (CROs), biopharma and biotech companies alike can jumpstart their diversity efforts. Read more below as Rose expands on patient diversity and inclusion.

Q: Describe the momentum around diversity in clinical trials.

A: The worldwide regulatory community has played a significant role in building momentum to create more diversity in clinical trials. For example, the U.S. Food and Drug Administration updated its guidance this year to broaden its eligibility criteria, having worked over the past few decades to promote enrollment practices that would lead to clinical trials that better reflect populations most likely to use approved therapies. The European Medicines Agency and other regulatory bodies around the world are making changes as well. Regulatory guidance has considered both demographic characteristics of study populations (e.g., sex, race, ethnicity, age, location of residency) and non-demographic characteristics of populations (e.g., patients with organ dysfunction, co-morbid conditions, disabilities, those at the extremes of the weight range, and populations with diseases or conditions with low prevalence).

Despite these efforts, challenges remain to participate in clinical trials, and certain groups continue to be underrepresented. There is a need to enroll participants with a wide range of baseline characteristics that makes a study population more accurate and reflective of patients likely to take the drug if approved. This allows for assessment on the impact of safety and effectiveness of a drug within those populations. 

What we’ve really experienced in the past year is marked intentionality in building strategies to increase participation of diverse, clinically relevant population from pharmaceutical and biotech companies. Four or five years ago, there were fewer requests from customers to understand capabilities in recruiting diverse clinical trial participants.

The industry is preparing for more inclusivity in clinical trials by applying in-depth knowledge of the target disease population to study and protocol design, in addition to using data-driven research site selection practices and innovative, virtual technologies to facilitate participation from different races and ethnicities. Leveraging advanced epidemiology, data analytics and modeling also help to identify and recruit patients within specific racial and ethnic groups. PPD has been engaging in deeper ways with customers who want to understand our capabilities in operationalizing inclusive clinical trials.

Q: What are some of the reasons we lack diversity in clinical trials?

A: There are many factors leading to a lack of appropriate diversity in clinical trials.

Some populations simply aren’t aware of clinical trials. Sometimes that’s because of exposure. Research reveals that providers may also have a bias that prevents them from offering trials to certain populations. For instance, they unintentionally pre-judge whether a mom with three kids might be “too busy” to stick with a trial when, in fact, she might be really motivated.

Of course, there are some populations that are distrustful of the healthcare system or have anxieties around what’s required, and that’s what’s holding them back. We really need to look at trials through the eyes of that underrepresented population.

Then, there are patients who are interested but the burden of participating is too much. Burdens might be anything from distance to a study site to the number of in-person visits required. It can be as simple as needing funds up front to pay for bus fare to more complex requirements for frequent lab visits.

Q: How does the industry address the multifaceted challenges that impact inclusion of clinically relevant diverse clinical trial populations?

A: First, you have to understand that real change happens when everyone plays a part — changes need to be cross-functional and operationalized. Second, it’s important to realize that even small changes can have a big impact. For instance, during the pandemic, the industry relied heavily on telemedicine and home health visits to keep trials running. These technologies allowed trials to stay on track while maintaining patient safety and data integrity. We saw that small changes could really help reduce the burden on patients. That was a huge step for building diversity in clinical trials.

Another area that needs focus is training. By developing education materials for internal teams, we are training our workforce to understand and overcome barriers to inclusive clinical trials, decrease patient burden and recognize and eliminate bias.

Q: What else can help boost patient diversity?

A: Honestly, one of the best tactics is word of mouth from patients with positive trial experiences. Also, training community-based physicians about clinical research is key. Patients trust their primary physicians to discuss a clinical trial, participate in a clinical trial or be referred to a clinical trial. That means going beyond best practices for recruiting underserved populations to make sure that every step of the trial reduces the patient burden and contributes to favorable word of mouth.

Steps that are vital to achieving diversity in patient recruitment include:

• Making trials more logistically accessible and user-friendly to patients in order to reduce the burden of study visits. Examples include scheduling evening or weekend appointments, using telemedicine appointments for interval study visits or deploying home health care nurses/infusion specialists
• Incorporating new technology like telemedicine and wearables.
• Engaging and training ethnically diverse community physicians as clinical trial investigators to enhance recruitment and retention of patients by a “trusted” entity that knows them and understands the dynamics of a neighborhood.
• Staffing sites with diverse teams and ensuring that they are trained in good clinical practices, cultural competence and unconscious bias.
• Revising outtake forms to inform and thank patients for their participation.

These steps not only help patients appreciate the value of their efforts, but also turn patients into clinical trial ambassadors.

Q: What are additional strategies to further the momentum of inclusive clinical research?

A: There are many! The truth is that as much as we’re seeing momentum, there’s still so much work that we need to do. I’m encouraged, however, by the work that’s being done throughout the industry. Companies are learning that most CROs have existing and well-defined capabilities in patient recruitment, site identification and site training, and digital technology, which can be deployed intentionally to enhance inclusion of underrepresented populations. In turn, CROs understand the different ways pharmaceutical and biotech companies incorporate innovative, enterprise-wide strategies to achieve representative clinical trial populations and investigative sites into their corporate strategies and business goals. Research is being done into where and why diversity targets are being missed. PPD works collaboratively with industry thought leaders on joint task forces, supported globally by associations like Pharmaceutical Research Manufacturers of America (PhRMA), Society for Clinical Research Sites (SCRS), the Association of Clinical Research Organizations (ACRO) and others to develop cohesive strategies and industry-specific policies on diversity in clinical trials

Ensuring that clinical trials adequately represent the populations most affected by a disease is more than a nice-to-have. It aligns with ensuring strong science, patient safety and evolving regulatory requirements. Most importantly, we as an industry are obligated to lead the way in developing effective and safe products for all patients.