Clinical Researcher—April 2021 (Volume 35, Issue 3)

GOOD MANAGEMENT PRACTICE

Scott Gray

It’s no secret that patient diversity is a major issue in clinical trials, and that a lack of diversity makes it difficult to measure efficacy and safety. However, even given the U.S. Food and Drug Administration’s recent guidance, there is not a lot of actionable advice for trial sponsors and research organizations looking to increase enrollment among historically underrepresented groups. The conversation has stalled at why, and the time is long overdue to start talking about how.

The Benefits of Diversity in Clinical Trials

One goal of all clinical trials should be to represent the affected population as a whole. Because most conditions are not specific to a single demographic, sample populations shouldn’t be either. Ethically, a lack of representation in trials raises concerns about ensuring equal access to new treatments and cures. A person’s socioeconomic status or demographics should not exclude them from participating in a clinical trial or from being assured the approved treatment will be safe and effective for them once approved.

Historically, 60% to 70% of clinical trial participants have been white males because they were the ones who could afford the cost of travel and the time off work to participate. Today, ethnic diversity in clinical trials continues to be an issue. For instance, in the United States, African Americans make up 13.4% of the population, but only 5% of clinical trial participants. The disparity is even worse for the Hispanic or Latino population; they make up 18.1% of the U.S. population and only 1% of trial participants. Asian Americans account for 6% of the population and only 1% in clinical trials, and two-thirds of clinical trials are absent any Native American participation.

Such lack of diversity is not limited to U.S. trials, however. One recent study of trials across 29 countries over the past 21 years shows that 86% of participants were white. The disparity is concerning because it is scientifically crucial to understand how a new drug or therapy affects patients of varying ages, genders, and ethnic backgrounds, as reactions to many new treatments are proven to differ depending on the individual’s demographics. For example, African Americans react differently than white populations to certain blood thinners and asthma medications.

Proportional representation in clinical trials helps to accurately understand how or if a drug affects a particular demographic group negatively or differently. More investigation can then define the issue or address it before the therapy receives approval. Representation is essential because once a drug is commercially available, access will not be restricted to certain demographics, so a clear understanding of each demographic’s reaction is an imperative before going to market.

Practical Methods for Overcoming Barriers to Participation

To achieve our shared diversity goals, we need to directly address the challenges historically underrepresented populations experience in clinical trial participation. Many of these barriers could be mitigated through improved communication with targeted populations. Educating each group on the importance of trial participation and the potential benefits for participants can go a long way to increase enrollment. For instance, ensuring these groups are aware of the compensation and travel stipends available can sway patients to become participants. In the U.S., this might include discussing the Ensuring Access Act, which deducts the first $2,000 a patient receives for clinical trial participation from federal benefit eligibility determinations and aims to remove some of the financial barriers lower-income families experience.

This type of education requires dedicated resources for community outreach, including building relationships with community leaders in underrepresented demographics to act as knowledge ambassadors. It should also include direct communication with patient advocacy groups, patients and caregivers, and physicians to help inform the design of clinical trial protocols to mitigate any steps that discourage participation.

Additionally, trials can be designed with more resources and communications to make trial participation less burdensome for participants. For example, choosing payment or reimbursement methods that best serve a particular population and partnering with companies that manage patient logistics and expense prepayment can significantly reduce the barriers to participation and improve retention rates in trials.

By providing direct support to patients from enrollment through the end of the clinical trial, these services ease the financial, logistical, and psychological barriers to participation. Where possible, increasing site locations in communities with high populations of underrepresented populations and holding recruitment events during weekend and evening hours can also make trials more accessible.

Addressing these challenges may not only increase participation, but also increase the participants’ satisfaction with the entire experience.

How Dedicated Patient Coordinators Impact Trust and Retention

Recruiting diverse populations to participate in trials is only the first challenge. Participants must be supported and remain in the trials through completion in order to be represented in the trial outcomes. Partnering with a company that provides patient logistics coordination and patient support may be one of the best tactics available to improve retention rates while reducing the burden on trial staff. By providing coordinators who are assigned to each participant through the length of the trial, patients receive personalized support for travel, financial prepayments or reimbursements, pandemic-related issues, relocation services, and other logistical support they may need.

These services are most effective when coordinators provide one-on-one support and are matched with participants based on things like location or time zone, culture, and language. This approach reduces the burden on participants and caregivers, allowing them to focus on their health and improves trust and satisfaction with the experience, increasing the likelihood that a participant will remain in the trial through completion.

Unique Challenges in Global Studies and Studies of Rare Diseases

Achieving diversity in clinical studies involving rare and ultra-rare diseases presents its own challenges. There are significantly smaller patient populations available in rare disease studies, so special efforts are necessary to recruit and retain them. In the rare disease space, a specific clinical trial may be the patient’s only hope for medical treatment, so including them isn’t just ethically necessary; it’s also medically necessary.

In these cases, extensive travel is generally unavoidable as patients may be spread out across the world, or in the case of a genetic condition, multiple patients may be concentrated in one specific geographical area in a different country from the clinical site. For example, a significant amount of research may be done at a single site in Frankfurt, Germany, but the babies who would benefit from this life-saving research are clustered in China and India. In these cases, entire families may need to relocate to Germany for months, requiring airline travel where medical equipment is permitted on-board the plane, apartment rentals, translation services so the family can communicate with medical staff, and more.

Additionally, patients may be very ill or have diseases that are debilitating and impact cognition or mobility. A patient’s location or ability level should have no bearing on his or her right to participate in a clinical trial, but these factors do present challenges.

This is another area where patient logistics companies can provide invaluable support to improve trial performance. Their trained coordinators understand the medical challenges faced by patients and caregivers and can provide complex cross-border support, translation and interpretation services, and logistics management for participants in remote locations or with complex medical needs. Their needs might include visa and passport procurement, managing exchange rates and reimbursement, navigating local regulations, and coordinating multi-leg travel and relocation efforts.

When you consider the complicated nature of participating in rare and ultra-rare disease trials, it is no wonder that drop-out rates are typically relatively high for these types of studies. It has been shown that 85% of all clinical trials are delayed due to not retaining enough participants to continue, and that the cost of just one patient dropping out of a rare disease trial far exceeds the average cost of losing a participant in a common clinical trial of approximately $20,000. Considering how expensive and time-consuming it is to lose a patient, investing in services that increase retention rates and retain diverse participant pools once they are recruited is a logical choice.

Diversity is Our Responsibility

We can all agree that achieving diversity in clinical trials is complicated. However, with these specific steps to improve community outreach, education, and accessibility—along with an investment in retaining those populations once they are recruited—adequate representation is achievable.