Gaining Trust, Giving Support, & Forgetting The "Perfect Patient": Ways To Achieve Diverse Clinical Trial Recruitment

By Pandy Opima, M.D., director study leader, Early Oncology R&D, AstraZeneca

Historically, clinical trials have lacked racial and ethnic diversity, and this has led to a disparity between clinical trial populations and more realistic patient populations. Underrepresentation in clinical trials of the populations most impacted by a disease can limit the applicability of the findings of those trials. Without proactive effort to diversify clinical trial cohorts, these cohorts can easily become homogenous, as there are many barriers to recruiting and retaining clinical trial participants of marginalized backgrounds. Shifting to a patient-centric mindset can help address diverse recruitment concerns and calls for incorporating participants of backgrounds that mirror those afflicted by the disease being studied.

Recent updates to FDA guidelines about trial diversity have left many industry leaders seeking to address data gaps due to historic underrepresentation of racially and ethnically diverse populations. These safeguards and considerations call for a greater investment of resources into trial protocols and planning in terms of both money and effort. These draft guidelines also now request sponsors to have race and ethnicity diversity plans in place at the onset of recruitment, and many leaders need an innovative strategy to build trust within the community and develop plans suited for optimal patient recruitment.

Becoming Patient-Centric

For a trial to provide the most applicable results possible, it is necessary to consider what segment of the population is most impacted by the disease and whether the trial participants mirror that patient population. Shifting the focus of clinical trials from the disease itself to the patient population most impacted by the disease in question will lead to higher-quality results with greater real-world applicability. Consider the prevalence and incidence of the disease, then identify the factors that drive population-level disparities in disease impact for racial/ethnic minorities and if these factors are intrinsic (genetic) or extrinsic (socioeconomic status, access to care, social determinants of health, etc.). This is the difference between just getting a drug approved and getting the right drug approved for the right population. Without studying the drug’s impact on the most heavily impacted segments of the populations, questions emerge about the drug’s efficacy and safety when given to that affected group. Lack of representation of the groups most impacted by a disease could result in findings that are not relevant to the most common application of the treatments under consideration.

Historically, recruiting practices haven’t reflected the most accurate representation of the impacted patient population, and shifting to a patient-first recruiting approach requires intentional action. These efforts require overcoming various societal barriers that have barred marginalized populations from participating in clinical trials. Without proactive effort to overcome these impediments facing marginalized populations, clinical trial leaders may not be able to meet the new FDA guidelines for diversity in clinical trial participation. Arriving at a patient-first clinical trial model involves rebuilding trust and removing barriers to participation.

Engendering Trust And Lending Support

With the historical misdeeds of the USPHS Syphilis Study at Tuskegee and the mistreatment of Henrietta Lacks still top of mind, many minority populations may lack trust in the medical establishment and exhibit reluctance to participate in clinical trials. Rebuilding trust and creating awareness by explaining the many benefits of participating in a trial is essential to improving diversity of clinical trial participants and providing the best possible treatments. Many patients see participation in a clinical trial as a burden, rather than a potential benefit. But clinical trials allow patients access to cutting-edge drugs and treatments that can improve or even save patients’ lives. The industry must take a role in shifting the perspective on clinical trials so that potential participants have more awareness of what they stand to gain through participation. Compounding the barrier of mistrust, economic and personal factors may also discourage marginalized populations from participating in clinical trials. Being part of a clinical trial demands time and resources. Many potential participants may require childcare, transportation, or other assistance. Additionally, trials often occur during work hours, and not all participants are able to miss work or are afforded paid time off. Trial designers ought to consider supplemental resources they may offer to participants to lessen the burden of trial participation. These resources may include monetary reimbursement for time, food, and transportation or alternate sites of care, such as mobile treatment units and telemedicine appointments.

Moving toward a patient-focused clinical trial approach involves enrolling trial participants who are representative of the world that patients live in and moving away from the “perfect patient” approach. To simplify the research process, clinical trial leaders have historically attempted to enroll participants suffering from only the disease being studied. Often, patients with comorbidities are excluded from participation in clinical trials. While this practice is intended to simplify the interpretation of the results of the trial, this standard prevents many patients from participating and it is not a true reflection of the reality of many patients. Many chronic conditions often occur in conjunction with one another, and it is essential to study the potential impact of a treatment on other co-occurring diseases.

As trial sponsors work toward establishing diversity plans at the onset of trial recruitment, TransCelerate’s Diversity of Participants in Clinical Trials Initiative recently developed the Portfolio & Program-Level Considerations for Diversity, Equity & Inclusion of Participants in Clinical Trials Toolkit. It has a patient-focused mindset and is designed to support sponsor efforts to leverage key portfolio and program/compound level diversity considerations to facilitate meaningful inclusion of diverse patient populations in the drug development life cycle. This toolkit is designed to:

• Help sponsors understand the patient populations impacted by the disease being studied and how disease impact varies for racial and ethnic groups.
• Help increase underrepresented patient populations' access to innovative targeted therapies.
• Enable the collection of more robust data to establish the safety and efficacy of investigational medical products for underrepresented diverse patient populations.

Overcoming participation barriers that marginalized populations face improves not only the accuracy of trials but also the safety of approved products for the entire population. Adopting a patient-centric mindset allows the pharmaceutical industry to provide the greatest possible impact to the populations they serve. Incorporating diverse participants into clinical trials is not only the right thing to do, but it is the smart thing to do to obtain the most meaningful and applicable results.

About the Author:

Pandy Opima, M.D., is a director study leader, Early Oncology R&D, AstraZeneca. Born and raised in Uganda, Dr. Opima is passionate about finding ways to address health disparities within underserved populations and has been advocating for clinical trial diversity since joining AstraZeneca in 2015.

In 2020, he joined the Chief Medical Office as a secondment clinical trial diversity project lead, where he supported the CMO to lead a team composed of cross functional volunteers in the development of a strategy and tactics focused on enhancing clinical trial diversity (CTD). He is also involved in collaborations with other organizations such as PhRMA, TransCelerate, and NMQF to enhance CTD across the clinical trial ecosystem.

He holds a medical degree in stomatology, MPH in epidemiology, and MBA in global management and is using his 30 years of medical, public health, and pharmaceutical experience to improve health equity.