Racial and ethnic diversity among clinical trial participants is important to support science-driven strategies aimed at understanding the needs of those who are affected by the disease or condition being investigated. How can the biopharma industry achieve this? The answer lies in multistakeholder partnerships.

Enhancing clinical trial diversity

Clinical trial diversity is an imperative for health equity

Racially and ethnically diverse clinical trial participants, representative of the intended patient population likely to use a medicine once approved, can help inform the safety and effectiveness evaluation of new medicines and the use of new medicines for patients. These outcomes can increase the understanding of certain diseases that can improve prevention and treatment for all populations, particularly racially and ethnically diverse communities. Members of underrepresented communities who participate in clinical trials not only help progress scientific discoveries, but their participation can also help improve public perceptions and build public confidence about drugs when others see and hear about clinical trial participants who look like them. Lastly, enhancing diverse participation in clinical trials may also help improve health equity by providing access to potentially lifesaving therapies and quality health care that might not otherwise be available. Clinical trials are the primary route by which patients may receive unapproved investigational drugs.¹

The COVID-19 pandemic and racial unrest in 2020 that grew following the tragic deaths of George Floyd, Breonna Taylor, and so many others illuminated long-existing health and social inequities in the United States. These inequities are particularly stark in health care and health outcomes, as demonstrated by the disproportionate impact of COVID-19 in Black and Latinx communities across the United States. Significant, quantifiable health disparities have been documented across many dimensions but are especially apparent along lines of race, with racially and ethnically diverse communities experiencing barriers that can lead to poorer health overall than white populations. Systemic racism in health care is often a prime culprit. In the United States, racism has significantly influenced the way the health ecosystem is built, contributing to worse health outcomes for marginalized communities.² Enhancing diverse participation in clinical trials is one way to help foster better health outcomes and improve care for racially and ethnically diverse communities.

In response to the ongoing need to address health care disparities, stakeholders across the clinical trial ecosystem, including members of academia, government, patient advocacy, community leaders, health care providers, technology experts, and clinical research organizations (CROs) have recently increased their diversity, equity, and inclusion (DE&I) efforts around clinical trials. These efforts include increasing clinical trial awareness and enhancing clinical trial representation, recognizing that none of the stakeholders can make sustainable change alone. Some examples include:

• The FDA issued draft guidance³ on enhancing the diversity of clinical trial populations in the summer of 2019 and finalized the guidance in November 2020.⁴
• The Multi-Regional Clinical Trials (MRCT) Center of Brigham and Women’s Hospital and Harvard formed an MRCT Center Diversity Workgroup in February 2018 and published a guidance document⁵ in 2020 and a toolkit⁶ in early 2021.
• Pharmaceutical Research and Manufacturers of America (PhRMA) and its member companies published the first-ever industrywide principles on clinical trial diversity in November 2020, "Principles on Conduct of Clinical Trials & Communication of Clinical Trial Results." These principles became effective in April 2021.
• Last year, the American Medical Association partnered with the All of Us program to “gain better insights into the biological, environmental, and behavioral influences on disease to enhance prevention and treatment.”⁷
• The National Academies of Sciences, Engineering, and Medicine also published a report titled "Strategies for ensuring diversity, inclusion, and meaningful participation in clinical trials."⁸

PhRMA’s recently published principles on clinical trial diversity reinforce PhRMA member companies’ efforts and reflect their voluntary commitment to enhance diversity in future clinical trials.⁹ PhRMA’s principles state that sponsors and investigators should consider the incidence, prevalence, and severity of the condition or disease in various populations, as well as other prognostic factors that might influence the response to any intervention or outcome variable when designing clinical trials. Proactive science-driven strategies that are prospectively planned and designed into medical product development programs can help promote inclusion of diverse populations in clinical trials with an aim to understand the needs of those who are affected by the disease or condition being investigated. To support science-driven strategies for the conduct of clinical trials, it is important to identify sites where diverse patients with a particular disease or condition may be located, identify health care providers that treat underserved or underrepresented populations, and collaborate with investigators to address the goals of enrolling a diverse population in a clinical trial. Additionally, PhRMA’s principles recognize that broadening eligibility criteria for a clinical trial, when scientifically and clinically appropriate, maximizes the generalizability of trial results and the ability to understand the therapy’s benefit-risk profile across the patient population likely to use the drug in clinical practice, without jeopardizing patient safety. Patient-centric approaches can help improve the availability and quality of data that is representative of the population(s) most likely to use the drug. Study design, target enrollment population, endpoint selection, and recruitment and retention plans should be scientifically driven and responsive to the patient perspective.

PhRMA’s principles to enhance clinical trial diversity were a first step as the industry came together to address this important topic. Many PhRMA member companies are taking action to identify potential leading practices and implement strategies to increase clinical trial diversity. A survey of and interviews with PhRMA member companies were conducted in January 2021 to identify current industry practices, inform potential leading practices and future opportunities to enhance clinical trial diversity, and better understand how to measure progress and success in enhancing clinical trial diversity. Surveyed PhRMA member companies are all committed to enhancing clinical trial diversity though currently they are approaching this goal in different ways. The approach that most companies reported as leading to increased enrollment or retention of patients from racially and ethnically diverse communities is identifying and engaging diverse sites or locations in underserved communities. Other strategies PhRMA member companies are pursuing include adapting protocol design (including feedback from patients), reducing barriers to access, increasing patient education and awareness, and pursuing partnerships with patient organizations and community leaders. Member company interviews and discussions conducted alongside the survey supported the conclusion that partnerships in the community are critical and that the industry cannot improve diversity in clinical trial participation alone or as individual companies.

Recognizing the need to bring stakeholders of the clinical trial ecosystem and underrepresented communities together, PhRMA convened a large, multistakeholder workshop dedicated to the topic of building partnerships to improve clinical trial diversity in June 2021, titled “Partnering for health equity: Advancing research through representative clinical trials.” During this two-day virtual workshop, over 500 stakeholders from 150 organizations interested in improving clinical trial diversity, including the FDA and other government officials, gathered to discuss the need to rebuild trust in underrepresented communities; the root causes of health inequity; patient perspective in clinical trials; the community perception of clinical trials; the critical role of community-based providers and health systems; clinical trial digital innovations; and innovative efforts focused on diversity across the clinical trial ecosystem.

The workshop provided a forum to acknowledge the reality of the past and present experiences of racially and ethnically diverse communities, to have listening sessions with patients and community leaders, present recent research from stakeholders, and discuss cross-stakeholder solutions to drive change. The workshop concluded with a discussion on how these key stakeholders, along with trusted community leaders in the clinical trial ecosystem, can partner to build a sustainable community-based infrastructure focused on enhancing diversity in clinical trial participation in underserved communities.

Stakeholders identify opportunities to improve diversity in clinical trial participation

The workshop resulted from several months of joint research conducted collaboratively between PhRMA and Deloitte’s Center for Health Solutions. This research included a literature review, a survey and interviews with PhRMA member companies, community focus groups and surveys, and interviews with patient advocates and clinical research stakeholders.

This research concluded that barriers to enhancing clinical trial diversity include lack of awareness of clinical trials, lack of access, and mistrust by underrepresented communities and populations.

Participants in the research and workshop pointed to the importance of meeting patients and caregivers where they live to improve awareness, access, and trust. Working with community leaders and networks to rebuild trust and trustworthiness and to allow bidirectional dialogue could make clinical trials more accessible to patients who want to participate. Stakeholders emphasized that it is critical to have a patient-centric approach by seeking patient input throughout the medical product development process.

Further, reliable information from trusted messengers in the community is critical to empower individuals to make decisions about participation in clinical trials. Communications should be culturally sensitive and consider unconscious/unintended bias. Partnering with community leaders for local engagement is necessary to build trust and trustworthiness. Many community leaders (e.g., faith-based organizations, civic leaders, educators, barbers, and beauty shops) and health care providers (e.g., nurses, pharmacists) can serve as trusted messengers for communicating health information. For example, nurses can engage with the communities in which they live and can serve as advocates and educators on clinical trials. This is especially important in rural communities. Community health workers are community extenders for underserved populations and can be valuable resources to reach underrepresented populations.

Ecosystem stakeholders recommend a path forward

Shifting the paradigm will likely require substantial cross-stakeholder commitment and collaboration. Partnering to build scalable and sustainable relationships and solutions, embedded within underrepresented communities and that extend beyond any one clinical trial can be key elements to help ensure success. Patients can benefit from solutions cultivated by the clinical trial ecosystem coming together and developing long-term strategies across multiple disease areas. Some possible collaborations include:

• Partnering with stakeholders across the clinical trial ecosystem (patient groups, community members, clinical research sites, CROs, academia, nonprofit and advocacy organizations, federal and state agencies, industry, etc.) to establish a sustainable, community-based clinical trial infrastructure
• Establishing relationships with the target community through community leaders, Historically Black Colleges and Universities (HBCUs), other minority-serving institutions (e.g., Indian Health Service), Federally Qualified Health Centers (FQHCs), patients, and others to provide outreach, training and education, and mentorship/job pathways
• Adopting enhanced data collection capabilities to help support appropriate collection and sharing of racial and ethnic data and real-world data (RWD) through a cloud-based platform
• Utilizing a common source of disease incidence and prevalence by race and ethnicity to inform clinical trial design
• Developing patient-friendly resources that make it easier to identify and enroll in relevant clinical trials

Research participants as well as the multistakeholder workshop discussions emphasized the tremendous potential of supporting community-based clinical trial sites. The current clinical trial ecosystem depends on independent pharmaceutical companies or academic researchers preparing and training clinical research sites for individual trials. These disparate sites all have varying levels of capability and connectivity to the community and are most often driven by the design of the trial. It is not sustainable for any one company or researcher to adequately resource new potential sites focused on enhancing clinical trial diversity, if only one clinical trial will be conducted. On the other hand, dedicated resources supporting a network of clinical trial sites in the communities that serve underrepresented populations could over time create a sustainable national infrastructure focused on enhancing clinical trial diversity.

There can be several benefits to a community-based clinical trial infrastructure. First and foremost, the health care providers that serve those communities are often best positioned to engender trust and establish relationships with trusted messengers that live in those communities. Examples of some of these community health providers include Catholic health centers, Federally Qualified Health Centers (FQHCs), military health, HBCUs and other minority-serving institutions, pharmacies, or other academic centers with established, trusted relationships with the community. Using geotargeting techniques, other new site locations may be identified. Stakeholders that support this network could invest in increased training and education among investigators and site staff, including supporting new sites in these underserved communities. Those participating in the network would be able to share potential leading practices and learn from each other. Industry members within this network may also provide access to a consistent pipeline of research studies to create opportunities for investigators and sustain the network of ready clinical trial sites over time.

Workshop participants and others who participated in our research identified steps the research clinical trial ecosystem should take to change the current paradigm and create a sustainable community-based infrastructure that is focused on clinical trial diversity, including:

• Creating a network of clinical trial sites in underserved communities to make access easier so that those who want to participate can: Infrastructure stakeholders should develop research sites that meet potential participants where they already receive care, including nontraditional locations such as FQHCs or Catholic health systems. These providers may be best positioned to engender trust and establish relationships with trusted messengers and leaders that are reflective of the cultural norms and demographics of the community, and with the right support and investment could be scaled into clinical trial sites in underserved communities. Infrastructure efforts should consider and leverage—to the extent possible—any existing networks or structures involved in the conduct of clinical trials. It is critical for clinical trial sponsors to provide necessary funding and resources to sites and nontraditional locations that serve underrepresented populations.
• Developing a diverse pool of investigators and staff at sites in underserved communities: Developing diverse staff to support clinical trials and serve as trusted sources of information requires good clinical practice training as well as training in cultural competence and unconscious/implicit bias, mentoring, and opportunities and resources for investigators and site personnel to use when engaging with the community. Establishing a pipeline of clinical trial opportunities can also be critical to help ensure continued engagement and commitment.
• Establishing long-term relationships and investing in the community: It is critical to build long-lasting relationships rooted in a shared commitment to improve health equity in the communities being approached. These relationships should be established well before a clinical trial is proposed. Stakeholders of a community-based clinical trial infrastructure should invest in uplifting the community in ways that expand beyond their specific objectives. Investments could range from health education to donating buildings for educational programs or volunteering to help those in need. It is important to understand the diverse needs of the communities being served, including who the trusted messengers are. This could look different in each community and could, for example, include barber shops and beauty salons, church and ministerial staff, local food shops, consulates, and other community organizations. These community leaders and trusted messengers could serve as liaisons that can provide information about clinical trials and what participation means for patients.
• Engaging the community in bidirectional conversations: Educating the community on opportunities to improve health, including through clinical trials, can be a key component of sustainable relationships. Emphasis should be placed on improving patient health first. Education helps to build trust by empowering community leaders and other members of the community. Empowered patients and their families are enabled to make informed choices about clinical trial participation. At the same time, sponsors should seek community input on clinical trial design, including endpoints of interest and elements of design that might impact patients' ability to participate (e.g., testing requirements). Sponsors should emphasize that without generous volunteer participation, the development of new medicines would not be possible. They should also share research findings with the participants once a trial is over and consider their research questions and needs in future trials. Sponsors of clinical research should engage in bidirectional conversations with the community to increase education, awareness, access, and enrollment into clinical trials in a way that is tailored for each individual community and made available to patients who want to participate. This includes offering information in various languages and taking into account varying levels of health literacy.
• Providing sustainable support and standardized platforms: Some community clinical trial sites may not have the technology platforms and databases in place to help identify patients for enrollment in clinical trials. Investigators who participated in the workshop pointed out that building a data infrastructure is a necessary first step to enable sites to participate in clinical trials. This type of infrastructure, leveraging real world data, could make it seamless for investigators to identify and engage with patients appropriate for clinical trials with minimal disruption to existing workflows. A community-based clinical trial infrastructure should include the development of standard data collection, baseline measurements to improve data on race and ethnicity, and measures of success for tracking progress on enhanced participation in clinical trials of diverse populations.

Discussions at the multistakeholder workshop concluded with recognition across the clinical trial ecosystem that a partnership to address clinical trial diversity is needed. A sustainable community-based partnership that begins well before clinical trials start can be essential to both building trust and creating a successful infrastructure that serves underrepresented populations. Sustainability of the sites (e.g., a pipeline of clinical trials, consistent funding) can enable them to focus on building and maintaining community relationships. A toolbox of proven strategies of can minimize the need to reinvent the wheel with each new study or the current one-and-done clinical trial approach that results when a site is trained for a particular study but does not have a steady stream of additional studies following behind. Technology and data investments will be needed to measure and track progress moving forward. Many industry, health systems, academic institutions, government entities, patient advocacy, community leaders, technology experts, and CROs are working toward partnerships dedicated to enhancing clinical trial diversity through the formation of a sustainable, community-based clinical trial infrastructure. Ultimately, investing in and supporting underserved communities can have a longer-term impact on the health and well-being of those communities beyond improving clinical trial diversity.

The in-depth report can be found here.

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