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.
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.
Enhancing meaningful representation of diverse participants in clinical trials would help provide information about drug response and measures of safety and efficacy in populations that have been historically underrepresented and understudied, Black and Latinx communities in particular.
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:
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.
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.
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:
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:
Elements of a community-based infrastructure supporting clinical trial diversity would include:
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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