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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
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1

The Committee’s Task

Throughout history, biomedical research has contributed enormously to progress in treating and preventing disease and overall life expectancy. Such progress requires clinical, translational, and population studies—including clinical trials, observational studies, and implementation designs—in which people volunteer as participants to help researchers find answers to specific questions about how health, disease, and therapeutic interventions work.1 Studies such as these are critical for ensuring that fundamental discovery translates into improvements in human health. The data and evidence these studies generate also are critical for securing reimbursement—and therefore patient access—to therapeutic interventions. Yet, advancing the nation’s capacity to protect and improve health is unobtainable if large swaths of the U.S. population, often those with the greatest health challenges (or most premature morbidity and mortality), are less able to benefit from these discoveries because they are not adequately represented in clinical research studies.

The scientific necessity to improve research equity is urgent. The 2020 U.S. Census found that the number of people who identify as white has decreased for the first time since a census started being taken in 1790 (Bahrampour and Mellnik, 2021), and despite the country becoming more diverse, the nation’s health disparities persist. Without major advancements in the inclusion of traditionally underrepresented groups in health research, meaningful reductions in racial and ethnic inequities in chronic diseases such as diabetes, cancer, and Alzheimer’s disease remain unlikely. The critical need for research findings to generalize to

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1 In many implementation designs there is a waiver of consent, so participants do not overtly volunteer.

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

the entire U.S.US population has also long been recognized and underscored by the National Institutes of Health (NIH) mandate in the Public Health Service Act, Section 492B (42 U.S.C. § 289a-2), to ensure inclusion of women and minority populations in all NIH-funded clinical research in a manner that is appropriate to the study question; however, more needs to be done to achieve the goals of representation and inclusion.

As described in detail in Chapter 2, lack of representation has a range of serious consequences. Failing to adequately represent underrepresented and excluded populations in clinical trials and research may limit scientific innovation and exacerbate health disparities. Lack of representation can shift the direction of research toward majority groups, feed mistrust, and ultimately may impugn the integrity of the scientific enterprise or of a specific therapeutic or discovery. Gaps in representation are particularly problematic when one considers the stark and deep disparities in disease burden experienced by the same populations that have not been represented in clinical research. In short, lack of representation compromises generalizability of clinical research findings to the U.S.population, hinders innovation, compounds low accrual rates causing trials to fail, leads to lack of effective medical interventions, undermines trust, compounds health disparities, and costs the United States billions of dollars each year.

Some may note that even if clinical trials look like the population affected by the disease under study, the trials likely are not powered to examine subgroup differences. However, inclusion of underrepresented and excluded populations in clinical trials is crucial—even and perhaps especially when these groups are small in number and even in studies that are not adequately powered to draw conclusions about specific populations. The need for equity demands it. In many settings, clinical research represents the best available health-care option. Individuals from underrepresented populations must have the opportunity to access it.

Additionally, studies that are not powered to draw conclusions about subgroups can and do lead to testable hypotheses that can and should be followed up in subsequent research that uses oversampling to achieve adequate power. Studies that do not include individuals from underrepresented populations yield neither knowledge nor testable hypotheses about these groups, and instead perpetuate and exacerbate gaps in access, gaps in knowledge, and disparities in health outcomes.

Although the committee routinely calls for clinical trials to represent the populations affected by the disease under study, there are certainly cases where this would not apply. For example, some trials may oversample certain populations for which we have limited information or where diversity may be limited by the geography of the trial site. The committee’s recommendations reflect this nuance, with the opportunity to provide justification when trial enrollment does not match the demographics of the intended patient population. However, the committee believes that in aggregate, clinical trials should look like the populations affected by the disease under study.

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

While Chapter 2 presents the committee’s detailed analysis of these costs and consequences of a lack of inclusion in clinical research, and provides an overview of the current state of representation within the clinical research ecosystem, this chapter describes the committee’s process in carrying out the study and the statement of task that guided the committee’s work, and it outlines the structure of the report.

COMMITTEE TASK AND APPROACH

In the 2020 appropriations process, Congress mandated that the NIH fund a National Academies of Sciences, Engineering, and Medicine study “examining and quantifying the long-term medical and economic impacts of the inclusion of women and racial and ethnic minority population groups in biomedical research and subsequent translational work” (see Box 1-1). In accordance with this mandate, the National Academies appointed and tasked this diverse committee of experts with carrying out a study that would identify policies, procedures, programs, or projects aimed at increasing the inclusion of these groups in clinical research and the specific strategies used by those conducting clinical trials and clinical and translational research to improve diversity and inclusion. The committee was tasked with modeling the potential economic benefits of full inclusion of men, women, and racial and ethnic groups in clinical research and to highlight new programs and interventions in medical centers and other clinical settings designed to increase participation. The full Statement of Task can be found in Box 1-2.

The Committee on Improving the Representation of Women and Underrepresented Minorities in Clinical Trials and Research consisted of 13 members with a broad range of expertise, including health disparities, health-care policy, health economics, community-engaged research, running diverse clinical trials in academia and biopharma, nursing science, clinical outreach to medically underserved populations, and patient-centered care.

The committee deliberated over five virtual meetings and one hybrid meeting between January 2021 and December 2021, as well as over many conference calls between January 2022 and April 2022. In addition to the closed meetings, the committee held three virtual public workshops, where outside speakers were invited to inform the committee’s deliberations and members of the public were invited to comment and ask questions. The speakers provided valuable input on a wide range of topics that helped to inform and shape the committee’s approach to the report. In addition, the committee commissioned five papers to address various aspects of the Statement of Task. First, to accomplish the economic analysis, the committee worked with authors to commission an economic analysis using the Future Elderly Model to estimate the social costs of health disparities for groups that have historically been underrepresented in clinical trials and clinical research (see Chapter 2 for a summary of this work and Appendix A for the full analysis). Second, the committee commissioned a paper to estimate the current demographic

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

status of clinical trial and clinical research participants, as this analysis is not readily available in the literature (see Chapter 2 for a summary of this work and Appendix B for the full analysis). Third, the committee commissioned a paper on successful facilitators for having representative clinical trials, based on 20 qualitative interviews conducted in 2021 with research teams (investigators and staff) involved in clinical trials that successfully achieved diverse enrollment (see the full analysis in Appendix C). The remaining two commissioned papers were reviews of the literature: one focusing on federal policies that have influenced the diversity of clinical trials and clinical research (see Chapter 3) and the second focusing on the structural barriers to having representative trial enrollment (forming the basis of Chapter 4). The authors of these papers worked closely with committee members to help scope the papers, develop methodology, and ultimately ensure that the papers informed the report. These commissioned analyses were critical for helping the committee meet its task and form the basis of this report.

The committee notes that although the Statement of Task is rather broad, the task of this study was to focus specifically on women and racial and ethnic minority population groups in clinical trials and clinical research. Although it was out of scope for the committee to examine other excluded and underrepresented populations in clinical trials and clinical research, such as children, older adults,

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

persons with disabilities, rural and frontier populations, and more, the committee would like to underscore that these are critical topics for future work. In addition, the committee’s scope was narrowed to clinical trials and clinical research, but the topic of database-based studies is another important area for future work, as these studies also suffer from representation issues and data-reporting issues.

The committee recognizes the importance of including the participant, patient, and caregiver voice throughout the research process, from study design and recruitment to the dissemination of findings. Centering these voices are essential to conducting research that is more equitable and responsive to the needs of communities. However, the committee faced several structural barriers that limited the representation of these voices in the report, many of which the committee addresses throughout the following chapters, including challenges with remuneration and agency policies. While we recognize the need for a more intentional approach

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

and room for improvement, the committee made efforts to include the perspective of patient advocacy stakeholders throughout the development of this report, including as speakers in the committee’s public forums and by including representatives from the patient advocacy community on the committee. In addition, the committee cites studies throughout the report that do include the voices of community members to bring these perspectives from the literature into this report.

Throughout the report, the committee uses examples from specific disease areas to illustrate points and provide evidence. Although these diseases are mentioned throughout the report, it is important to note this study focuses on diversifying clinical trials and clinical research across all disease areas.

DEFINITION OF TERMS

Throughout this report the committee uses several terms that would benefit from definition. Clinical research or medical research includes (1) research conducted with human subjects or on material of human origin for which direct human participant interaction is needed, including clinical trials; (2) epidemiologic and behavioral studies; and (3) outcomes, health services, and large database research.

The very task of this committee—to examine the long-term medical and economic impacts of including women and racial and ethnic minority populations in clinical trials and biomedical research, and to identify and describe policies and programs that support inclusion—raised and continues to raise critical questions about the meaning of its terms. The committee acknowledges and believes race is a social construct that has, from its inception to the present day, exclusively benefited people who identify as or appear to be white or European and profoundly harmed those who do not. It further acknowledges that the social construction of race, and its racist derivatives have created measurable, sustained, and life-threatening biological outcomes. To meet our statement of task, and to look beyond it toward a just and equitable society, we use the imperfect language we have to describe and offer resolutions for observable inequities in health outcomes and clinical research. To that end, the committee chose the term underrepresented and excluded populations as the broadest term to refer to the populations and communities that are the focus of this report. The term “Underrepresented” calls attention to studies, research foci, funding streams, and other components of the research ecosystem that draw from populations whose demographic characteristics are not representative of the people who ought to benefit from it. “Excluded” emphasizes that the choices and actions of various entities in the research ecosystem result in de facto exclusion of people from underrepresented groups, even when these individuals meet inclusion criteria. We also note that evidence-based exclusion criteria (such as for pregnant and lactating individuals) can result in underrepresentation.2 So while most women

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2 The committee recognizes that exclusion criteria are necessary, and that the inclusion of pregnant and lactating individuals may require special consideration, as discussed at length in Chapter 2.

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

and racial and ethnic minority population groups are technically included, they are often underenrolled. Thus, in this report the committee shifted the focus away from inclusion toward representativeness, which is defined as matching the self-reported demographics of those enrolled in clinical trials and clinical research to the demographic characteristics of the population affected by the particular illness or condition under study, including self-reported age, sex, gender, race, ethnicity, and socioeconomic status.

To describe excluded and underrepresented populations, the committee uses the terms Black, white, Latinx, American Indian/Alaska Native, Native Hawaiian/Pacific Islander, and Asian American in the broadest sense, acknowledging that these terms do not capture the complexity and intersectionality between and within these groups. When possible, more specific terms are used. However, when describing published research, the committee uses the same language as the referenced publication to ensure accuracy. Therefore, the reader may see inconsistencies in terminology throughout the report due to inconsistencies in language throughout the literature. Literature on other underrepresented groups, including the elderly, LGBTQIA+ individuals, transgender/gender nonbinary individuals, and residents of rural and frontier areas, is not nearly as complete or detailed as that for ethnic/racial groups. The literature that does exist is most often at the intersection of these groups with the ethnic/racial minority groups of which they are a part (older Hmong populations, Black women). The committee therefore discusses these groups only when relevant and/or specific data are available.

ORGANIZATION OF THE REPORT

In the chapters that follow, the committee provides an overview of the threats posed by lack of representation in clinical studies, the current status of representation in clinical research participation, and the clinical research ecosystem (Chapter 2); offers an overview of the existing landscape of current and past federal policies and practices aimed at addressing this issue (Chapter 3); outlines the range of barriers to full inclusion of underrepresented and excluded populations in clinical trials and research (Chapter 4); identifies facilitators of successful inclusion in clinical research (Chapter 5); and summarizes key principles and recommendations for how a range of stakeholders can take action to address this critical national issue (Chapter 6).

The committee’s work focuses on tangible actions that must urgently be taken within the context of the existing structures of the clinical research ecosystem in order to achieve the goals of representation and inclusion. In addition, the committee recognizes that a more transformative and equitable future is possible and desirable; the epilogue describes such a potential vision.

Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×

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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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Suggested Citation:"1 The Committee's Task." National Academies of Sciences, Engineering, and Medicine. 2022. Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups. Washington, DC: The National Academies Press. doi: 10.17226/26479.
×
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The United States has long made substantial investments in clinical research with the goal of improving the health and well-being of our nation. There is no doubt that these investments have contributed significantly to treating and preventing disease and extending human life. Nevertheless, clinical research faces a critical shortcoming. Currently, large swaths of the U.S. population, and those that often face the greatest health challenges, are less able to benefit from these discoveries because they are not adequately represented in clinical research studies. While progress has been made with representation of white women in clinical trials and clinical research, there has been little progress in the last three decades to increase participation of racial and ethnic minority population groups. This underrepresentation is compounding health disparities, with serious consequences for underrepresented groups and for the nation.

At the request of Congress, Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups identifies policies, procedures, programs, or projects aimed at increasing the inclusion of these groups in clinical research and the specific strategies used by those conducting clinical trials and clinical and translational research to improve diversity and inclusion. This report models the potential economic benefits of full inclusion of men, women, and racial and ethnic groups in clinical research and highlights new programs and interventions in medical centers and other clinical settings designed to increase participation.

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