Improving Representation in Clinical Trials and Research Building Research Equity for Women and Underrepresented Groups (2022) / Chapter Skim
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2 Why Diverse Representation in Clinical Research Matters and the Current State of Representation within the Clinical Research Ecosystem
2 Why Diverse Representation in Clinical Research Matters and the Current State of Representation within the Clinical Research Ecosystem
Pages 23-46
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The next section describes the current status of clinical research representation with a focus on women and racial/ethnic minority populations. The chapter ends with a description of the clinical research ecosystem with a focus on the processes that might address diverse representativeness.
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Therefore, these analyses are complex and demand nuanced analyses with detailed and high-quality measures on genetic and non-genetic factors, and interpretation of population-specific data in clinical trials. Ensuring di verse participation in scientific studies allow for exploration of all of these factors and their interactions and is critical to the interpretations that allow for generalizability of findings to the population.
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It is important to also quantify the potential economic benefits of greater inclusion in clinical trials. The committee commissioned a study using
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(See Appendix A for more detail.) Using this model, the committee estimated the social costs of health disparities for groups that have historically been underrepresented in clinical trials and in clinical research.
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. • Given the assumption that better representation in clinical trials would be able to eliminate even modest reductions in health disparities, the value to society of better representation in these three conditions would be worth billions of dollars.
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However, to the extent that representa tion in clinical research may improve generalizability of scientific findings across a range of clinical studies for these important health states, drive new discoveries and increase innovation, improve access, and increase trust, representative clinical research may play a role in alleviating these inequities. Even if only 1 percent of these health disparities could be al
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Relative to white males, being in an underrepresented group and having diabetes is associated with an increase in mortality of 10 to 11 percent, an increase in disability of 10 to 12 percent, and a decrease in workforce participation of 9 to 12 percent. Heart disease is associated with a mortality increase of 14 to 15 percent, an increase in disability of 19 to 23 percent, and a decrease in workforce participation of 11 to 14 percent.
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Thus, increasing enroll ment of underrepresented populations would help solve the number one problem that causes clinical trials to fail, while also helping to ensure clinical data that are more representative of the whole population that could benefit from a studied intervention. Moreover, improving representation in a way that increases the overall numbers of people who enroll in studies would reduce inefficiency and
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or earlier age to start diabetes screening in Asian, Black, or Latinx populations; earlier age to start screening for lung, colorectal, breast, or prostate cancer in some populations)
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Healthy People 2020 defines a health disparity as "a particular type of health difference that is closely linked with social, economic, and/or envi ronmental disadvantage. Health disparities adversely affect groups of peo ple who have systematically experienced greater obstacles to health based on their racial or ethnic group; religion; socioeconomic status; gender; age; mental health; cognitive, sensory, or physical disability; sexual orientation or gender identity; geographic location; or other characteristics historically linked to discrimination or exclusion." Health disparities are pervasive and prevent us from achieving health equity, defined as the "attainment of the highest level of health for all people.
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In 2010, the FDA completed a report on the extent to which data submitted to the FDA was from foreign clinical trials. It found that 80 percent of approved applications for drugs and biologics contained data from clinical trials and that over half of clinical trial subjects and sites were located outside of the United States (HHS, 2010)
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Federal agencies, such as the FDA, have the authority to ensure that trials are diverse and representative. Offices such as the Office for Human Research Protections do not oversee individual studies, but have the authority to revoke assurance within an institution if it is out of compliance with the Common Rule (45 CFR 46)
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are primary consumers of clinical trial results, since this informs coverage and health decisions for individuals. CURRENT STATUS OF CLINICAL TRIAL AND CLINICAL RESEARCH PARTICIPATION: LITTLE CHANGE OVER TIME In the past three decades, diversity in clinical trials has become an important policy priority advanced by federal agencies such as the NIH Office of Research on Women's Health, the FDA Office of Women's Health and the Society for Women's Health Research, and later by the FDA Office of Minority Health and Health Equity (FDA, 2011a)
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The process includes the following key elements: requiring Good Clinical Practice training for investigators and staff; requiring investigators to submit clinical trial applications to trialspecific funding opportunity announcements (FOAs) , which require more detailed descriptions of trial design, recruitment, and retention, and analyses plans compared with more generic parent FOAs; requiring more specific notices of award, which describe principal investigator responsibilities for publication of results and data sharing, where applicable; establishing a single IRB requirement to prevent delays in study implementation; and requiring clinical trial registration and summary results with financial penalties for failure to comply (Hudson et al., 2016)
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Gender Diversity in Clinical Trials Despite the regulatory efforts to increase gender diversity in trial enrollment, evidence from the 1990s and early 2000s suggested relative underrepresentation of women and racial and ethnic minority population groups in clinical trials (Cotton, 1990; Harris and Douglas, 2000; Mak et al., 2007; Murthy et al., 2004) , which persisted until 2016, when women surpassed men in their participation in clinical trials (FDA, 2020a)
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While the committee did not examine clinical trial enrollment by specific disease burden, it is important to note that matching disease burden with trial representation is ideal, and therefore, 50 percent may not be the accurate threshold by which to measure women's participation in clinical trials and clinical research. The committee also found similarly positive trends in clinical research participation of women in NIH-sponsored trials.
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FIGURE 2-3 Participation of women in clinical trials supported by NIH institutes (top 10 institutes/centers by 2018 enrollment)
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Thus, representation of women, particularly white women, has improved in clinical research over the past decade; however, the evidence specifically on pregnant and lactating individuals, sexual- and gender-minority populations, and racial and ethnic subgroups of women is lacking (see the Racial and Ethnic Diversity in Clinical Trials section) .1 Participation of Pregnant and Lactating Individuals in Clinical Trials Nearly 4 million persons in the United States give birth every year, and 3 out of 4 gestational parents start out breastfeeding (Hamilton et al., 2021; HHS, 2022b)
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In addition to safety concerns, physiologic changes that occur during pregnancy and while lactating can affect drug metabolism, leading to increased complexity in a clinical trial. A recent example of the complexity of clinical trial design with pregnant and lactating persons is the COVID-19 vaccine trials, which excluded pregnant and lactating individuals from participating.
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Develop and implement evidence-based communication strategies with health care providers on information relevant to research on pregnant women and lactating women 7. Develop separate programs to study therapeutic products used off-patent in pregnant women and lactating women using the National Institute of Health (NIH)
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Participation of Sex, Sexual and Gender Minority Populations in Clinical Trials and Clinical Research Sex, sexual and gender minority (SGM) populations experience significant health disparities compared with their cisgender, heterosexual peers, including higher rates of cardiovascular disease, myocardial infarction, some cancers, and HIV.
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A 2004 analysis of cancer clinical trials found that Hispanic and Black patients were 28 percent and 29 percent, respectively, less likely to be enrolled than white patients after adjustment for disease incidence, age, and other factors. The difference was largest in lung cancer, where Black patients were 39 percent and Hispanic patients were 53 percent less likely to be enrolled than white patients.
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. In the commissioned analysis of the FDA Drug Trials Snapshots data, the committee found that among approved drugs, participation of white patients has ranged from 84 percent in 2014 to 73.7 percent in 2020, indicating a relatively consistent decrease in the share of white participants in trials resulting in FDA approval during this period (2021 data are yet incomplete)
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Overall, it appears that demographic trends in NIHfunded clinical research have not changed much over the years (see Appendix B for details)
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