Improving Representation in Clinical Trials and Research Building Research Equity for Women and Underrepresented Groups (2022) / Chapter Skim
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Appendix B: Key Trends in Demographic Diversity in Clinical Trials
Appendix B: Key Trends in Demographic Diversity in Clinical Trials
Pages 191-212
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From page 191... ...
-- the historical predecessor to the Government Accountability Office -- released the report Women's Health: FDA Needs to Ensure More Study of Gender Differences in Prescription Drug Testing, ushering a new era of focus on the issue of diversity in clinical trials. Soon after, the National Institutes of Health Revitalization Act of 1993 followed, which directed the National Institutes of Health (NIH)
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From page 192... ...
made a reference encouraging the "enrollment of more diverse patient populations," and the Final Rule for Clinical Trials Registration and Results Information Submission (42 CFR Part 11) went into effect in January 2017.5 The rule requires the submission of "baseline or demographic characteristic measured in the clinical trial, including age, sex/gender, race, ethnicity (if collected under the protocol)
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PAST EVIDENCE Historically, data on population demographics across clinical trials have not been consistently reported, particularly prior to the 2017 guidance, which requires reporting by sex/gender, race, and/or ethnicity in applicable clinical trials. Evidence so far has emerged in multiple individual research reports on different aspects of diversity in clinical trials.
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From page 194... ...
More recent work has confirmed the challenge of enrolling women in some therapeutic areas: in stroke clinical trials, for instance, women have been underrepresented even after incidence and prevalence of the disease is taken into account (Carcel, 2021) , with highest underrepresentation reported in secondary prevention trials (10 percent in one study)
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From page 195... ...
A report on the diversity of mRNA vaccine trials for COVID-19 by the Kaiser Family Foundation has found a relatively higher share of white participants in both trials compared with the U.S. population, resulting in relative underrepresentation of Black and Asian participants.
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Participation of White Patients 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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FIGURE B-2 Mean % of females by year of FDA approval (non-gender-specific trials only, n = 255)
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. Participation of the Elderly Among approved drugs, participation of patients over 65 has ranged from 10 percent in 2014 to 39.9 percent in 2020, indicating a consistent increase in the share of elderly participants in trials resulting in FDA approval during this period (2021 data are yet incomplete)
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From page 199... ...
, females represented at least 50 percent of participants in trials supported by the National Institute of Environmental Health Sciences, the National Institute of Diabetes and Digestive and Kidney Diseases, and the National Institute of Child Health and Human Development across all years of reporting, and at least 50 percent of participants in at least 3 years of reporting in trials supported by the National Institute on Aging, the Clinical Center, the National Institute of Allergy and Infectious Diseases, and the National Heart, Lung, and Blood 9 Changes included the requirement for career development awards (Ks) and fellowships (Fs)
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However, ethnicity data are more available than race demographics data, as shown in Table B-1. In Figure B-6, the share of white participants in clinical trials sponsored by top NIH institutes is reported.
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2018 (%) Female 44.3 47.2 54.1 47.9 52.4 American Indian 2.1 1.3 0.8 0.7 1.0 Asian 15.1 17.2 8.4 26.4 7.8 Black/African American 12.2 14.3 10.0 10.8 13.5 Native Hawaiian/Pacific Islander 0.3 0.3 0.6 0.1 0.2 White 52.9 49.5 49.6 49.9 60.0 More than 1 race 1.1 1.1 2.0 1.9 2.3 Unknown race 1.1 1.1 2.0 1.9 2.3 Hispanic 9.8 8.1 10.8 6.7 8.5 Non-Hispanic 86.1 89.6 62.6 81.8 76.2 Unknown ethnicity 4.1 2.3 22.4 9.8 12.0 Sum of all races 84.7 84.8 73.5 91.8 87.2 Sum of all ethnicities 100.0 100.0 95.8 98.3 96.7 FIGURE B-6 Share of white participants in clinical trials by NIH institutes (top 10 institutes/centers by 2018 enrollment)
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as a share of the U.S. population was 13.4 percent in 2019.11 In Figure B-8, the share of Asian participants in clinical trials sponsored by top NIH institutes is reported.
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. The lowest representation of Hispanic participants was in trials sponsored by the National Cancer Institute and FIGURE B-9 Share of Hispanic participants in clinical trials by NIH institutes (top 10 institutes/centers by 2018 enrollment)
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Reporting of ethnicity was 100 percent complete for Phase 3 trials, where reporting was completed, and race information was available in at least 92.9 percent of trial participants in Phase 3 trials supported by NIH centers and institutes, suggesting the gaps in minority reporting originate predominantly in earlier-stage trials. We have also assessed the quality of stratified results reporting in clinical trials registered at ClinicalTrials.gov with at least one site in the United States, with annual reporting by year of primary completion shown in Figures B-10a and B-10b (stratified by funder as reported by ClinicalTrials.gov, and the difference indicated between all phases and just Phase 3 [commonly registrational]
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APPENDIX B 205 FIGURE B-10a Availability of results among all trials, by primary completion year, as of June 2021. FIGURE B-10b Availability of results among Phase 3 trials, by primary completion year, as of June 2021.
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DISCUSSION This analysis provided a brief summary of published research on diversity challenges of different types in clinical trial enrollment, and presented data on trial enrollment as reported by NIH institutes and centers for trials they sponsored, and by the FDA for trials that resulted in an approval. The reporting of demographic representation in clinical trials has historically not been consistent and comprehensive.
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From page 207... ...
Second, the participation of racial minorities in trials resulting in FDA approval has generally increased since 2014, with the number of white participants declining gradually to less than 75 percent in 2020. Finally, the participation of the elderly (over 65)
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CONCLUSIONS Some promising trends indicate more diverse trial enrollment in trials resulting in FDA approval as well as different diversity profiles of trials sponsored by NIH institutes/centers. The representativeness of trials resulting in an FDA approval has been gradually improving in several dimensions since 2014.
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2015. Demographics of clinical trials participants in pivotal clinical trials for new molecular entity drugs and biologics approved by FDA from 2010 to 2012.
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2019. Factors associated with age disparities among cancer clinical trial participants.
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Data for Phase 3 trials have a smaller share of participants with missing race or ethnicity information than all trials supported by NIH centers and institutes. SUPPLEMENTARY TABLE Demographics of Participants in Phase 3 Trials Supported by NIH Centers and Institutes 2013 (%)
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