Racial and Ethnic Diversity in Studies Funded Under the Best Pharmaceuticals for Children Act

BACKGROUND AND OBJECTIVES: The Best Pharmaceuticals for Children Act (BPCA) incentivizes the study of on-patent medicines in children and mandates that the National Institutes of Health sponsor research on off-patent drugs important to pediatric therapeutics. Failing to enroll cohorts that reflect the pediatric population at large restricts the generalizability of such studies. In this investigation, we evaluate racial and ethnic minority representation among participants enrolled in BPCA-sponsored studies.

METHODS: Data were obtained for all participants enrolled in 33 federally funded studies of drugs and devices conducted from 2008 through June 2020. Observed racial and ethnic distributions were compared with expected distributions by sampling Census data at the same geographic frequency as in the studies. Racial and ethnic enrollment was examined by demography, geography, study type, study burden, and expected bias. Standard descriptive statistics, χ2, generalized linear models, and linear regression were applied.

RESULTS: A total of 10 918 participants (51% male, 6.6 ± 8.2 years) were enrolled across 46 US states and 4 countries. Studies ranged from treatment outcome reviews to randomized, placebo-controlled trials. Minority enrollment was comparable to, or higher than, expected (+0.1% to +2.6%) for all groups except Asian Americans (-3.7%, P < .001). American Indian and Alaskan Native and multiracial enrollment significantly increased over the evaluation period (P < .01). There were no significant differences in racial distribution as a function of age or sex, although differences were observed on the basis of geography, study type, and study burden.

CONCLUSIONS AND RELEVANCE: This study revealed no evidence of racial and ethnic bias in enrollment for pediatric studies conducted with funding from BPCA, fulfilling the legislation's expectation to ensure adequate representation of all children.

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Work Title Racial and Ethnic Diversity in Studies Funded Under the Best Pharmaceuticals for Children Act
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Open Access
Creators
  1. Susan M. Abdel-Rahman
  2. Ian M. Paul
  3. Chi Hornik
  4. Janice E. Sullivan
  5. Kelly Wade
  6. Paula Delmore
  7. Gaurav Sharma
  8. Daniel K. Benjamin
  9. Kanecia O. Zimmerman
Keyword
  1. Alaska Natives
  2. Asian Americans
  3. Best Pharmaceuticals for Children Act
  4. Ethnic group
  5. Hispanics or Latinos
  6. Medical devices
  7. Minority groups
  8. Native Americans
  9. Patents
  10. Racial/ethnic diversity
License In Copyright (Rights Reserved)
Work Type Article
Publisher
  1. Pediatrics
Publication Date May 1, 2021
Publisher Identifier (DOI)
  1. https://doi.org/10.1542/peds.2020-042903
Deposited July 20, 2022

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Version 1
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  • Created
  • Added 2021_Diversity_in_BPCA_studies_Pediatrics.pdf
  • Added Creator Susan M. Abdel-Rahman
  • Added Creator Ian M. Paul
  • Added Creator Chi Hornik
  • Added Creator Janice E. Sullivan
  • Added Creator Kelly Wade
  • Added Creator Paula Delmore
  • Added Creator Gaurav Sharma
  • Added Creator Daniel K. Benjamin
  • Added Creator Kanecia O. Zimmerman
  • Published
  • Updated Work Title, Keyword, Description Show Changes
    Work Title
    • Racial and ethnic diversity in studies funded under the best pharmaceuticals for children act
    • Racial and Ethnic Diversity in Studies Funded Under the Best Pharmaceuticals for Children Act
    Keyword
    • Alaska Natives, Asian Americans, Best Pharmaceuticals for Children Act, Ethnic group, Hispanics or Latinos, Medical devices, Minority groups, Native Americans, Patents, Racial/ethnic diversity
    Description
    • <p>BACKGROUND AND OBJECTIVES: The Best Pharmaceuticals for Children Act (BPCA) incentivizes the study of on-patent medicines in children and mandates that the National Institutes of Health sponsor research on off-patent drugs important to pediatric therapeutics. Failing to enroll cohorts that reflect the pediatric population at large restricts the generalizability of such studies. In this investigation, we evaluate racial and ethnic minority representation among participants enrolled in BPCA-sponsored studies. METHODS: Data were obtained for all participants enrolled in 33 federally funded studies of drugs and devices conducted from 2008 through June 2020. Observed racial and ethnic distributions were compared with expected distributions by sampling Census data at the same geographic frequency as in the studies. Racial and ethnic enrollment was examined by demography, geography, study type, study burden, and expected bias. Standard descriptive statistics, χ2, generalized linear models, and linear regression were applied. RESULTS: A total of 10 918 participants (51% male, 6.6 ± 8.2 years) were enrolled across 46 US states and 4 countries. Studies ranged from treatment outcome reviews to randomized, placebo-controlled trials. Minority enrollment was comparable to, or higher than, expected (+0.1% to +2.6%) for all groups except Asian Americans (-3.7%, P &lt; .001). American Indian and Alaskan Native and multiracial enrollment significantly increased over the evaluation period (P &lt; .01). There were no significant differences in racial distribution as a function of age or sex, although differences were observed on the basis of geography, study type, and study burden. CONCLUSIONS AND RELEVANCE: This study revealed no evidence of racial and ethnic bias in enrollment for pediatric studies conducted with funding from BPCA, fulfilling the legislation's expectation to ensure adequate representation of all children. </p>
    • BACKGROUND AND OBJECTIVES: The Best Pharmaceuticals for Children Act (BPCA) incentivizes the study of on-patent medicines in children and mandates that the National Institutes of Health sponsor research on off-patent drugs important to pediatric therapeutics. Failing to enroll cohorts that reflect the pediatric population at large restricts the generalizability of such studies. In this investigation, we evaluate racial and ethnic minority representation among participants enrolled in BPCA-sponsored studies.
    • METHODS: Data were obtained for all participants enrolled in 33 federally funded studies of drugs and devices conducted from 2008 through June 2020. Observed racial and ethnic distributions were compared with expected distributions by sampling Census data at the same geographic frequency as in the studies. Racial and ethnic enrollment was examined by demography, geography, study type, study burden, and expected bias. Standard descriptive statistics, χ2, generalized linear models, and linear regression were applied.
    • RESULTS: A total of 10 918 participants (51% male, 6.6 ± 8.2 years) were enrolled across 46 US states and 4 countries. Studies ranged from treatment outcome reviews to randomized, placebo-controlled trials. Minority enrollment was comparable to, or higher than, expected (+0.1% to +2.6%) for all groups except Asian Americans (-3.7%, P &lt; .001). American Indian and Alaskan Native and multiracial enrollment significantly increased over the evaluation period (P &lt; .01). There were no significant differences in racial distribution as a function of age or sex, although differences were observed on the basis of geography, study type, and study burden.
    • CONCLUSIONS AND RELEVANCE: This study revealed no evidence of racial and ethnic bias in enrollment for pediatric studies conducted with funding from BPCA, fulfilling the legislation's expectation to ensure adequate representation of all children.
  • Updated