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Clinical trial representativeness and treatment intensity in a real-world sample of women with early stage breast cancer

  • Epidemiology
  • Published:
Breast Cancer Research and Treatment Aims and scope Submit manuscript

Abstract

Purpose

The extent to which evidence-based treatments are applied to populations not well represented in early stage breast cancer (EBC) trials remains unknown. This study evaluated treatment intensity for patients traditionally well represented, underrepresented, and unrepresented in clinical trials.

Methods

This retrospective cohort study used real-world data to evaluate the intensity (high or low) of EBC chemotherapy by patient characteristics (age, race and ethnicity, presence of comorbidity) denoting clinical trial representation status (well represented, underrepresented, unrepresented) for patients diagnosed from 2011 to 2020. Odds ratios (OR) from a logistic regression model was used to evaluate the association between receipt of high-intensity chemotherapy and clinical trial representation status characteristics adjusting for cancer stage and subtype.

Results

Of 970 patients with EBC, 41% were characterized as well represented, 45% as underrepresented, and 13% as unrepresented in clinical trials. In adjusted models, patients aged ≥ 70 versus 45–69 had lower odds of receiving a high-intensity treatment (OR 0.40, 95% CI 0.26–0.60), while those aged < 45 versus 45–69 had higher odds of receiving high-intensity treatment (OR 1.82, 95% CI 1.10–3.01). In predicted estimates, the proportion of patients receiving a high-intensity treatment was 87% for patients aged < 45, 79% for patients aged 45–69, and 60% for patients aged ≥ 70.

Conclusion

59% of the EBC population is not well represented in clinical trials. Age was associated with differential treatment intensity. Widening clinical trial eligibility criteria should be considered to better understand survival outcomes, toxicity effects, and ultimately make evidence-based treatment decisions using a more diverse sample.

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Availability of data, material, and code

The data that support the findings of this study are available from Flatiron Health. Restrictions apply to the availability of these data, which were used under license for this study. Data and code are available from the authors with the permission of Flatiron Health.

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Funding

The Robert Wood Johnson Foundation (#76306) provided funding for this study. Dr. Rocque is supported by an American Cancer Society Mentored Research Scholar Grants (MRSG- 17–051-01 -PCSM).

Author information

Authors and Affiliations

  1. Division of Hematology and Oncology, University of Alabama at Birmingham (UAB), WTI 240E, Birmingham, AL, 35294, USA

    Gabrielle B. Rocque, Nicole E. Caston, Jeffrey A. Franks & Courtney P. Williams

  2. UAB School of Health Professions, Birmingham, AL, USA

    Monica S. Aswani

  3. UAB School of Nursing, Birmingham, AL, USA

    Andres Azuero

  4. Department of Health Management & Policy, UCLA Fielding School of Public Health, Los Angeles, CA, USA

    Risha Gidwani

Authors
  1. Gabrielle B. Rocque
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  2. Nicole E. Caston
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  5. Monica S. Aswani
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  7. Risha Gidwani
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Contributions

Study concept/design: GR, CW, AA, RG Provision of study material or patients: GR. Data collection/assembly: NC, JF, CW, MA, AA. Data analysis and interpretation: all authors. Manuscript writing: all authors. Final approval of manuscript: all authors.

Corresponding author

Correspondence to Gabrielle B. Rocque.

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Conflict of interest

Dr. Rocque received research funding from Genentech, Pfizer, and Carevive and consulting fees for Genentech and Pfizer.

Ethical approval

This study was approved by the University of Alabama at Birmingham Institutional Review Board (IRB) and the WCG central IRB with waiver of informed consent.

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Rocque, G.B., Caston, N.E., Franks, J.A. et al. Clinical trial representativeness and treatment intensity in a real-world sample of women with early stage breast cancer. Breast Cancer Res Treat 190, 531–540 (2021). https://doi.org/10.1007/s10549-021-06381-7

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  • DOI: https://doi.org/10.1007/s10549-021-06381-7

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