Abstract
Background
A lack of diversity and representation in clinical trials is an established issue in drug development, and the COVID-19 pandemic increased awareness of the problem among the general public. This awareness has led to increased pressure on drug development sponsors, as well as additional attention and regulation from federal bodies, to improve the diversity of clinical trials. This study updates existing baselines regarding demographic disparities, as well as detecting early signs that the situation may be starting to improve.
Methods
Building on an existing dataset, this study collected and analyzed pivotal trial demographic data for drugs and biologics approved by the FDA between 2007 and 2021. Demographic data were collected from applications on the FDA website and clinicaltrials.gov, and compared to indication-specific demographic data when available, or US census estimates when they were not. Regression analyses were used to test for significant trends in reporting of demographic data and representation in pivotal trials, as well as the effect of representation on clinical trial duration and FDA review.
Results
Reporting of demographic data has improved significantly for all three demographic categories (sex, racial identity, and ethnic identity) over the observed time period (p < 0.0001). During this time period, overrepresentation of white participants has decreased significantly (p < 0.0001), and representation of Black participants has increased (p = 0.0003). Other racial and ethnic identities did not show significant trends. Representation of demographic subgroups was not significant predictors of trial duration except for the representation of Black participants, which was a negative correlation, indicating that as representation of Black participants increases, trial duration decreases (p = 0.0350).
Similar content being viewed by others
Data availability
These data were derived from the following resources available in the public domain: Drugs@FDA, clinicaltrials.gov, CDC Wonder, the CDC website, the American Cancer Society, patient advocacy websites, and peer-reviewed publications.
References
Getz KA, Smith ZP, Peña Y. Quantifying patient subpopulation disparities in new drugs and biologics approved between 2007 and 2017. Ther Innov Regul Sci. 2020;54(6):1541–50. https://doi.org/10.1007/s43441-020-00181-9.
Loree JM, Anand S, Dasari A, et al. Disparity of race reporting and representation in clinical trials leading to cancer drug approvals from 2008 to 2018. JAMA Oncol. 2019;5(10):e191870. https://doi.org/10.1001/jamaoncol.2019.1870.
Bierer BE, White SA, Meloney LG, Ahmed HR, Strauss DH, Clark LT. Achieving diversity, inclusion, and equity in clinical research. : multi-regional clinical trials center of Brigham and Women’s Hospital and Harvard (MRCT Center); 2021. https://mrctcenter.org/diversity-in-clinical-research/guidance/guidance-document/. Accessed 15 June 2023.
Rubin E. Striving for diversity in research studies. N Engl J Med. 2021;385(15):1429–1430. doi:https://doi.org/10.1056/NEJMe2114651
Oh SS, Galanter J, Thakur N, et al. Diversity in clinical and biomedical research: a promise yet to be fulfilled. PLOS Med. 2015;12(12):e1001918. https://doi.org/10.1371/journal.pmed.1001918.
Hamel LM, Penner LA, Albrecht TL, Heath E, Gwede CK, Eggly S. Barriers to clinical trial enrollment in racial and ethnic minority patients with cancer. Cancer Control J Moffitt Cancer Cent. 2016;23(4):327–37.
Research C for DE and Drug Trials Snapshots. FDA. Published online May 17, 2023. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots. Accessed 17 May 2023.
Hwang TJ, Brawley OW. New federal incentives for diversity in clinical trials. N Engl J Med. 2022;387(15):1347–9. https://doi.org/10.1056/NEJMp2209043.
Diversity plans to improve enrollment of participants from underrepresented racial and ethnic populations in clinical trials guidance for industry. Draft Guidance, Food and Drug Administration; 2022:12. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/diversity-plans-improve-enrollment-participants-underrepresented-racial-and-ethnic-populations
Sen. Menendez R [D N. S.2706 - 117th congress (2021–2022): DIVERSE trials act. Published August 10, 2021. http://www.congress.gov/. Accessed 17 May 2023.
Rep. Eshoo AG [D C 18. H.R.6584 - 117th congress (2021–2022): DEPICT act. Published February 4, 2022. http://www.congress.gov/. Accessed 17 May 2023.
Diversity in Clinical Trials at FDA Gets a Boost From New Law. https://news.bloomberglaw.com/pharma-and-life-sciences/diversity-in-clinical-trials-at-fda-gets-a-boost-from-new-law. Accessed 17 May 2023.
Rep. Connolly GE [D V 11. H.R.2617 - 117th congress (2021–2022): consolidated appropriations act, 2023. Published December 29, 2022. http://www.congress.gov/. Accessed 17 May 2023.
Smith Z, Botto E, Getz K. Quantifying diversity and representation in pivotal trials leading to marketing authorization in Europe. Ther Innov Regul Sci. 2022;56(5):795–804. https://doi.org/10.1007/s43441-022-00421-0.
Top 25 Pharma & BioPharma in 2022|Contract Pharma. https://www.contractpharma.com/heaps/view/10305/1/. Accessed 15 June 2023.
Acknowledgements
This study was supported by a research grant from Clario.
Funding
This article is funded by Clario.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Conflict of interest
Zachary Smith, Emily Botto, Otis Johnson and Kenneth Getz have no conflicts to disclose. Todd Rudo discloses that he is a Clario employee and a shareholder.
Additional information
Publisher's Note
Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Rights and permissions
Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
About this article
Cite this article
Smith, Z., Botto, E., Johnson, O. et al. New Benchmarks on Demographic Disparities in Pivotal Trials Supporting FDA-Approved Drugs and Biologics. Ther Innov Regul Sci 58, 143–152 (2024). https://doi.org/10.1007/s43441-023-00579-1
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s43441-023-00579-1