Illuminating the landscape of high-level clinical trial opportunities in the All of Us Research Program

Cathy Shyr; Lina Sulieman; Paul A Harris

doi:10.1093/jamia/ocae062

Illuminating the landscape of high-level clinical trial opportunities in the All of Us Research Program

J Am Med Inform Assoc. 2024 Apr 15:ocae062. doi: 10.1093/jamia/ocae062. Online ahead of print.

Authors

Cathy Shyr¹, Lina Sulieman¹, Paul A Harris^{1

2

3}

Affiliations

¹ Department of Biomedical Informatics, Vanderbilt University Medical Center, Nashville, TN 37203, United States.
² Department of Biostatistics, Vanderbilt University Medical Center, Nashville, TN 37203, United States.
³ Department of Biomedical Engineering, Vanderbilt University, Nashville, TN 37240, United States.

PMID: 38622899
DOI: 10.1093/jamia/ocae062

Abstract

Objective: With its size and diversity, the All of Us Research Program has the potential to power and improve representation in clinical trials through ancillary studies like Nutrition for Precision Health. We sought to characterize high-level trial opportunities for the diverse participants and sponsors of future trial investment.

Materials and methods: We matched All of Us participants with available trials on ClinicalTrials.gov based on medical conditions, age, sex, and geographic location. Based on the number of matched trials, we (1) developed the Trial Opportunities Compass (TOC) to help sponsors assess trial investment portfolios, (2) characterized the landscape of trial opportunities in a phenome-wide association study (PheWAS), and (3) assessed the relationship between trial opportunities and social determinants of health (SDoH) to identify potential barriers to trial participation.

Results: Our study included 181 529 All of Us participants and 18 634 trials. The TOC identified opportunities for portfolio investment and gaps in currently available trials across federal, industrial, and academic sponsors. PheWAS results revealed an emphasis on mental disorder-related trials, with anxiety disorder having the highest adjusted increase in the number of matched trials (59% [95% CI, 57-62]; P < 1e-300). Participants from certain communities underrepresented in biomedical research, including self-reported racial and ethnic minorities, had more matched trials after adjusting for other factors. Living in a nonmetropolitan area was associated with up to 13.1 times fewer matched trials.

Discussion and conclusion: All of Us data are a valuable resource for identifying trial opportunities to inform trial portfolio planning. Characterizing these opportunities with consideration for SDoH can provide guidance on prioritizing the most pressing barriers to trial participation.

Keywords: All of Us Research Program; clinical trial diversity; electronic health records; phenome-wide association study; social determinants of health.

Abstract

Grants and funding