Pragmatic Trials Funded by the National Cancer Institute, 2009-2023: A Portfolio Analysis.

Abstract

Pragmatic clinical trials aim to optimize and balance internal and external validity, creating evidence that is both rigorous and generalizable. To better understand characteristics of cancer-related pragmatic trials, we conducted a portfolio analysis of U.S. National Cancer Institute (NCI)-funded grants. We used a multi-step process to identify NCI-funded grants and select cooperative agreement mechanisms that proposed to conduct a pragmatic trial from September 2009 to December 2023. A portfolio analysis codebook was developed, refined, and applied to each grant to capture descriptive characteristics of the proposed pragmatic trial. Two independent reviewers coded the sample of grants. A total of 38 grants met inclusion criteria for full coding and analysis. Approximately half of the sample of grants were funded between 2021 to 2023 and nearly 60% used the investigator-initiated R01 grant mechanism. Studies proposed to test a range of interventions and delivery methods. Approximately one-quarter included a sample size of fewer than 1,000 participants. Many grants proposed to conduct trials in community healthcare settings and academic medical centers. Study participants reflected a range of racial/ethnic groups and included both men and women. Only 11 grants referenced key articles or tools for designing pragmatic trials. This analysis highlights the growing use of the term "pragmatic trial" in NCI-funded research. Future efforts should further amplify the importance of pragmatic trials in generating evidence to improve cancer-related outcomes. Suggestions for increasing the number and scope of rigorous pragmatic trials are discussed.